Materiały źródłowe

• #1 FDA Approves First Treatment for Hutchinson-Gilford Progeria Syndrome and Some Progeroid Laminopathies | FDA

  https://www.fda.gov/news-events/press-announcements/fda-approves-first-treatment-hutchinson-gilford-progeria-syndrome-and-some-progeroid-laminopathies

  Today, the U.S. Food and Drug Administration approved Zokinvy (lonafarnib) capsules to reduce the risk of death due to Hutchinson-Gilford progeria syndrome and for the treatment of certain processing-deficient progeroid laminopathies in patients one year of age and older. […] With todays approval, Zokinvy is the first FDA-approved medication for these devastating diseases. […] Before todays approval, the only treatment options included supportive care and therapies directed towards the complications arising from the disease. […] Zokinvy, a farnesyltransferase inhibitor, is an oral medication that helps prevent the buildup of defective progerin or progerin-like protein. […] The effectiveness of Zokinvy for the treatment of Hutchinson-Gilford progeria syndrome was demonstrated in 62 patients from two single-arm trials that were compared to matched, untreated patients from a separate natural history study. […] Zokinvys approval for the treatment of certain processing-deficient progeroid laminopathies that are very rare took into account similarities in the underlying genetic mechanism of disease and other available data.

• #2 Sentynl Therapeutics Announces Global Acquisition of Zokinvy® (Lonafarnib) for Treatment of Hutchinson-Gilford Progeria Syndrome from Eiger BioPharmaceuticals – Sentynl Therapeutics, Inc.

  https://sentynl.com/news/sentynl-therapeutics-announces-global-acquisition-of-zokinvy-lonafarnib-for-treatment-of-hutchinson-gilford-progeria-syndrome-from-eiger-biopharmaceuticals/

  Sentynl Therapeutics Announces Global Acquisition of Zokinvy (Lonafarnib) for Treatment of Hutchinson-Gilford Progeria Syndrome from Eiger BioPharmaceuticals. […] Zokinvy is approved in the U.S. (2020), European Union and Great Britain (2022), and Japan (2024) for the treatment of progeria, a collection of ultra-rare, fatal, genetic premature aging diseases that accelerate mortality in young patients. […] Zokinvy is the first and only treatment approved by the U.S. Food and Drug Administration (FDA) to target the cause and symptoms of progeria, also known as Hutchinson-Gilford progeria syndrome (HGPS) and processing-deficient progeroid laminopathies (PDPL), in young people 12 months of age and older. […] Without Zokinvy therapy, children with HGPS commonly die of the same heart disease that affects millions of normally aging adults (arteriosclerosis), by an average age of 14.5 years.

• #3 Progeria: From the unknown to the first FDA-approved treatment

  https://www.asbmb.org/asbmb-today/science/022521/progeria-from-the-unknown-to-the-first-fda-approve

  Lonafarnib is the first drug approved by the Food and Drug Administration to treat the rare disease progeria. […] On Nov. 20, the Food and Drug Administration approved lonafarnib, which goes by the brand name Zokinvy. In trials, lonafarnib, a farnesyltransferase inhibitor, increased progeria patients lifespans by an average of 2.5 years. […] This is encouraging, as it sets a standard of care for progeria patients while we and other progeria researchers explore even better therapeutics leading to the cure, said Mike Erdos, a staff scientist at the National Institutes of Healths National Human Genome Research Institute. […] The development of the first treatment for progeria highlights the importance of basic research, collaboration between scientists and clinicians and, above all, the close involvement of patient-advocacy groups.

• #4 Progeria – Symptoms and causes – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/progeria/symptoms-causes/syc-20356038

  There’s no cure for progeria, but new treatments and research show some promise for managing symptoms and complications. […] FDA approves first treatment for Hutchinson-Gilford progeria syndrome and some progeroid laminopathies.

• #5 How Zokinvy works | Zokinvy (lonafarnib) Patient

  https://www.zokinvy.com/introducing-zokinvy

  Zokinvy interrupts a key disease process in young people with Progeria and processing-deficient Progeroid Laminopathies (PDPL), helping to block progerin and progerin-like proteins from building up and damaging cells. […] Zokinvy is the first and only treatment for Progeria and PDPL that targets the cause and symptoms of Progeria and PDPL. It interrupts a key step in the disease process, helping to block progerin and progerin-like proteins from building up and damaging cells. […] ZOKINVY (lonafarnib) is a prescription medicine used to treat Hutchinson-Gilford Progeria Syndrome and some types of Progeroid Laminopathies known as processing-deficient Progeroid Laminopathies. These illnesses are caused when the body makes harmful proteins called progerin and progerin-like proteins.

• #6 Progeria treatment advances: Why lonafarnib could have huge impact

  https://www.pharmaceutical-technology.com/features/progeria-treatment-advances/

  Lonafarnib has emerged as a potential treatment for progeria and progeroid laminopathies, an ultra-rare condition for which no approved treatment exists. […] Having successfully demonstrated survival benefits in progeria patients, lonafarnib is improving the hope for the patients, who generally succumb to death at an early age of 14 years. […] The drug currently has orphan drug, breakthrough therapy, as well as rare paediatric disease (RPD) designations from the US Food and Drug Administration (FDA). […] Lonafarnib has demonstrated survival benefits in patients with progeria, an ultra-rare genetic disease. […] Eiger expanded the licence agreement in May 2018 for worldwide rights for regulatory submission, commercialisation, and distribution of lonafarnib for progeria treatment. […] Lonafarnib was investigated for progeria for the first time in a phase two, open-label clinical study in Boston Childrens Hospital in Massachusetts, US, in 2007.

• #7 Small-Molecule Therapeutic Perspectives for the Treatment of Progeria

  https://www.mdpi.com/1422-0067/22/13/7190

  Hutchinson–Gilford progeria syndrome (HGPS), or progeria, is an extremely rare disorder that belongs to the class of laminopathies, diseases characterized by alterations in the genes that encode for the lamin proteins or for their associated interacting proteins. […] In 2020, lonafarnib became the first (and only) FDA approved drug for treating progeria. […] However, this small molecule represents a treatment, but it does not cure the disease, and it has several limitations that make the development of new therapeutic strategies a critical need in the field. […] The identification of the molecular cause of the disease two decades ago spurred the quest for an effective pharmacological treatment for progeria. […] Within this overall and ambitious aim, different strategies have received attention.

• #8 It’s personal: How the Boston Children’s progeria research community brought new life to an old drug – Boston Children’s Answers

  https://answers.childrenshospital.org/progeria-lonafarnib-clinical-trials/

  In November, the FDA approved lonafarnib, the first and only drug targeting the cause of progeria. […] Lonafarnib is the only drug available anywhere in the world approved to treat the cause of progeria, an overabundance of an abnormal protein called progerin. […] „Fairly quickly we realized that a certain class of drugs, which included lonafarnib, might have some promise as a progeria treatment,” says Dr. Kleinman. […] The trio soon decided to begin clinical testing of lonafarnib in children with progeria at Boston Children’s. […] In September 2012, the first results were in. After two years of lonafarnib treatment, 1 in 3 children had greater than a 50 percent increase in annual weight gain, or had stopped losing weight and started gaining. […] „I’m very thankful for the drug and I think it has done a lot for me and other kids, particularly around heart health and gaining weight,” she says.

• #9 FDA Approves First Treatment for Hutchinson-Gilford Progeria Syndrome and Some Progeroid Laminopathies | FDA

  https://www.fda.gov/news-events/press-announcements/fda-approves-first-treatment-hutchinson-gilford-progeria-syndrome-and-some-progeroid-laminopathies

  Today, the U.S. Food and Drug Administration approved Zokinvy (lonafarnib) capsules to reduce the risk of death due to Hutchinson-Gilford progeria syndrome and for the treatment of certain processing-deficient progeroid laminopathies in patients one year of age and older. […] With todays approval, Zokinvy is the first FDA-approved medication for these devastating diseases. […] Before todays approval, the only treatment options included supportive care and therapies directed towards the complications arising from the disease. […] Zokinvy, a farnesyltransferase inhibitor, is an oral medication that helps prevent the buildup of defective progerin or progerin-like protein. […] The effectiveness of Zokinvy for the treatment of Hutchinson-Gilford progeria syndrome was demonstrated in 62 patients from two single-arm trials that were compared to matched, untreated patients from a separate natural history study. […] Zokinvys approval for the treatment of certain processing-deficient progeroid laminopathies that are very rare took into account similarities in the underlying genetic mechanism of disease and other available data.

• #10 FDA Approves Drug for Progeria, a Rare Disease Causing Rapid Aging in Children

  https://www.ajmc.com/view/fda-approves-drug-for-progeria-a-rare-disease-causing-rapid-aging-in-children

  The FDA approved the first treatment to reduce the risk of mortality from progeria, an extremely rare disease that causes rapid aging and death, usually by age 15. […] Lonafarnib is an oral farnesyltransferase inhibitor (FTI) that has shown survival benefit in children with Hutchinson-Gilford progeria syndrome and for the treatment of certain processing-deficient progeroid laminopathies in patients 1 year and older. […] Lonafarnib helps to prevent the buildup of the defective protein. […] Effectiveness was shown in 62 patients from 2 single-arm trials who were compared with matched, untreated patients from a separate natural history study. […] Data based on information from the PRF International Patient Registry and clinical trials co-coordinated by PRF and Boston Children’s Hospital demonstrated that lonafarnib reduced the incidence of mortality by 60% (P = .0064) and increased average survival time by 2.5 years through the maximum follow-up time of 11 years. […] With todays approval, Zokinvy is the first FDA-approved medication for these devastating diseases. […] The FDA will continue to work with stakeholders to advance the development of additional new, effective and safe therapies for these patients.

• #11 First-Ever Treatment for Ultra-Rare Rapid-Aging Disease Progeria Receives Approval in Japan

  https://www.prnewswire.com/news-releases/first-ever-treatment-for-ultra-rare-rapid-aging-disease-progeria-receives-approval-in-japan-302039965.html

  The Progeria Research Foundation Marks Significant Achievement as Zokinvy (lonafarnib) Receives Japanese Approval for the Treatment of Progeria and Processing-Deficient Progeroid Laminopathies. […] Approval from the U.S. Food and Drug Administration (FDA) for Zokinvy was granted in November, 2020 and the European Medicines Administration (EMA) in July, 2022. […] Zokinvy is a farnesyltransferase inhibitor (FTI) that has shown survival benefit in children with Progeria. […] Data based on information from the PRF International Patient Registry and clinical trials co-coordinated by PRF and Boston Children’s Hospital (BCH) demonstrated that in patients with Progeria, Zokinvy reduced the incidence of mortality by 72% and increased average survival time by an average of 30% (4.3 years). […] „On the heels of the 2020 FDA approval and the 2022 EMA approval for Zokinvy, now children and young adults with Progeria and PL in Japan will be able to access this life-extending, heart-strengthening treatment through the convenience of a prescription.”

• #12 FDA Approves Drug for Progeria, a Rare Disease Causing Rapid Aging in Children

  https://www.ajmc.com/view/fda-approves-drug-for-progeria-a-rare-disease-causing-rapid-aging-in-children

  The FDA approved the first treatment to reduce the risk of mortality from progeria, an extremely rare disease that causes rapid aging and death, usually by age 15. […] Lonafarnib is an oral farnesyltransferase inhibitor (FTI) that has shown survival benefit in children with Hutchinson-Gilford progeria syndrome and for the treatment of certain processing-deficient progeroid laminopathies in patients 1 year and older. […] Lonafarnib helps to prevent the buildup of the defective protein. […] Effectiveness was shown in 62 patients from 2 single-arm trials who were compared with matched, untreated patients from a separate natural history study. […] Data based on information from the PRF International Patient Registry and clinical trials co-coordinated by PRF and Boston Children’s Hospital demonstrated that lonafarnib reduced the incidence of mortality by 60% (P = .0064) and increased average survival time by 2.5 years through the maximum follow-up time of 11 years. […] With todays approval, Zokinvy is the first FDA-approved medication for these devastating diseases. […] The FDA will continue to work with stakeholders to advance the development of additional new, effective and safe therapies for these patients.

• #13 Clinical trials | The Progeria Research Foundation

  https://www.progeriaresearch.org/clinical-trials/

  Progeria clinical drug trials are the best hope for children with Progeria, testing potential treatments that may enable them to live longer, healthier lives. […] PRF, in collaboration with Korean-based study sponsor PRG Science Technology (PRG ST), has started enrollment for a brand-new clinical trial with a drug called Progerinin. Laboratory evidence shows that this drug, when taken in combination with lonafarnib, may be more effective than lonafarnib alone. […] PRF has taken the first patient-involved steps toward a clinical trial in RNA therapy SO exciting! […] The FTI lonafarnib is now a proven treatment for Progeria. […] In 2012, the study results were published, demonstrating that every child experienced improvement in one or more areas, including the vital cardiovascular system.

• #14 Treatment for Hutchchinson-Gilford Progeria Syndrome and Processing-Deficient Progeroid Laminopathies | Zokinvy (lonafarnib) Patient

  https://www.zokinvy.com/

  Zokinvy is the first and only treatment to target the cause and symptoms of Progeria, also known as Hutchinson-Gilford Progeria Syndrome, and processing-deficient Progeroid Laminopathies (PDPL) in young people 12 months of age and older. […] ZOKINVY (lonafarnib) is a prescription medicine used to treat Hutchinson-Gilford Progeria Syndrome and some types of Progeroid Laminopathies known as processing-deficient Progeroid Laminopathies. These illnesses are caused when the body makes harmful proteins called progerin and progerin-like proteins. […] Do not give ZOKINVY to children under the age of 12 months because it has not been studied in this age group.

• #15 First-Ever Treatment for Ultra-Rare Rapid-Aging Disease Progeria Receives Approval in Japan

  https://www.prnewswire.com/news-releases/first-ever-treatment-for-ultra-rare-rapid-aging-disease-progeria-receives-approval-in-japan-302039965.html

  The Progeria Research Foundation Marks Significant Achievement as Zokinvy (lonafarnib) Receives Japanese Approval for the Treatment of Progeria and Processing-Deficient Progeroid Laminopathies. […] Approval from the U.S. Food and Drug Administration (FDA) for Zokinvy was granted in November, 2020 and the European Medicines Administration (EMA) in July, 2022. […] Zokinvy is a farnesyltransferase inhibitor (FTI) that has shown survival benefit in children with Progeria. […] Data based on information from the PRF International Patient Registry and clinical trials co-coordinated by PRF and Boston Children’s Hospital (BCH) demonstrated that in patients with Progeria, Zokinvy reduced the incidence of mortality by 72% and increased average survival time by an average of 30% (4.3 years). […] „On the heels of the 2020 FDA approval and the 2022 EMA approval for Zokinvy, now children and young adults with Progeria and PL in Japan will be able to access this life-extending, heart-strengthening treatment through the convenience of a prescription.”

• #16 Progeria – Diagnosis and treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/progeria/diagnosis-treatment/drc-20356043

  There’s no cure for progeria. But regular monitoring for heart and blood vessel disease may help with managing your child’s condition. […] Certain therapies may ease or delay some of the symptoms of progeria. Treatments depend on your child’s condition and symptoms. These may include: […] Lonafarnib (Zokinvy). This oral medicine helps prevent the buildup of faulty progerin and progerin-like proteins in cells. Preventing this buildup in cells can slow the progression of symptoms that occur in progeria, which can help some children live longer. The medicine is approved by the U.S. Food and Drug Administration for children 1 year and older. […] Low-dose aspirin. A daily dose may help prevent heart attacks and strokes. […] Other medicines. Depending on your child’s condition, the health care provider may prescribe other medicines to treat complications. These may include dietary therapy, possibly with statins to help blood vessels and heart function. Also, blood thinners to help prevent blood clots. Medicines to treat headaches and other symptoms may be needed.

• #17 Progeria – Diagnosis and treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/progeria/diagnosis-treatment/drc-20356043

  There’s no cure for progeria. But regular monitoring for heart and blood vessel disease may help with managing your child’s condition. […] Certain therapies may ease or delay some of the symptoms of progeria. Treatments depend on your child’s condition and symptoms. These may include: […] Lonafarnib (Zokinvy). This oral medicine helps prevent the buildup of faulty progerin and progerin-like proteins in cells. Preventing this buildup in cells can slow the progression of symptoms that occur in progeria, which can help some children live longer. The medicine is approved by the U.S. Food and Drug Administration for children 1 year and older. […] Low-dose aspirin. A daily dose may help prevent heart attacks and strokes. […] Other medicines. Depending on your child’s condition, the health care provider may prescribe other medicines to treat complications. These may include dietary therapy, possibly with statins to help blood vessels and heart function. Also, blood thinners to help prevent blood clots. Medicines to treat headaches and other symptoms may be needed.

• #18 Progeria (Hutchinson-Gilford Progeria Syndrome HGPS): Symptoms and Causes

  https://www.webmd.com/children/progeria

  Though drugs cant yet cure progeria, some treatments can improve the symptoms of the disease and address its complications, such as heart disease. These include: Low-dose aspirin to help prevent heart attacks and stroke, Statins or other cholesterol-lowering medications, Blood thinners to prevent blood clots, Physical therapy to help with movement and daily activities, Dietary changes that ensure adequate nutrition, Hearing aids, Vision care. […] Treatments can help ease or delay some of the disease’s symptoms. […] Your child’s doctor may suggest drugs and changes to your childs diet to lower cholesterol or prevent blood clots. A low dose of aspirin every day can help. Growth hormone can help build height and weight. […] A specially planned exercise program can help your child keep moving if they have stiff joints or hip problems.

• #19 Progeria – Diagnosis and treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/progeria/diagnosis-treatment/drc-20356043

  There’s no cure for progeria. But regular monitoring for heart and blood vessel disease may help with managing your child’s condition. […] Certain therapies may ease or delay some of the symptoms of progeria. Treatments depend on your child’s condition and symptoms. These may include: […] Lonafarnib (Zokinvy). This oral medicine helps prevent the buildup of faulty progerin and progerin-like proteins in cells. Preventing this buildup in cells can slow the progression of symptoms that occur in progeria, which can help some children live longer. The medicine is approved by the U.S. Food and Drug Administration for children 1 year and older. […] Low-dose aspirin. A daily dose may help prevent heart attacks and strokes. […] Other medicines. Depending on your child’s condition, the health care provider may prescribe other medicines to treat complications. These may include dietary therapy, possibly with statins to help blood vessels and heart function. Also, blood thinners to help prevent blood clots. Medicines to treat headaches and other symptoms may be needed.

• #20 Progeria (Hutchinson-Gilford Progeria Syndrome HGPS): Symptoms and Causes

  https://www.webmd.com/children/progeria

  Though drugs cant yet cure progeria, some treatments can improve the symptoms of the disease and address its complications, such as heart disease. These include: Low-dose aspirin to help prevent heart attacks and stroke, Statins or other cholesterol-lowering medications, Blood thinners to prevent blood clots, Physical therapy to help with movement and daily activities, Dietary changes that ensure adequate nutrition, Hearing aids, Vision care. […] Treatments can help ease or delay some of the disease’s symptoms. […] Your child’s doctor may suggest drugs and changes to your childs diet to lower cholesterol or prevent blood clots. A low dose of aspirin every day can help. Growth hormone can help build height and weight. […] A specially planned exercise program can help your child keep moving if they have stiff joints or hip problems.

• #21 Progeria (Hutchinson-Gilford Progeria Syndrome HGPS): Symptoms and Causes

  https://www.webmd.com/children/progeria

  Though drugs cant yet cure progeria, some treatments can improve the symptoms of the disease and address its complications, such as heart disease. These include: Low-dose aspirin to help prevent heart attacks and stroke, Statins or other cholesterol-lowering medications, Blood thinners to prevent blood clots, Physical therapy to help with movement and daily activities, Dietary changes that ensure adequate nutrition, Hearing aids, Vision care. […] Treatments can help ease or delay some of the disease’s symptoms. […] Your child’s doctor may suggest drugs and changes to your childs diet to lower cholesterol or prevent blood clots. A low dose of aspirin every day can help. Growth hormone can help build height and weight. […] A specially planned exercise program can help your child keep moving if they have stiff joints or hip problems.

• #22 Progeria (Hutchinson-Gilford Progeria Syndrome HGPS): Symptoms and Causes

  https://www.webmd.com/children/progeria

  Though drugs cant yet cure progeria, some treatments can improve the symptoms of the disease and address its complications, such as heart disease. These include: Low-dose aspirin to help prevent heart attacks and stroke, Statins or other cholesterol-lowering medications, Blood thinners to prevent blood clots, Physical therapy to help with movement and daily activities, Dietary changes that ensure adequate nutrition, Hearing aids, Vision care. […] Treatments can help ease or delay some of the disease’s symptoms. […] Your child’s doctor may suggest drugs and changes to your childs diet to lower cholesterol or prevent blood clots. A low dose of aspirin every day can help. Growth hormone can help build height and weight. […] A specially planned exercise program can help your child keep moving if they have stiff joints or hip problems.

• #23 Progeria-Hutchinson-Gilford Progeria Syndrome (HGPS)

  https://www.prepladder.com/neet-ss-pediatrics/general-pediatrics/progeria-hutchinson-gilford-progeria-syndrome-hgps-pathogenesis-clinical-features-and-treatment

  Progeria-Hutchinson-Gilford Progeria Syndrome (HGPS)- Pathogenesis, Clinical Features and Treatment […] A disease-modifying drug called lonafarnib inhibits the first step, preventing progerin production. It reduces the effects of progerin on functional Lamin proteins. […] Lonafarnib is a farnesyltransferase inhibitor that prevents the adverse effects of progerin. Studies have shown the drug to improve cardiovascular ailments and overall mortality. However, it does not improve dermatological, dental, ocular, bony, and cartilaginous manifestations. Lonafarnib combined with mTOR inhibitor sirolimus is an experimental therapy. 2.Supportive therapy- Human recombinant growth hormone at 0.05 mg/Kg/day subcutaneously helps growth-related abnormalities and short stature. Low-dose, 2 mg/Kg/day of aspirin helps reduce the risk of cerebrovascular incidents.

• #24 Clinical trials | The Progeria Research Foundation

  https://www.progeriaresearch.org/clinical-trials/

  The addition of a newer drug: Everolimus. […] Everolimus is a form of the drug rapamycin; everolimus could be more easily given to children with Progeria because it requires fewer blood draws to measure drug levels. […] Our work with genetic therapies is advancing full-speed ahead! RNA therapy and DNA Gene Editing studies have shown a vast improvement in Progeria mice lifespan. PRF continues to invest substantial funds into their development, with the hope that these research efforts will lead to clinical trials, and, ultimately, the cure.

• #25 Small-Molecule Therapeutic Perspectives for the Treatment of Progeria

  https://www.mdpi.com/1422-0067/22/13/7190

  The fact that one of them, progerinin, has started phase I preclinical studies is indicative of its promising potential. […] All in sum, and although an extensive workload still lies ahead, it is indubitable that the next years to come will bring exciting new treatment strategies which, either by themselves or in combination with others, will finally provide a definitive cure for this rare disease.

• #26 From dye to base editing, early aging may soon have a cure | Drug Discovery News

  https://www.drugdiscoverynews.com/from-dye-to-base-editing-early-aging-may-soon-have-a-cure-15616

  The idea behind that trial is to see whether we can ask the cell to remove the mutant lamin A protein by activating autophagy using the drug rapamycin. […] Progeria researchers wanted to see whether this method could correct the lamin A mutation in progeria cells. […] This has brought a lot of hope for the future of progeria treatment. […] I just hope things will work, and we can help progeria patients as soon as possible.

• #27 US FDA Authorizes Launch of Clinical Trial to Support New Treatment Development for Progeria – BioSpace

  https://www.biospace.com/fda/us-fda-authorizes-launch-of-clinical-trial-to-support-new-treatment-development-for-progeria

  Zokinvy works by blocking the toxic, disease-causing protein, progerin, from developing and attacking normal cells, while Progerinin appears to reduce the level of progerin that accumulates in the body. […] Currently, Zokinvy is the only treatment for Progeria and PL approved by the FDA (in 2020), the European Medicines Agency (EMA, in 2022) and the Japanese Ministry of Health, Labor and Welfare (MHLW, in 2024). […] „This new trial represents another step forward in our mission to treat and cure these amazing children and young adults with Progeria,” said PRF Executive Director Audrey Gordon, Esq. […] „Patients with Progeria and their families deserve more options,” said Dr. Kush Dhody, President, Amarex Clinical Research. […] Progerinin, the investigational treatment for Progeria has been approved by the FDA to enter phase 2 clinical trials, and is about to enter Phase2 at Boston Children’s Hospital.

• #28 US FDA Authorizes Launch of Clinical Trial to Support New Treatment Development for Progeria – BioSpace

  https://www.biospace.com/fda/us-fda-authorizes-launch-of-clinical-trial-to-support-new-treatment-development-for-progeria

  The United States Food and Drug Administration (FDA) has authorized the start of patient enrollment for a Phase 2a clinical trial of Progerinin, an investigational drug being developed for the treatment of Progeria. […] Advancing the development of Progerinin could potentially provide an additional treatment for this fatal disease. […] In the Phase 2a randomized open label trial, Progerinin will be administered in combination with the current standard-of-care, Zokinvy, to 10 patients with Progeria and Progeroid Laminopathies (PL) who are one year of age and older. […] Zokinvy has been shown to increase lifespan in a Progeria mouse model by 25%, and data published in the journal Cells in April 2023 indicated that Progerinin showed a potential increase in Progeria mouse lifespan by 50%.

• #29 US FDA Authorizes Launch of Clinical Trial to Support New Treatment Development for Progeria – BioSpace

  https://www.biospace.com/fda/us-fda-authorizes-launch-of-clinical-trial-to-support-new-treatment-development-for-progeria

  The United States Food and Drug Administration (FDA) has authorized the start of patient enrollment for a Phase 2a clinical trial of Progerinin, an investigational drug being developed for the treatment of Progeria. […] Advancing the development of Progerinin could potentially provide an additional treatment for this fatal disease. […] In the Phase 2a randomized open label trial, Progerinin will be administered in combination with the current standard-of-care, Zokinvy, to 10 patients with Progeria and Progeroid Laminopathies (PL) who are one year of age and older. […] Zokinvy has been shown to increase lifespan in a Progeria mouse model by 25%, and data published in the journal Cells in April 2023 indicated that Progerinin showed a potential increase in Progeria mouse lifespan by 50%.

• #30 An overview of treatment strategies for Hutchinson-Gilford Progeria syndrome

  https://pmc.ncbi.nlm.nih.gov/articles/PMC5973194/

  Hutchinson-Gilford progeria syndrome (HGPS) is a sporadic, autosomal dominant disorder characterized by premature and accelerated aging symptoms leading to death at the mean age of 14.6 years usually due to cardiovascular complications. […] A wide spectrum of treatment strategies, targeting several processes with different specificities, has been proposed to correct the defects in HGPS: (i) to directly repair the disease-causing mutation; (ii) to inhibit pre-mRNA aberrant splicing leading to progerin mRNA production; (iii) to decrease the toxicity of isoprenylated and methylated progerin; (iv) to induce progerin clearance; (v) to decrease the noxious downstream effects linked to progerin accumulation. […] Specificity of a wide spectrum of treatment strategies for progeria. Several therapeutic strategies for progeria are shown. In decreasing order for their target specificity, treatment strategies can be mentioned as follows: the mutation-causing disease repair by genome editing (CRISPR as a future possibility to be evaluated), the inhibition of pre-mRNA aberrant splicing (Antisense OligoNucleotides: AONs, Metformin, MG132), the reduction of isoprenylated and methylated progerin levels (Farnesyl transferase inhibitors: FTIs, ZOledronate-PRAvastatin: ZOPRA, Monoaminopyrimidines: Mono-APs, Isoprenylcysteine CarboxyMethyl Transferase (ICMT) inhibitor, GeranylGeranylTransferase Inhibitor: GGTI-2147), the induction of progerin clearance (Rapamycin, Sulforaphane, Retinoids, MG132) and the reduction of the noxious downstream effects due to progerin accumulation (Rock inhibitor: Y-27632, Nuclear factor erythroid-2-Related Factor 2: NRF2 reactivation, Methylene blue, sodium salicylate, DOT1L inhibitor: epz-4777, Remodelin, Vitamin D, Resveratrol, N-Acetyl-Cysteine: NAC, inorganic pyrophosphate: PPi and JH4).

• #31 Clinical trials | The Progeria Research Foundation

  https://www.progeriaresearch.org/clinical-trials/

  The addition of a newer drug: Everolimus. […] Everolimus is a form of the drug rapamycin; everolimus could be more easily given to children with Progeria because it requires fewer blood draws to measure drug levels. […] Our work with genetic therapies is advancing full-speed ahead! RNA therapy and DNA Gene Editing studies have shown a vast improvement in Progeria mice lifespan. PRF continues to invest substantial funds into their development, with the hope that these research efforts will lead to clinical trials, and, ultimately, the cure.

• #32 DNA-editing method shows promise to treat mouse model of progeria

  https://www.genome.gov/news/news-release/DNA-editing-method-shows-promise-to-treat-mouse-model-of-progeria

  Researchers have successfully used a DNA-editing technique to extend the lifespan of mice with the genetic variation associated with progeria, a rare genetic disease that causes extreme premature aging in children and can significantly shorten their life expectancy. […] For this study, researchers used a breakthrough DNA-editing technique called base editing, which substitutes a single DNA letter for another without damaging the DNA, to study how changing this mutation might affect progeria-like symptoms in mice. […] The study follows another recent milestone for progeria research, as the U.S. Food and Drug Administration approved the first treatment for progeria in November 2020, a drug called lonafarnib. The drug therapy provides some life extension, but it is not a cure. The DNA-editing method may provide an additional and even more dramatic treatment option in the future.

• #33 Interview: CRISPR Base Editors Offer First One-Time Treatment for the Lethal Premature-ageing Disease Progeria – CRISPR Medicine

  https://crisprmedicinenews.com/news/crispr-base-editors-offer-first-one-time-treatment-for-the-lethal-premature-ageing-disease-progeria/

  The CRISPR gene-editing therapy more than doubled the lifespan in a mouse model by tackling the root cause of Hutchinson-Gilford progeria. […] The study represents a major step forward on that front and Liu was eager to give proper credit to the team effort. […] In the actual study, the team treated that animal model as well as cultured fibroblasts from children with Progeria with an Adenine Base Editor (ABE). […] The treated mice also looked generally healthier than saline-injected Progeria mice, who aged rapidly and died early not unlike human patients with the disease. […] The experimental gene therapy extended the mice’s lifespan roughly 2.37 times over. […] To have a 2.4-times longer lifespan is beyond what we could have hoped for. […] This approach is much more direct than lonafarnib that’s the treatment recently approved by the FDA which doesn’t reverse the mutation but instead tries to undo the damage to the progerin protein.

• #34 DNA-editing method shows promise to treat mouse model of progeria

  https://www.genome.gov/news/news-release/DNA-editing-method-shows-promise-to-treat-mouse-model-of-progeria

  Following this success, the researchers tested the gene-editing technique by delivering a single intravenous injection of the DNA-editing mix into nearly a dozen mice with the progeria-causing mutation soon after birth. The gene editor successfully restored the normal DNA sequence of the LMNA gene in a significant percentage of cells in various organs, including the heart and aorta. […] Most dramatically, the treated mice’s lifespan increased from seven months to almost 1.5 years. The average normal lifespan of the mice used in the study is two years. […] Ultimately our goal will be to try to develop this for humans, but there are additional key questions that we need to first address in these model systems.

• #35 An overview of treatment strategies for Hutchinson-Gilford Progeria syndrome

  https://pmc.ncbi.nlm.nih.gov/articles/PMC5973194/

  The efficiency of an antisense therapeutic approach using morpholino antisense oligonucleotides (AON) in sterically blocking the aberrant LMNA splicing site leading to progerin production has been previously proven in vitro on human HGPS patients cells and in vivo on a knock-in LmnaG609G/G609G mouse model. […] Metformin decreases SRSF-1 and progerin expression in mesenchymal stem cells derived from HGPS induced pluripotent stem cells (HGPS MSCs) and in several other in vitro models of HGPS, i.e., human primary HGPS fibroblasts and LmnaG609G/G609G mouse fibroblasts, resulting in improved nuclear shape abnormalities and premature osteoblastic differentiation of HGPS MSCs. […] MG132 treatment improves cellular HGPS phenotypes, reduces cellular senescence and enhances viability and proliferation in HGPS fibroblasts.

• #36 Splice-inhibition therapy targets progeria | Nature Medicine

  https://www.nature.com/articles/s41591-021-01267-z

  Antisense oligonucleotides that target lamin A/C pre-mRNA decrease both progerin transcript levels and progerin protein levels and extend lifespan in an animal model of Hutchinson-Gilford progeria syndrome. […] Inhibiting the splicing of progerin is a promising therapy for HGPS.

• #37 An overview of treatment strategies for Hutchinson-Gilford Progeria syndrome

  https://pmc.ncbi.nlm.nih.gov/articles/PMC5973194/

  Recent studies have shown the involvement of microRNAs and in particular, mir-9 which prevents progerin accumulation in HGPS neurons. Therefore, another possibility for developing new therapeutic approaches for progeria would be the modulation of microRNAs, taking into consideration off-targets that, again, must be evaluated in vitro and in vivo before transposition to humans.

• #38 Small-Molecule Therapeutic for Progeria Treatment | Encyclopedia MDPI

  https://encyclopedia.pub/entry/11972

  Considering the importance of the farnesyl and methyl groups in increasing the lipophilicity of progerin and the efficacy of the FTI lonafarnib to improve the phenotype of the disease, the possibility that reducing methylation could reduce the abnormal accumulation of progerin in the nuclear membrane and hence improve the phenotype of the disease has received increasing attention. […] In this context, the finding that genetic disruption of ICMT improved the phenotype in the ZMPSTE24 mouse model of progeria supported that a small molecule ICMT inhibitor could represent a new therapeutic strategy to address HGPS. […] Together, these results support the potential of the ICMT inhibitors, by themselves or in combination therapies, for treating progeria. […] Hence, it is conceivable that an inhibitor of this protein–protein interaction could improve the progeroid phenotype.

• #39 New potential drug target for treating progeria

  https://frontlinegenomics.com/new-potential-drug-target-for-treating-progeria/

  Scientists have identified a potential new treatment approach for a rare genetic disorder known as Hutchinson-Gilford progeria syndrome (HGPS). […] Researchers have found that inhibiting the methylation of progerin by inactivating the isoprenylcysteine carboxylmethyltransferase (ICMT) gene overcomes senescence and increases proliferation of HGPS cells. […] These results provide hope that ICMT inhibitors may be useful drug candidates for treating children with HGPS. […] “We hope these findings will further incentivise the development of efficacious compounds targeting ICMT. This approach would also likely lack the detrimental properties of current protocols treating already frail children with drugs originally developed to treat cancer.”

• #40 An overview of treatment strategies for Hutchinson-Gilford Progeria syndrome

  https://pmc.ncbi.nlm.nih.gov/articles/PMC5973194/

  The efficiency of an antisense therapeutic approach using morpholino antisense oligonucleotides (AON) in sterically blocking the aberrant LMNA splicing site leading to progerin production has been previously proven in vitro on human HGPS patients cells and in vivo on a knock-in LmnaG609G/G609G mouse model. […] Metformin decreases SRSF-1 and progerin expression in mesenchymal stem cells derived from HGPS induced pluripotent stem cells (HGPS MSCs) and in several other in vitro models of HGPS, i.e., human primary HGPS fibroblasts and LmnaG609G/G609G mouse fibroblasts, resulting in improved nuclear shape abnormalities and premature osteoblastic differentiation of HGPS MSCs. […] MG132 treatment improves cellular HGPS phenotypes, reduces cellular senescence and enhances viability and proliferation in HGPS fibroblasts.

• #41 Progeria – Diagnosis and treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/progeria/diagnosis-treatment/drc-20356043

  Physical and occupational therapy. Physical therapy can help with joint stiffness and hip problems to help your child remain active. Occupational therapy can help your child learn ways to manage daily activities, such as dressing, brushing teeth and eating. […] Nutrition. A balanced diet that includes healthy, high-calorie foods can help maintain adequate nutrition. Sometimes nutrition supplements are needed to provide extra calories. […] Current research seeks to understand progeria and identify new treatment options. Some areas of research include: […] Testing more medicines for treatment of progeria.

• #42 Progeria – Diagnosis and treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/progeria/diagnosis-treatment/drc-20356043

  Physical and occupational therapy. Physical therapy can help with joint stiffness and hip problems to help your child remain active. Occupational therapy can help your child learn ways to manage daily activities, such as dressing, brushing teeth and eating. […] Nutrition. A balanced diet that includes healthy, high-calorie foods can help maintain adequate nutrition. Sometimes nutrition supplements are needed to provide extra calories. […] Current research seeks to understand progeria and identify new treatment options. Some areas of research include: […] Testing more medicines for treatment of progeria.

• #43 Progeria | UM Health-Sparrow

  https://www.uofmhealthsparrow.org/departments-conditions/conditions/progeria

  Physical and occupational therapy. Physical therapy can help with joint stiffness and hip problems to help your child remain active. Occupational therapy can help your child learn ways to manage daily activities, such as dressing, brushing teeth and eating. […] Nutrition. A balanced diet that includes healthy, high-calorie foods can help maintain adequate nutrition. Sometimes nutrition supplements are needed to provide extra calories. […] Current research seeks to understand progeria and identify new treatment options. Some areas of research include: […] Studying ways to prevent heart and blood vessel disease. […] Testing more medicines for treatment of progeria.

• #44 Progeria – Diagnosis and treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/progeria/diagnosis-treatment/drc-20356043

  Physical and occupational therapy. Physical therapy can help with joint stiffness and hip problems to help your child remain active. Occupational therapy can help your child learn ways to manage daily activities, such as dressing, brushing teeth and eating. […] Nutrition. A balanced diet that includes healthy, high-calorie foods can help maintain adequate nutrition. Sometimes nutrition supplements are needed to provide extra calories. […] Current research seeks to understand progeria and identify new treatment options. Some areas of research include: […] Testing more medicines for treatment of progeria.

• #45 Progeria | UM Health-Sparrow

  https://www.uofmhealthsparrow.org/departments-conditions/conditions/progeria

  Physical and occupational therapy. Physical therapy can help with joint stiffness and hip problems to help your child remain active. Occupational therapy can help your child learn ways to manage daily activities, such as dressing, brushing teeth and eating. […] Nutrition. A balanced diet that includes healthy, high-calorie foods can help maintain adequate nutrition. Sometimes nutrition supplements are needed to provide extra calories. […] Current research seeks to understand progeria and identify new treatment options. Some areas of research include: […] Studying ways to prevent heart and blood vessel disease. […] Testing more medicines for treatment of progeria.

• #46 Progeria (Hutchinson-Gilford Progeria Syndrome) | Boston Children’s Hospital

  https://www.childrenshospital.org/conditions/progeria

  Additional drug treatments are currently in clinical trials (see the research and innovation section). Other treatments are still in the early research stages and are not yet ready for clinical trials. […] Because of their increased cardiovascular risk, children with progeria should be monitored by a cardiologist and receive annual blood pressure, cholesterol, and heart function tests, including an electrocardiogram and echocardiogram (cardiac ultrasound). […] Your childs doctor may prescribe low-dose aspirin therapy to thin the blood and prevent heart attack or stroke. […] For more information on caring for children with progeria, the Progeria Research Foundation provides a detailed handbook with information for both clinicians and families. […] In partnership with the Progeria Research Foundation, Boston Childrens has hosted multiple clinical trials of lonafarnib, drawing children from all over the world. These trials showed an increased life expectancy and have led the Food and Drug Administration to approve lonafarnib.

• #47 Progeria (Hutchinson-Gilford Progeria Syndrome HGPS): Symptoms and Causes

  https://www.webmd.com/children/progeria

  Some children may have an angioplasty, a minimally invasive procedure in which doctors insert a stent to widen an artery to allow blood to flow more easily. Heart valve replacement surgery also may be necessary for people with the disease who develop a narrowed aortic valve, a condition called aortic stenosis. This can happen in those who live longer with the disease as a result of taking lonafarnib.

• #48 Progeria (Hutchinson-Gilford Progeria Syndrome HGPS): Symptoms and Causes

  https://www.webmd.com/children/progeria

  Some children may have an angioplasty, a minimally invasive procedure in which doctors insert a stent to widen an artery to allow blood to flow more easily. Heart valve replacement surgery also may be necessary for people with the disease who develop a narrowed aortic valve, a condition called aortic stenosis. This can happen in those who live longer with the disease as a result of taking lonafarnib.

• #49 Progeria (Hutchinson-Gilford Progeria Syndrome HGPS): Symptoms and Causes

  https://www.webmd.com/children/progeria

  Some children may have an angioplasty, a minimally invasive procedure in which doctors insert a stent to widen an artery to allow blood to flow more easily. Heart valve replacement surgery also may be necessary for people with the disease who develop a narrowed aortic valve, a condition called aortic stenosis. This can happen in those who live longer with the disease as a result of taking lonafarnib.

• #50 Small-Molecule Therapeutic Perspectives for the Treatment of Progeria

  https://www.mdpi.com/1422-0067/22/13/7190

  Among the different FTIs showing high efficacy in the inhibition of FTase in vivo, lonafarnib soon stood out as the most promising candidate to start a single-arm phase II clinical trial in 2007. […] Importantly, it also decreased mortality rate (3.7% vs. 33.3% after a median of 2.2 years of follow-up in individuals receiving lonafarnib monotherapy compared with no treatment). […] Although lonafarnib does not correct all the alterations of the disease, such as lipodystrophy, skin features, alopecia, and joint contractures, the fact that it is the only available drug for treating this lethal pathology and that it has a positive impact in ameliorating specific features of HGPS, has led to its recent approval by the FDA under the name of Zokinvy. […] In spite of the enormous advances in therapies based on gene editing strategies or ASOs, small molecules still endure as the most straightforward candidates for generating a specific therapy for progeria in the short term, as actually demonstrated by lonafarnib, the recently (and only) approved drug for this disease.

• #51 Small-Molecule Therapeutic Perspectives for the Treatment of Progeria

  https://www.mdpi.com/1422-0067/22/13/7190

  Among the different FTIs showing high efficacy in the inhibition of FTase in vivo, lonafarnib soon stood out as the most promising candidate to start a single-arm phase II clinical trial in 2007. […] Importantly, it also decreased mortality rate (3.7% vs. 33.3% after a median of 2.2 years of follow-up in individuals receiving lonafarnib monotherapy compared with no treatment). […] Although lonafarnib does not correct all the alterations of the disease, such as lipodystrophy, skin features, alopecia, and joint contractures, the fact that it is the only available drug for treating this lethal pathology and that it has a positive impact in ameliorating specific features of HGPS, has led to its recent approval by the FDA under the name of Zokinvy. […] In spite of the enormous advances in therapies based on gene editing strategies or ASOs, small molecules still endure as the most straightforward candidates for generating a specific therapy for progeria in the short term, as actually demonstrated by lonafarnib, the recently (and only) approved drug for this disease.

• #52 Progeria: From the unknown to the first FDA-approved treatment

  https://www.asbmb.org/asbmb-today/science/022521/progeria-from-the-unknown-to-the-first-fda-approve

  While lonafarnib relieves progerias cardiovascular symptoms and extends lifespan by almost 20%, it doesnt alleviate other symptoms, such as lack of subcutaneous fat, hair loss, stunted growth and joint contractures. […] Researchers pursuing second-generation drugs have a few avenues: small-molecule inhibitors that target progerin, RNA therapeutics that prevent the production of progerin, and genome editing to fix the mutation at the DNA level.

• #53 Progeria (Hutchinson-Gilford Progeria Syndrome) | Boston Children’s Hospital

  https://www.childrenshospital.org/conditions/progeria

  Treatment has extended their average life expectancy from 14.5 years to almost 20 years. […] With long-term medical treatment, however, average life expectancy has increased to almost 20 years. A few children have lived into their mid-20s. […] Based on several clinical trials conducted at Boston Childrens Hospital, the U.S. Food and Drug Administration (FDA) has approved the first treatment, lonafarnib (Zokinvy), for most children with progeria 12 months of age and older. It is currently the only approved drug that treats progerias root cause. […] Lonafarnib blocks the production of progerin by inhibiting an enzyme called farnesyltransferase. Clinical trials have found improvements in life expectancy, weight gain, blood vessel stiffness, bone structure, and cardiovascular health in children who have received it.

• #54 It’s personal: How the Boston Children’s progeria research community brought new life to an old drug – Boston Children’s Answers

  https://answers.childrenshospital.org/progeria-lonafarnib-clinical-trials/

  „That’s an 88 percent decrease in the risk of death during the time the kids were being treated,” says Dr. Kleinman. […] When the survival data was updated to include a second Boston Children’s lonafarnib trial, the children lived an average of 2.5 years longer — almost a 20 percent increase over the average life span of 14.5 years. […] Boston Children’s is now running the largest progeria drug trial in history. Currently, 62 children are involved in a study with lonafarnib and everolimus, an existing drug widely used by transplant recipients and in cancer. […] „This drug shows us that we can push this disease towards health,” says Dr. Gordon.

• #55 Progeria: New treatment could extend lives of children with 'premature aging’ syndrome – Big Think

  https://bigthink.com/health/progeria-treatment-extend-lives-children/

  Progeria is an accelerated aging disease that causes children to die of old age at around 13 to 15 years. […] There are only two existing treatments, and both have unpleasant side effects. […] A promising new therapy based on biotechnology increases the lifespan of mice by over 60% and is ready for human clinical trials. […] Existing treatments are not ideal, as they both have serious side effects. […] A more targeted approach, therefore, is needed. […] The authors believe this evidence justifies proceeding to human clinical trials. […] But this potential new treatment provides hope for those suffering from one of the worlds most devastating genetic diseases.

• #56 Experimental mouse model reveals potential progeria treatment strategy

  https://www.drugtargetreview.com/news/98981/experimental-mouse-model-reveals-potential-progeria-treatment-strategy/

  Scientists have found that Hutchinson-Gilford Progeria Syndrome (HGPS) can be prevented with treatments targeting the cardiovascular system using a novel mouse model. […] Using the new model, the researchers have demonstrated that it is possible to treat HGPS. […] A true cure would require the elimination of the culprit mutation, commented Lpez. […] The team used CRISPR-Cas9 technology to generate HGPSrev mice and found that elimination of progerin and restoration of lamin A expression increased life expectancy by 84.5 percent in HGPSrev mice with very mild symptoms. […] Moreover, this approach extended lifespan by 6.7 percent, including in mice with very advanced symptoms. […] The study, published in Circulation, also establishes that the cardiovascular alterations and early death associated with HGPS can be prevented with treatments specifically targeting cells of the cardiovascular system. […] The researchers concluded that these results could contribute to the design of future clinical treatments, given that they suggest that strategies exclusively targeting the cardiovascular system could have a very significantly beneficial effect on patients life quality and expectancy.

• #57 A Disease That Makes Children Age Rapidly Gets Closer to a Cure – The New York Times

  https://www.nytimes.com/2024/07/24/health/progeria-dna-base-editing.html

  A cure for an ultrarare disease, progeria, could be on the horizon. The disease speeds up aging in children and dramatically shortens their lives. But, until recently, there was no path toward a highly effective treatment. […] Now, a small group of academics and government scientists, including Dr. Francis Collins, the former director of the National Institutes of Health, is working with no expectation of financial gain to halt progeria in its tracks with an innovative gene editing technique. […] After a quarter-century of research, the group is approaching manufacturers and planning to seek approval from regulators for a clinical trial on progeria gene editing. […] The new types of gene editing are “potentially the answer to a dream we all want to come true,” Dr. Collins said. […] But it took years, and the emergence of a new era of molecular medicine with advances in gene editing, for the prospect of a cure for progeria to seem possible.

• #58 The Progeria Research Foundation – For the Children ♥ For the Cure

  https://www.progeriaresearch.org/

  Our vision is a world in which every child with Progeria is cured. […] To discover treatments and the cure for Progeria and its aging-related disorders, including heart disease. […] Progeria is an ultra-rare, fatal, rapid-aging disease that afflicts children who, without the FDA-approved treatment lonafarnib, die of heart disease at an average age of 14.5 years. […] PRF is the only non-profit organization solely dedicated to finding treatments and the cure for Progeria, and is making phenomenal progress toward that goal. […] PRF is thrilled to announce that the first Progerinin clinical trial patient visits are complete! […] PRF is thrilled to announce the start of a new Progeria clinical trial with a new drug called Progerinin. […] Our lonafarnib clinical trials enrolled 107 children from 42 different countries to test this now-FDA-approved treatment. Because of your support, these children and young adults are living longer, healthier lives. […] Your donation helps The Progeria Research Foundation treat children with Progeria today, and cure them in the future.

• #59 First-Ever Treatment for Ultra-Rare Rapid-Aging Disease Progeria Receives Approval in Japan

  https://www.prnewswire.com/news-releases/first-ever-treatment-for-ultra-rare-rapid-aging-disease-progeria-receives-approval-in-japan-302039965.html

  „After more than a decade of Zokinvy research funded by PRF and co-conducted by PRF and BCH, Zokinvy’s authorization in Japan is a win for the worldwide Progeria community.” […] PRF has funded and co-coordinated all Zokinvy-associated clinical trials for Progeria and Progeroid Laminopathies, conducted at Boston Children’s Hospital.

• #60 It’s personal: How the Boston Children’s progeria research community brought new life to an old drug – Boston Children’s Answers

  https://answers.childrenshospital.org/progeria-lonafarnib-clinical-trials/

  „That’s an 88 percent decrease in the risk of death during the time the kids were being treated,” says Dr. Kleinman. […] When the survival data was updated to include a second Boston Children’s lonafarnib trial, the children lived an average of 2.5 years longer — almost a 20 percent increase over the average life span of 14.5 years. […] Boston Children’s is now running the largest progeria drug trial in history. Currently, 62 children are involved in a study with lonafarnib and everolimus, an existing drug widely used by transplant recipients and in cancer. […] „This drug shows us that we can push this disease towards health,” says Dr. Gordon.

• #61 Small-Molecule Therapeutic for Progeria Treatment | Encyclopedia MDPI

  https://encyclopedia.pub/entry/11972

  Beyond targeted therapies, alternative approaches have addressed the search of small molecules able to ameliorate or reverse the phenotypic harmful effects elicited by the abnormal progerin accumulation in the nuclear lamina. […] These results have been the basis of the phase I/II clinical trial (NCT02579044) of everolimus, a structurally-related analogue of rapamycin, in combination with lonafarnib, which is still ongoing.

• #62 Progeria: From the unknown to the first FDA-approved treatment

  https://www.asbmb.org/asbmb-today/science/022521/progeria-from-the-unknown-to-the-first-fda-approve

  While lonafarnib relieves progerias cardiovascular symptoms and extends lifespan by almost 20%, it doesnt alleviate other symptoms, such as lack of subcutaneous fat, hair loss, stunted growth and joint contractures. […] Researchers pursuing second-generation drugs have a few avenues: small-molecule inhibitors that target progerin, RNA therapeutics that prevent the production of progerin, and genome editing to fix the mutation at the DNA level.

• #63 Progeria: From the unknown to the first FDA-approved treatment

  https://www.asbmb.org/asbmb-today/science/022521/progeria-from-the-unknown-to-the-first-fda-approve

  While lonafarnib relieves progerias cardiovascular symptoms and extends lifespan by almost 20%, it doesnt alleviate other symptoms, such as lack of subcutaneous fat, hair loss, stunted growth and joint contractures. […] Researchers pursuing second-generation drugs have a few avenues: small-molecule inhibitors that target progerin, RNA therapeutics that prevent the production of progerin, and genome editing to fix the mutation at the DNA level.

• #64 Clinical trials | The Progeria Research Foundation

  https://www.progeriaresearch.org/clinical-trials/

  Progeria clinical drug trials are the best hope for children with Progeria, testing potential treatments that may enable them to live longer, healthier lives. […] PRF, in collaboration with Korean-based study sponsor PRG Science Technology (PRG ST), has started enrollment for a brand-new clinical trial with a drug called Progerinin. Laboratory evidence shows that this drug, when taken in combination with lonafarnib, may be more effective than lonafarnib alone. […] PRF has taken the first patient-involved steps toward a clinical trial in RNA therapy SO exciting! […] The FTI lonafarnib is now a proven treatment for Progeria. […] In 2012, the study results were published, demonstrating that every child experienced improvement in one or more areas, including the vital cardiovascular system.

• #65 Progeria: From the unknown to the first FDA-approved treatment

  https://www.asbmb.org/asbmb-today/science/022521/progeria-from-the-unknown-to-the-first-fda-approve

  While lonafarnib relieves progerias cardiovascular symptoms and extends lifespan by almost 20%, it doesnt alleviate other symptoms, such as lack of subcutaneous fat, hair loss, stunted growth and joint contractures. […] Researchers pursuing second-generation drugs have a few avenues: small-molecule inhibitors that target progerin, RNA therapeutics that prevent the production of progerin, and genome editing to fix the mutation at the DNA level.

• #66 Interview: CRISPR Base Editors Offer First One-Time Treatment for the Lethal Premature-ageing Disease Progeria – CRISPR Medicine

  https://crisprmedicinenews.com/news/crispr-base-editors-offer-first-one-time-treatment-for-the-lethal-premature-ageing-disease-progeria/

  Using both lonafarnib and the new ABE therapy in concert, Liu speculates and hopes, may give patients an even better prognosis than either treatment does alone, but it’s far too soon to say so with any confidence. […] The hope is that an affected Progeria child would get a one-time injection into their bloodstream a base editor packaged into some kind of delivery vehicle that could then directly and permanently correct the root cause of the disease, Liu adds. […] But Liu was quick to reiterate that any assumptions about how well the treatment might work in human children was speculative he very much wanted to avoid giving the Progeria community any false hope before he had the data to back it up. […] All of these studies represent the very early stages of gene therapies for Progeria.

• #67 The Progeria Research Foundation – For the Children ♥ For the Cure

  https://www.progeriaresearch.org/

  Our vision is a world in which every child with Progeria is cured. […] To discover treatments and the cure for Progeria and its aging-related disorders, including heart disease. […] Progeria is an ultra-rare, fatal, rapid-aging disease that afflicts children who, without the FDA-approved treatment lonafarnib, die of heart disease at an average age of 14.5 years. […] PRF is the only non-profit organization solely dedicated to finding treatments and the cure for Progeria, and is making phenomenal progress toward that goal. […] PRF is thrilled to announce that the first Progerinin clinical trial patient visits are complete! […] PRF is thrilled to announce the start of a new Progeria clinical trial with a new drug called Progerinin. […] Our lonafarnib clinical trials enrolled 107 children from 42 different countries to test this now-FDA-approved treatment. Because of your support, these children and young adults are living longer, healthier lives. […] Your donation helps The Progeria Research Foundation treat children with Progeria today, and cure them in the future.

• #68 Protein discovery could pave the way for improved treatment of premature aging disease

  https://medicalxpress.com/news/2024-10-protein-discovery-pave-treatment-premature.html

  As a next step, Cao plans to conduct a follow-up study in collaboration with a group at the NIH to explore different methods of administering Ang2 to animal models with progeria. […] „We are getting really close to a cure for progeria,” she said. „Research-wise, we are pushing hard, and I can see the light at the end of the tunnel.”

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