Lonafarnib

Generic Name
Lonafarnib
Brand Names
Zokinvy
Drug Type
Small Molecule
Chemical Formula
C27H31Br2ClN4O2
CAS Number
193275-84-2
Unique Ingredient Identifier
IOW153004F
Background

Hutchinson-Gilford progeria syndrome (HGPS) is a rare autosomal dominant disorder estimated to affect approximately one in 20 million individuals resulting in adverse symptoms associated with premature ageing: skeletal dysplasia, joint contractures, atherosclerosis, myocardial fibrosis/dysfunction, scleroderma-like cutaneous effects, lipoatrophy, alopecia, a...

Indication

Lonafarnib is a farnesyltransferase inhibitor indicated in patients aged 12 months and older with a body surface area of at least 0.39 m to reduce the risk of mortality associated with Hutchinson-Gilford progeria syndrome (HGPS). It is also indicated in this same population for the treatment of processing-deficient progeroid laminopathies that either involve...

Associated Conditions
Death, Hutchinson-Gilford Progeria Syndrome, Processing-deficient Progeroid Laminopathies
Associated Therapies
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