Hutchinson-Gilford Progeria Treatment Market Size, Share, Trend, Outlook, Future Analysis And Synthesis By 2026 Hutchinson-Gilford progeria syndrome is a genetic condition characterized by the dramatic, rapid, appearance of aging from the childhood. Hutchinson-Gilford progeria syndrome is caused by a mutation in the lamin A (LMNA) gene. The affected children develop a characteristic facial experience including prominent eyes, small chin, protruding ears, thin lips and a thin nose with a beaked tip. This syndrome also causes hair loss (alopecia), joint abnormalities, aged-looking skin, and a loss of fat under the skin (subcutaneous fat). Moreover, patients of Hutchinson-Gilford progeria syndrome experiences severe hardening of the arteries (arteriosclerosis) from the childhood. The condition worsens with age and increases the risk of heart attack or stroke even at a young age.
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Hutchinson-Gilford progeria is a rare condition that affects about one in four million newborns worldwide according to the National Institutes of Health (NIH). Until now more than 130 cases have been reported as per the NIH statistics. The affected patients live up to 30 years maximum, with an average life span of 13 years. Nearly 90% of the patients die from complications related to atherosclerosis. Till 2012 there wasn’t any effective treatment therapy discovered for Hutchinson-Gilford progeria syndrome. The treatments available focused mostly on reducing cardiovascular symptoms and growth abnormalities.
Farnesyltransferase Inhibitor (FTI)
In 2012, findings of the first clinical trial of the drug Lonafarnib, a farnesyltransferase inhibitor (FTI), gave a new hope for the treatment of children with Hutchinson-Gilford progeria syndrome. Clinical trial results demonstrated improvement in weight gain, increase in bone mineral density, reduced vascular stiffness, and improved sensorineural hearing in patients with progeria. Previous treatments with growth hormone, and Sulforaphane helped in reducing the