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Home > Drugs > Treatments > Hutchinson-Gilford Syndrome

Medications for Hutchinson-Gilford Syndrome

Other names: HGPS; Hutchinson-Gilford Progeria Syndrome; Progeria

Hutchinson-Gilford syndrome is a rare and fatal genetic condition of accelerated aging in children.

Hutchinson-Gilford syndrome is caused by a mutation in the LMNA gene, which encodes the lamin A protein, yielding the farnesylated aberrant protein, progerin. The lamin A protein is the scaffolding that holds the nucleus of a cell together, and it plays an important role in determining the shape of the nucleus within cells. Mutations in the LMNA gene result in the production of an abnormal version of the lamin A protein, making the nuclear envelope unstable and causing damage to the nucleus, making it more likely that cells die prematurely.

Children who have Hutchinson-Gilford syndrome can experience severe failure to thrive, scleroderma‐like skin, global lipodystrophy, alopecia, joint contractures, skeletal dysplasia, global accelerated atherosclerosis with cardiovascular decline, and debilitating strokes. Without treatment, affected children experience accelerated mortality with most dying of heart disease at an average age of 14.5 years.

Zokinvy (lonafarnib) is the first treatment FDA approved for reducing the risk of mortality in children with Hutchinson-Gilford syndrome. It is a farnesyltransferase inhibitor that works by inhibiting farnesyltransferase to block the accumulation of the defective, farnesylated proteins. 

Drugs used to treat Hutchinson-Gilford Syndrome

Name Drug Class Updated
Lonafarnib Miscellaneous metabolic agents 13-Aug-2023
Zokinvy Miscellaneous metabolic agents 14-Jul-2023
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