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ALLMedicine™ Hutchinson-Gilford Progeria Syndrome Center

Research & Reviews  177 results

Progeria: a perspective on potential drug targets and treatment strategies.
https://doi.org/10.1080/14728222.2022.2078699
Expert Opinion on Therapeutic Targets; Benedicto I, Chen X et. al.

May 17th, 2022 - Hutchinson-Gilford progeria syndrome (HGPS) is an ultra-rare genetic disease characterized by premature aging and death at an average age of 14.6 years. Most HGPS patients are heterozygous carriers of a de novo synonymous mutation in the LMNA gene...

Cell immortalization facilitates prelamin A clearance by increasing both cell prolifera...
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8954962
Aging González-Blanco C, Marqués P et. al.

Mar 21st, 2022 - Hutchinson-Gilford Progeria Syndrome is an ultrarare disease which is characterized by an accelerated senescence phenotype with deleterious consequences to people suffering this pathology. The production of an abnormal protein derived from lamin A...

Study of Zoledronic Acid, Pravastatin, and Lonafarnib for Patients With Progeria
https://clinicaltrials.gov/ct2/show/NCT00916747

Mar 9th, 2022 - Hutchinson-Gilford Progeria Syndrome (Progeria) is a rare autosomal disease that results in premature death at a median age of 13 years due to cardiovascular and cerebralvascular compromise. The mutation for this disease has been identified and re...

Abolishing the prelamin A ZMPSTE24 cleavage site leads to progeroid phenotypes with nea...
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8892526
Proceedings of the National Academy of Sciences of the Un... Wang Y, Shilagardi K et. al.

Feb 25th, 2022 - Prelamin A is a farnesylated precursor of lamin A, a nuclear lamina protein. Accumulation of the farnesylated prelamin A variant progerin, with an internal deletion including its processing site, causes Hutchinson-Gilford progeria syndrome. Loss-o...

LMNA-mediated nucleoskeleton dysregulation in Alzheimer disease.
https://doi.org/10.1002/alz.054396
Alzheimer's & Dementia : the Journal of the Alzheimer's A... Rosene MJ, Wen N et. al.

Feb 4th, 2022 - Nucleoskeleton dysfunction has been implicated in Alzheimer disease (AD). Tubular invaginations of the nuclear envelope observed in AD brains are consistent with the accumulation of farnesylated prelamin A (encoded by the LMNA gene) that occurs in...

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Drugs  1 results see all →

Clinicaltrials.gov  6 results

Study of Zoledronic Acid, Pravastatin, and Lonafarnib for Patients With Progeria
https://clinicaltrials.gov/ct2/show/NCT00916747

Mar 9th, 2022 - Hutchinson-Gilford Progeria Syndrome (Progeria) is a rare autosomal disease that results in premature death at a median age of 13 years due to cardiovascular and cerebralvascular compromise. The mutation for this disease has been identified and re...

Phase I Study of Progerinin in Healthy Volunteers
https://clinicaltrials.gov/ct2/show/NCT04512963

Sep 22nd, 2021 - PRG Science & Technology Co., Ltd. (PRG S&T) is developing Progerinin (SLC-D011) for the treatment of the rare aging diseases Hutchinson-Gilford Progeria Syndrome (HGPS) and Werner Syndrome (WS). Progerin, an abnormal splice variant of the inner n...

Phase I/II Trial of Everolimus in Combination With Lonafarnib in Progeria
https://clinicaltrials.gov/ct2/show/NCT02579044

Jun 14th, 2021 - This is a phase I/II dose-escalation trial of everolimus in combination with lonafarnib in Hutchinson-Gilford Progeria Syndrome (HGPS) and progeroid laminopathies (henceforth "progeria"). The study will be conducted at a single clinical site utili...

Lonafarnib for Patients With Hutchinson-Gilford Progeria Syndrome or Progeroid Laminopathy
https://clinicaltrials.gov/ct2/show/NCT03895528

Apr 15th, 2021 - This treatment IND protocol will allow patients with HGPS and progeroid laminopathies access to lonafarnib, the only compound shown to have an effect on the HGPS disease process resulting in improved outcomes (Gordon et al, 2018). There are no app...

Phase II Trial of Lonafarnib (a Farnesyltransferase Inhibitor) for Progeria
https://clinicaltrials.gov/ct2/show/NCT00425607

Jun 25th, 2019 - Hutchinson-Gilford Progeria Syndrome (HGPS) is a rare "premature aging" disease in which all children die at an average age of thirteen years (range 8-20 years) of severe atherosclerosis leading to strokes and heart attacks. It is a multisystem di...

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News  7 results

FDA Approvals, Highlights, and Summaries: Pediatrics
https://reference.medscape.com/viewarticle/945192

Feb 24th, 2021 - Pediatric Endocrinology Zokinvy (lonafarnib) Lonafarnib, an oral farnesyltransferase inhibitor, is indicated to reduce the risk of mortality in Hutchinson-Gilford progeria syndrome (HGPS). It was also approved for processing-deficient progeroid la...

FDA Approvals, Highlights, and Summaries: Endocrinology
https://reference.medscape.com/viewarticle/945183

Feb 24th, 2021 - Zokinvy (lonafarnib) Lonafarnib, an oral farnesyltransferase inhibitor, is indicated to reduce the risk of mortality in Hutchinson-Gilford progeria syndrome (HGPS). It was also approved for processing-deficient progeroid laminopathies with heteroz...

Progeria Often Comes with Silent LV Dysfunction
https://www.medpagetoday.com/cardiology/chf/71312

Feb 22nd, 2018 - The early cardiovascular death typical of Hutchinson-Gilford progeria syndrome may hide a surprising level of asymptomatic left ventricular (LV) diastolic dysfunction, researchers found. In a single-center series of 27 consecutive patients, ages 2...

FDA Approves First Treatment for Hutchinson-Gilford Progeria Syndrome and Some Progeroid Laminopathies
http://www.fda.gov/news-events/press-announcements/fda-approves-first-treatment-hutchinson-gilford-progeria-syndrome-and-some-progeroid-laminopathies

For Immediate Release: November 20, 2020 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-defici...

Drug Trials Snapshots: ZOKINVY
http://www.fda.gov/drugs/drug-approvals-and-databases/drug-trials-snapshots-zokinvy

HOW TO USE THIS SNAPSHOT The information provided in Snapshots highlights who participated in the clinical trials that supported the FDA approval of this drug, and whether there were differences among sex, race and age groups. The “MORE INFO” bar ...

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Patient Education  1 results see all →