Hutchinson-Gilford progeria syndrome
MONDO:0008310Hutchinson-Gilford progeria syndrome is a rare, fatal, autosomal dominant and premature aging disease, beginning in childhood and characterized by growth reduction, failure to thrive, a typical facial appearance (prominent forehead, protuberant eyes, thin nose with a beaked tip, thin lips, micrognathia and protruding ears) and distinct dermatologic features (generalized alopecia, aged-looking skin, sclerotic and dimpled skin over the abdomen and extremities, prominent cutaneous vasculature, dyspigmentation, nail hypoplasia and loss of subcutaneous fat).
Also known as: progeria, HGPS, Hutchinson-Gilford disease, Hutchinson-Gilford progeria, Hutchinson-Gilford progeria syndrome, premature senility syndrome, Hutchinson Gilford progeria syndrome, progeria syndrome, childhood-onset
3 clinical trials for this condition and its sub-types.
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New drug combo aims to slow Progeria's effects
Disease control ENROLLING_BY_INVITATIONThis study tests whether adding everolimus to the existing drug lonafarnib can better control progeria, a rare disease that causes rapid aging in children. About 80 children with confirmed progeria will take both pills by mouth. The first part finds the safest dose of everolimus,…
Phase: PHASE1, PHASE2 • Sponsor: Boston Children's Hospital • Aim: Disease control
Last updated Jun 27, 2026 12:03 UTC
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New hope for kids with rapid-aging disease: drug combo tested
Disease control OngoingThis study tests a new drug called progerinin, given together with the standard medicine lonafarnib, in 10 children with Hutchinson-Gilford progeria syndrome (a rare disease that causes rapid aging). The goal is to find the best dose and check if the combination is safe and toler…
Phase: PHASE2 • Sponsor: PRG Science & Technology Co., Ltd. • Aim: Disease control
Last updated Jun 27, 2026 09:03 UTC