WebJun 28, 2024 · SOUTH PLAINFIELD, N.J., June 28, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has completed their review of a proposed indication extension of Translarna™ (ataluren) for the … WebIn May 2014, ataluren received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) and received market authorization from the European Commission to treat people with nonsense mutation Duchenne muscular dystrophy in August 2014; a confirmatory phase III clinical trial was ...
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WebIn August 2014, Translarna (ataluren) became the first drug to be given a conditional approval, by the European Medicines Agency (EMA), to treat an underlying genetic cause of Duchenne muscular dystrophy. Translarna has been designed to target a particular genetic mutation, called a ’nonsense mutation’ that causes 10-15 percent of cases of the … Ataluren was discovered by scientists at PTC Therapeutics in a collaboration with Lee Sweeney's lab at the University of Pennsylvania, which was initially funded in part by Parent Project Muscular Dystrophy. The team used phenotypic screening of a chemical library to identify compounds that increased the amount of protein expressed by mutated genes, and then optimized one of the hits in the screen to create this drug. As with the results of many cell-based screens, the biological ta… terri bradshaw wv
PTC Provides Update on Translarna™ (ataluren) Application for …
WebSep 17, 2024 · Ataluren (Translarna) is an investigational treatment being developed to treat Duchenne muscular dystrophy (DMD) resulting from a nonsense mutation in the … http://bo-rec2024.afm-telethon.fr/fr/essais/dystrophie-musculaire-de-duchenne-les-essais-cliniques-en-cours WebJun 29, 2024 · SOUTH PLAINFIELD, N.J., June 29, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the Committee for Medicinal … trifexis walmart