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About 523 results

ALLMedicine™ Hutchinson-Gilford Progeria Syndrome Center

Research & Reviews  191 results

Plasma Progerin in Patients With Hutchinson-Gilford Progeria Syndrome: Immunoassay Deve...
https://doi.org/10.1161/CIRCULATIONAHA.122.060002
Circulation Gordon LB, Norris W et. al.

Mar 16th, 2023 - Hutchinson-Gilford progeria syndrome (HGPS) is an ultrarare, fatal, premature aging disease caused by a toxic protein called progerin. Circulating progerin has not been previously detected, precluding research using readily available biological sa...

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

Feb 27th, 2023 - 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/II Trial of Everolimus in Combination With Lonafarnib in Progeria
https://clinicaltrials.gov/ct2/show/NCT02579044

Feb 27th, 2023 - 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...

Baseline Range of Motion, Strength, Motor Function, and Participation in Youth with Hut...
https://doi.org/10.1080/01942638.2022.2158054
Physical & Occupational Therapy in Pediatrics; Malloy J, Berry E et. al.

Jan 12th, 2023 - Limited information is available on impairments, activity limitations and participation restrictions in youth with Hutchinson-Gilford progeria syndrome (HGPS), a rare genetic premature aging disease. The purposes were to: (1) describe range of mot...

FDA approval summary for lonafarnib (Zokinvy) for the treatment of Hutchinson-Gilford p...
https://doi.org/10.1016/j.gim.2022.11.003
Genetics in Medicine : Official Journal of the American C... Suzuki M, Jeng LJB et. al.

Dec 13th, 2022 - The U.S. Food and Drug Administration recently approved lonafarnib as the first treatment for Hutchinson-Gilford progeria syndrome (HGPS) and processing-deficient progeroid laminopathies. This approval was primarily based on a comparison of patien...

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Drugs  1 results see all →

Clinicaltrials.gov  7 results

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

Feb 27th, 2023 - 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/II Trial of Everolimus in Combination With Lonafarnib in Progeria
https://clinicaltrials.gov/ct2/show/NCT02579044

Feb 27th, 2023 - 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...

Once Daily Dosing of Lonafarnib Co-administered With Ritonavir for Treatment of Chronic Hepatitis D Virus Infection
https://clinicaltrials.gov/ct2/show/NCT05229991

Feb 8th, 2022 - Background: Hepatitis D virus (HDV) was identified as the infectious agent causing viral hepatitis in the presence of hepatitis B virus (HBV). While HDV can replicate autonomously inside the hepatocyte, the virus requires co-infection with HBV to ...

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...

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...

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News  9 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...

FTase Inhibition Holds Promise for RAS Targeting and Beyond
https://www.onclive.com/view/ftase-inhibition-holds-promise-for-ras-targeting-and-beyond

Dec 5th, 2020 - Alan L. Ho, MD, PhD For more than 20 years, researchers have known that the RAS pathway is involved in a wide variety of cancer types. RAS proteins normally switch between an active state, which is bound by guanosine triphosphate, and an inactive...

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...

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Patient Education  1 results see all →