Indication

Hutchinson-Gilford progeria syndrome

Hutchinson-Gilford progeria syndrome is an extremely rare, fatal genetic disorder of early childhood characterized by dramatically accelerated cellular aging and premature senescence. Read more about Hutchinson-Gilford progeria syndrome

2 drugs 2 manufacturers

Advertisement

Drugs that treat it

Common Hutchinson-Gilford progeria syndrome therapies.

Showing 1–2 of 2 drugs

ZOKINVY (lonafarnib)

PROGERIA SYNDROME TREATMENT AGENTS·HUTCHINSON-GILFORD PROGERIA SYNDROME·+2
SENTYNL THERAPU
Filter & compare in the drug finder →Opens the drug finder with Hutchinson-Gilford progeria syndrome preselected.

News & literature

Coming soon

Related codes

Coming soon
Coming soon

About Hutchinson-Gilford progeria syndrome.

Hutchinson-Gilford progeria syndrome is an extremely rare, fatal genetic disorder of early childhood characterized by dramatically accelerated cellular aging and premature senescence.

Pathophysiologically, it is caused by sporadic de novo mutations in the *LMNA* gene that produce progerin, a truncated, permanently farnesylated toxic variant of lamin A that disrupts nuclear envelope integrity and genomic stability.

Clinically, affected pediatric patients appear typical at birth but progressively develop profound growth failure, generalized lipodystrophy, alopecia, sclerodermatous skin changes, craniofacial disproportion, and skeletal dysplasia while retaining normal cognitive function.

The condition's defining systemic morbidity is rapidly progressive, premature atherosclerosis and arteriolosclerosis, which typically lead to fatal myocardial infarction or stroke during the second decade of life.

Personalize your experience

Follow Hutchinson-Gilford progeria syndrome and get updates as they land.

Create a free account

Follow an indication and get alerts when its drugs, labels, or guidelines change

Save any drug on this page to your favorites, synced across devices

AI-curated peer-reviewed research, summarized for your specialty

A morning digest of indication-relevant updates, when you want it