Tag Archives: Ataluren

October, 2018

8 October
New Data Shows PTC Therapeutics’ Translarna Slows Disease Progression in Children with Duchenne Caused by a Nonsense Mutation

SOUTH PLAINFIELD, N.J., Oct. 6, 2018 /PRNewswire/ — PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced preliminary data from the first international drug registry for Duchenne patients receiving Translarna™ (ataluren), underscoring the long-term clinical benefit of Translarna when used in routine clinical practice in delaying irreversible muscle loss in children with Duchenne caused by a nonsense …
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26 October
FDA Denies Approval of PTC’s Duchenne Muscular Dystrophy Drug Translarna

SOUTH PLAINFIELD, N.J., Oct. 25, 2017 /PRNewswire/ — PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the Office of Drug Evaluation I of the U.S. Food and Drug Administration (FDA) has issued a complete response letter (CRL) for the New Drug Application (NDA) of the investigational medicine ataluren for the …
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3 March
PTC Therapeutics Discontinues Ataluren Program After Failed Phase 3 Trial in Nonsense Mutation Cystic Fibrosis

SOUTH PLAINFIELD, N.J., March 2, 2017 /PRNewswire/ — PTC Therapeutics, Inc. (NASDAQ: PTCT), today announced that the Ataluren Confirmatory Trial (ACT CF) in nonsense mutation cystic fibrosis (nmCF) did not achieve its primary or secondary endpoints. Ataluren was generally well tolerated and ACT CF confirmed a favorable safety profile for ataluren, …
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18 April
UK’s NICE Recommends PTC Therapeutics’ Drug for Duchenne Muscular Dystrophy

NICE has today recommended ataluren (Translarna, PTC Therapeutics) for treating children aged 5 and over with Duchenne muscular dystrophy (DMD) caused by a nonsense mutation. The drug has been called a ‘step change’ in the management of the disease which causes progressive muscle wasting and is usually fatal by age …
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