Materiały źródłowe

• #1 Progeria primary prevention – wikidoc

  https://www.wikidoc.org/index.php/Progeria_primary_prevention

  There are no established measures for the primary prevention of Hutchinson-Gilford progeria syndrome (HGPS). […] There are no established measures for the primary prevention of Hutchinson-Gilford progeria syndrome (HGPS).

• #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

  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. […] Zokinvy, a farnesyltransferase inhibitor, is an oral medication that helps prevent the buildup of defective progerin or progerin-like protein. […] The FDA granted the approval of Zokinvy to Eiger BioPharmaceuticals, Inc.

• #1 Progeria and processing-deficient Progeroid Laminopathies | Sentynl Therapeutics, Inc.

  https://www.zokinvy.com/hcp/understanding-progeria-and-pdpl

  Zokinvy prevents farnesylation and the subsequent accumulation of progerin or progerin-like proteins within cells.6 This accumulation impairs tissue repair functions and further damages cells as they age, which can lead to early mortality.1,3,7 […] Once a diagnosis is confirmed, initiate Zokinvy immediately if the patient is 12 months of age or older and with a body surface area (BSA) of 0.39 m2 and above. […] ZOKINVY is indicated in adult and pediatric patients 12 months of age and older with a body surface area (BSA) of 0.39 m2 and above: To reduce the risk of mortality in Hutchinson-Gilford Progeria Syndrome (HGPS) […] For the treatment of processing-deficient Progeroid Laminopathies with either: Heterozygous LMNA mutation with progerin-like protein accumulation or Homozygous or compound heterozygous ZMPSTE24 mutations.

• #1 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 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. […] „Patients with Progeria and their families deserve more options,” said Dr. Kush Dhody, President, Amarex Clinical Research. „This is an especially devastating diagnosis given the shortened lifespan it causes. We’re honored to support a study that brings more hope to these patients.”

• #1 Progeria – Diagnosis and treatment – Mayo Clinic

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

  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. […] 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. […] Provide frequent, small meals. Because nutrition and growth can be an issue for children with progeria, giving your child smaller meals more often may help provide more calories. […] Make sure your child is up to date on childhood vaccinations. A child with progeria isn’t at increased risk of infection. But like all children, your child is at risk if exposed to infectious diseases.

• #1 Progeria – Wikipedia

  https://en.wikipedia.org/wiki/Progeria

  In November 2020, the U.S. Food and Drug Administration approved lonafarnib, which helps prevent buildup of defective progerin and similar proteins. […] A clinical trial in 2018 points to significantly lower mortality rates treatment with lonafarnib alone compared with no treatment (3.7% vs. 33.3%) at a median post-trial follow-up time span of 2.2 years. […] Other treatment options have focused on reducing complications (such as cardiovascular disease) with coronary artery bypass surgery and low-dose acetylsalicylic acid. […] The use of Morpholinos has also been attempted in mice and cell cultures in order to reduce progerin production. […] Farnesyltransferase inhibitors (FTIs) are drugs that inhibit the activity of an enzyme needed to make a link between progerin proteins and farnesyl groups.

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

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

  There’s no cure for progeria, but researchers are working on finding one. As of now, one drug has been approved by the FDA to slow the diseases course: lonafarnib (Zokinvy). This medication falls into a class of experimental cancer drugs called farnesyltransferase inhibitors (FTIs). Taking this drug helps people with progeria to gain weight. It also improves their bone and heart health. Studies have shown that the lonafarnib extends the lifespan of people with progeria by an average of 4.5 years. […] 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.

• #1 Progeria: Sign, Symptoms & Risk Factors | HGPS Causes & Treatment

  https://www.delveinsight.com/blog/progeria-causes-symptoms-risk-factors-and-treatment-options

  Progeria is not curable; however, the treatment option eases or delays some of the signs and symptoms. Specific therapies for individuals with HGPS are symptomatic and supportive. Medication, changes in diet lifestyle, and physical occupational therapy are the most common treatment options. The treatments also depend on the child’s condition and symptoms. […] At present, some of the key companies such as PRG Science Technology, Merck, Eiger Biopharmaceuticals, and others are involved in developing therapies to treat and prevent progeria. As per DelveInsights analysis, several potential treatments for progeria are under investigation. These include newer agents, i.e., pravastatin + lonafarnib + zoledronic acid combinational product, and Progerinin (SLC-D011), for the treatment of the rare aging diseases (Hutchinson-Gilford Progeria Syndrome (HGPS) and Werner Syndrome (WS)).

• #1 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

  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. […] 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.

• #1 All-trans retinoic acid and rapamycin normalize Hutchinson Gilford progeria fibroblast phenotype | Oncotarget

  https://www.oncotarget.com/article/4939/text/

  Hutchinson Gilford progeria syndrome is a fatal disorder characterized by accelerated aging, bone resorption and atherosclerosis, caused by a LMNA mutation which produces progerin, a mutant lamin A precursor. […] We demonstrate that all-trans retinoic acid acts synergistically with low-dosage rapamycin reducing progerin and prelamin A, via transcriptional downregulation associated with protein degradation, and increasing the lamin A to progerin ratio. […] The combined all-trans retinoic acid-rapamycin treatment is dramatically efficient, highly reproducible, represents a promising new approach in Hutchinson-Gilford Progeria therapy and deserves investigation in ageing-associated disorders. […] In the recent years, experimental studies were performed, in vitro and in animal models, with the goal of identifying novel targets for treating laminopathies and associated diseases.

• #1 Gene editing may be the potential cure for progeria, a condition causing rapid aging in children

  https://longevity.technology/lifestyle/gene-editing-may-be-the-potential-cure-for-progeria-a-condition-causing-rapid-aging-in-children/

  Researchers are investigating gene editing as a potential treatment for progeria, a rare genetic disorder that causes rapid aging in children. […] Recent studies have demonstrated significant progress using adenine base editors (ABEs) to correct the mutation responsible for progeria. […] The latest developments, highlighted in a New York Times article, report that researchers are now focusing on optimizing the delivery systems for ABEs to treat patients directly. […] The advancements are still experimental but suggest a viable pathway toward clinical application. […] These findings highlight the promise of in vivo base editing to treat progeria and other genetic disorders by directly addressing their genetic causes.

• #1 Unique progerin C-terminal peptide ameliorates Hutchinson–Gilford progeria syndrome phenotype by rescuing BUBR1 | Nature Aging

  https://www.nature.com/articles/s43587-023-00361-w

  An accumulating body of evidence indicates an association between mitotic defects and the aging process in Hutchinson-Gilford progeria syndrome (HGPS), which is a premature aging disease caused by progerin accumulation. […] Based on this, we established a unique progerin C-terminal peptide (UPCP) that effectively blocked the binding of progerin and BUBR1 and enhanced the expression of BUBR1 by interfering with the interaction between PTBP1 and progerin. […] Therapeutics designed around UPCP may be a beneficial strategy for HGPS treatment. […] Therefore, the development of peptide drugs will provide effective new strategies for HGPS treatment. […] This indicates that UPCP may be a new drug candidate for the treatment of HGPS. […] Our findings reveal an essential role for the progerin-PTBP1-BUBR1 axis in HGPS.

• #1 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.”

• #1 Maryland Today | UMD-led Study Could Lead to Lengthened Lives for…

  https://today.umd.edu/umd-led-study-could-lead-to-lengthened-lives-for-patients-with-premature-aging-disease

  UMD researchers found that adding a protein to cells that line blood vessels might be able to improve the cardiovascular health of people with the disease progeria, which causes accelerated aging and death from heart attack or stroke when those afflicted with the disease are teenagers. […] A new University of Maryland-led discovery could spur the development of new and improved treatments for Hutchinson-Gilford progeria syndrome (HGPS), often simply called progeria a rare genetic disorder with no known cure that causes accelerated aging in children. […] This could pave the way for new treatments targeting cardiovascular complications in HGPS, which are currently a major cause of mortality in the affected children, Vakili said. […] But the researchers discovered they could use Ang2 to rescue endothelial cells, improving their health despite dysfunction stemming from HGPS.

• #1 New target identified for treatment of premature aging disease – Salk Institute for Biological Studies

  https://www.salk.edu/news-release/new-target-identified-for-treatment-of-premature-aging-disease/

  Scientists discovered that a particular RNA accumulates in people with progeria, and that inhibiting the RNA reverses signs of aging and extends life span in mice […] The team then developed molecules that could specifically bind to LINE-1 RNA, preventing the RNA from accumulating and impacting the cells. This kind of treatment reversed the molecular signs of progeria in isolated cells and extended the life span of mice with genetic mutations that typically cause premature aging. […] Targeting LINE-1 RNA may be an effective way to treat progeroid syndromes, as well as other age-related diseases that have been connected to LINE-1, including neuropsychiatric, eye, metabolic disorders and cancers, says Izpisua Belmonte, holder of the Roger Guillemin Chair. Eventually, we think that this approach may lead to treatments to help extend human health span. […] The researchers are planning future studies to better understand what causes the accumulation of LINE-1 RNA and how it can be prevented with drugs in human patients.

• #1 Research suggests promising drug therapy for premature aging disease | Brown University

  https://www.brown.edu/news/2018-04-24/progeria

  A study published today in the Journal of the American Medical Association suggests that an experimental drug therapy can extend the lives of children with progeria, a rare genetic disorder that causes premature aging and death. […] The study showed that children with progeria who were treated with lonafarnib, a drug originally developed to treat cancer, were more likely to survive over the course the study compared with children with progeria who did not receive the drug. […] This study provides supporting evidence that we can begin to put the brakes on the rapid aging process for children with progeria. […] The study showed a significantly lower mortality rate in the group receiving lonafarnib treatment. After two years, mortality in the treatment group was 3.7 percent, compared to 33.3 percent in the untreated group. […] But because most children with progeria die of heart disease and strokes, the lower mortality rate may have been attributable to cardiovascular and possibly cerebrovascular benefit.

• #1 Progeria

  https://www.mymlc.com/health-information/diseases-and-conditions/p/progeria/

  There’s no cure for progeria, but ongoing research shows some promise for treatment. […] Regular monitoring for heart and blood vessel (cardiovascular) disease may help with managing your child’s condition. […] Certain therapies may ease or delay some of the signs and symptoms. Treatments depend on your child’s condition and symptoms. These may include: Low-dose aspirin. A daily dose may help prevent heart attacks and stroke. […] 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.

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

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

  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. […] 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.

• #1 Hutchinson-Gilford Progeria Syndrome: Symptoms, Causes, Treatment!

  https://www.lybrate.com/topic/hutchinson-gilford-progeria-syndrome

  Even though it is hard to cure the medical condition, here are some of the steps that one can follow in order to have a healthy lifestyle. […] Dehydration is one of the most common yet serious conditions in children suffering from Hutchinson Gilford progeria syndrome. It is important to keep the patient hydrated, especially during an illness or during hot weather. […] Small frequent and wholesome meals are preferred for the patient with HGPS as it aids better digestion and optimum nutrition. Add high-calorie foods that are healthy and rich in nutrients. […] Keep the patient physically active, indulge the child in regular physical activity that is safe and appropriate as per the doctor. […] Since there is a loss of body fat in HGPS, it is important to get accessories like cushioned shoes and sunscreen SPF of at least 15 to protect the child from external damage.

• #1 Hutchinson-Gilford Progeria Syndrome: Symptoms, Causes, Treatment!

  https://www.lybrate.com/topic/hutchinson-gilford-progeria-syndrome

  Keep your child fully immunized as a child with progeria is quite vulnerable to bacterial, fungal, or viral infections. […] It is important to keep the learning and social opportunities open for the patient for their overall mental health. Progeria may lower the oxygen level in the brain vessels but it does not hinder the child’s intellect. […] Make certain changes at home like easily accessible switches and extra cushioned bedding and chairs to make the patient safe and comfortable at home. Other lifestyle adaptations like clothing with soft fabrics and easy handling can make the patient’s life easy and manageable.

• #1 Common Front Against Progeria, the Aging Child Disease | CiMUS

  https://cimus.usc.gal/news/common-front-against-progeria-aging-child-disease

  „Currently, there is no cure, and existing palliative therapies provide only limited benefits.” […] „The delay in diagnosing degenerative diseases like progeria means losing precious time that cannot be recovered when applying preventive or palliative treatments. We must be able to shorten these timelines, as it hinders preventive efforts.” […] „In fact, it takes an average of four years to receive a diagnosis, and in 20% of cases, it takes ten years or more to reach an accurate diagnosis.” […] „Despite being considered minority diseases, they represent a major socio-health challenge.” […] „In the case of progeria, there is currently no cure, and existing palliative therapies provide only limited benefits.” […] „Currently, multiple research efforts including those led by CiMUS aim to evaluate new treatments that, when combined with lonafarnib, could further increase both life expectancy and quality of life for affected children.” […] „The search for treatments is a long and complex road, but with adequate and sustained funding, results will come, sooner or later.”

• #1 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. […] While they wait, the researchers are figuring out the next steps to use their innovations to cure children with progeria. The team meets on Zoom every Monday at 4 p.m. […] Their goal is to obtain permission from the Food and Drug Administration to start a clinical trial. […] A key step will be finding a manufacturing partner to make the base editor for use in humans. […] „We want to start this trial in two years or less,” Dr. Collins said.

• #1 Eiger BioPharmaceuticals Announces FDA Approval of Zokinvy™ (lonafarnib): The First Treatment for Hutchinson-Gilford Progeria Syndrome and Processing-Deficient Progeroid Laminopathies – BioSpace

  https://www.biospace.com/eiger-biopharmaceuticals-announces-fda-approval-of-zokinvy-lonafarnib-the-first-treatment-for-hutchinson-gilford-progeria-syndrome-and-processing-deficient-progeroid-laminopathies

  Zokinvy is a disease-modifying agent that has demonstrated a statistically significant survival benefit in children and young adults with Progeria. […] The Rare Pediatric Disease Priority Review Voucher program is designed to encourage development of new drugs and biologics for the prevention or treatment of rare pediatric diseases. […] Zokinvy is indicated in adult and pediatric patients 12 months of age and older with a body surface area (BSA) of 0.39 m2 and above: To reduce the risk of mortality in Hutchinson-Gilford Progeria Syndrome (HGPS) […] Eiger’s established global Managed Access Program, expected to span greater than 40 countries, ensures all children and young adults with Progeria and Progeroid Laminopathies have access to treatment.

• #2 Progeria – Wikipedia

  https://en.wikipedia.org/wiki/Progeria

  In November 2020, the U.S. Food and Drug Administration approved lonafarnib, which helps prevent buildup of defective progerin and similar proteins. […] A clinical trial in 2018 points to significantly lower mortality rates treatment with lonafarnib alone compared with no treatment (3.7% vs. 33.3%) at a median post-trial follow-up time span of 2.2 years. […] Other treatment options have focused on reducing complications (such as cardiovascular disease) with coronary artery bypass surgery and low-dose acetylsalicylic acid. […] The use of Morpholinos has also been attempted in mice and cell cultures in order to reduce progerin production. […] Farnesyltransferase inhibitors (FTIs) are drugs that inhibit the activity of an enzyme needed to make a link between progerin proteins and farnesyl groups.

• #2 Research suggests promising drug therapy for premature aging disease | Brown University

  https://www.brown.edu/news/2018-04-24/progeria

  A study published today in the Journal of the American Medical Association suggests that an experimental drug therapy can extend the lives of children with progeria, a rare genetic disorder that causes premature aging and death. […] The study showed that children with progeria who were treated with lonafarnib, a drug originally developed to treat cancer, were more likely to survive over the course the study compared with children with progeria who did not receive the drug. […] This study provides supporting evidence that we can begin to put the brakes on the rapid aging process for children with progeria. […] The study showed a significantly lower mortality rate in the group receiving lonafarnib treatment. After two years, mortality in the treatment group was 3.7 percent, compared to 33.3 percent in the untreated group. […] But because most children with progeria die of heart disease and strokes, the lower mortality rate may have been attributable to cardiovascular and possibly cerebrovascular benefit.

• #2 Progeria: Sign, Symptoms & Risk Factors | HGPS Causes & Treatment

  https://www.delveinsight.com/blog/progeria-causes-symptoms-risk-factors-and-treatment-options

  Progeria is not curable; however, the treatment option eases or delays some of the signs and symptoms. Specific therapies for individuals with HGPS are symptomatic and supportive. Medication, changes in diet lifestyle, and physical occupational therapy are the most common treatment options. The treatments also depend on the child’s condition and symptoms. […] At present, some of the key companies such as PRG Science Technology, Merck, Eiger Biopharmaceuticals, and others are involved in developing therapies to treat and prevent progeria. As per DelveInsights analysis, several potential treatments for progeria are under investigation. These include newer agents, i.e., pravastatin + lonafarnib + zoledronic acid combinational product, and Progerinin (SLC-D011), for the treatment of the rare aging diseases (Hutchinson-Gilford Progeria Syndrome (HGPS) and Werner Syndrome (WS)).

• #2 Progeria – Wikipedia

  https://en.wikipedia.org/wiki/Progeria

  Studies of sirolimus, an mTOR Inhibitor, demonstrate that it can minimize the phenotypic effects of progeria fibroblasts. […] Recently, it has been demonstrated that the CRM1 protein (a key component of the nuclear export machinery in mammalian) is upregulated in HGPS cells, which drives to the abnormal localization of NES containing proteins from the nucleus to the cytoplasm.

• #2 All-trans retinoic acid and rapamycin normalize Hutchinson Gilford progeria fibroblast phenotype | Oncotarget

  https://www.oncotarget.com/article/4939/text/

  Moreover, promising results have been obtained in vitro by the use of rapamycin acting as an mTOR inhibitor which contributes to progerin degradation by activating autophagy. […] Aiming to reduce the intranuclear progerin accumulation, we investigated the effect of ATRA prolonged administration to HGPS skin fibroblasts. […] We further evaluated PARP1 protein expression by western blot analysis. […] These results demonstrated that the combined action of ATRA and rapamycin leads not only to downregulation of progerin expression, as stated in the previous paragraph, but also to protein degradation through the lysosomal and proteasomal pathways. […] We deduce that the higher lamin A to progerin ratio we obtain by ATRA plus rapamycin treatment of HGPS fibroblasts is sufficient to repair a series of nuclear parameters resulting in an almost complete nuclear normalization. […] These observations imply that in vivo confirmation of the efficacy of combined ATRA and rapamycin treatment will require a heterozygous animal model of progeria expressing both progerin and wild-type lamin A.

• #2 CRISPR base editor treats premature-aging syndrome | Signal Transduction and Targeted Therapy

  https://www.nature.com/articles/s41392-021-00576-6

  A recent paper published in Nature by Koblan et al. reported the use of CRISPR-mediated adenine base editor (ABE) to repair mutations of the HutchinsonGilford progeria syndrome (HGPS or progeria), attenuate symptoms, and extend lifespan of mice, representing a major advance in design of treatments for human accelerated-ageing disorders and potentially other genetic diseases. […] Attempts to discover treatments for progeria initially focused on trying to reduce the accumulation of farnesylated progerin. […] Therefore, it is necessary to develop a new strategy to directly repair the mutation that causes HGPS. […] ABE treatment attenuates some typical symptoms of HGPS disease in vivo, which may extend the life expectancy in the diseased mice. […] Correcting the mutation in HGPS mouse models provides a potential utility of ABE as a possible therapeutic approach to cure human HGPS.

• #2 Unique progerin C-terminal peptide ameliorates Hutchinson–Gilford progeria syndrome phenotype by rescuing BUBR1 | Nature Aging

  https://www.nature.com/articles/s43587-023-00361-w

  UPCP alleviated HGPS cellular senescence through BUBR1. […] UPCP ameliorated HGPS cellular senescence and progeroid features of LmnaG609G/G609G mice, suggesting that UPCP might be specific in alleviating HGPS senescence, which is a prerequisite for subsequent translation of HGPS therapeutic strategy. […] Therefore, UPCP may have broad application prospects in the treatment of HGPS.

• #2 Maryland Today | UMD-led Study Could Lead to Lengthened Lives for…

  https://today.umd.edu/umd-led-study-could-lead-to-lengthened-lives-for-patients-with-premature-aging-disease

  Ang2 treatment also improves endothelial cell signaling to vascular smooth muscle cells, suggesting it could be a potential therapy for vascular dysfunctions in HGPS, Vakili said. […] Current treatments for HGPS can help reduce the risk of fatal complications like heart attack and stroke, but they do not target the underlying disease. […] Cao explained that her lab’s research is unlikely to offer a definitive progeria cure, but it could buy patients more time by improving their health in other ways. […] 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.

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

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

  There’s no cure for progeria, but researchers are working on finding one. As of now, one drug has been approved by the FDA to slow the diseases course: lonafarnib (Zokinvy). This medication falls into a class of experimental cancer drugs called farnesyltransferase inhibitors (FTIs). Taking this drug helps people with progeria to gain weight. It also improves their bone and heart health. Studies have shown that the lonafarnib extends the lifespan of people with progeria by an average of 4.5 years. […] 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.

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

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

  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. […] 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.

• #2 Hutchinson-Gilford Progeria Syndrome: Symptoms, Causes, Treatment!

  https://www.lybrate.com/topic/hutchinson-gilford-progeria-syndrome

  Keep your child fully immunized as a child with progeria is quite vulnerable to bacterial, fungal, or viral infections. […] It is important to keep the learning and social opportunities open for the patient for their overall mental health. Progeria may lower the oxygen level in the brain vessels but it does not hinder the child’s intellect. […] Make certain changes at home like easily accessible switches and extra cushioned bedding and chairs to make the patient safe and comfortable at home. Other lifestyle adaptations like clothing with soft fabrics and easy handling can make the patient’s life easy and manageable.

• #3 CRISPR base editor treats premature-aging syndrome | Signal Transduction and Targeted Therapy

  https://www.nature.com/articles/s41392-021-00576-6

  Taken together, the current preliminary results from Koblan et al. demonstrate that ABE treatment has potential to correct the HGPS mutation in mice, fixing phenotypic traits and extending animal lifespan in a mouse model. These findings suggest a potential future functional base editing treatment for human HGPS and possibly other genetic disorders.

• #3 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

  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. […] 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.

• #4 Hutchinson-Gilford progeria syndrome – Indian Journal of Dermatology, Venereology and Leprology

  https://ijdvl.com/hutchinson-gilford-progeria-syndrome/

  Long-term follow-up is needed to observe the cardiovascular and skeletal abnormalities in these patients. […] Low-dose aspirin is recommended as prophylaxis to prevent atherosclerotic changes. […] In vitro studies in mice suggest a possible role for the use of farnesyltransferase inhibitors (FTIs) in progeria. […] A phase II trial of lonafarnib, an FTI, in progeria is currently ongoing in USA, along with two other drugs: pravastatin, a cholesterol-lowering agent and zoledronic acid, a biphosphonate, to prevent atherosclerosis and osteoporosis, respectively.

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