PTC Therapeutics Announces Initial Data from Patient Registry Demonstrating Translarna™ (ataluren) Slows Disease Progression in Children with Duchenne Caused by a Nonsense Mutation | DuchenneXchange

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PTC Therapeutics Announces Initial Data from Patient Registry Demonstrating Translarna™ (ataluren) Slows Disease Progression in Children with Duchenne Caused by a Nonsense Mutation


PTC Therapeutics, Inc. 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 mutation, when compared with published natural history. The data show that children and adolescents receiving Translarna in the real-world setting are continuing to walk years longer than untreated children, and are staying more physically able. A time-to-event analysis for loss of ambulation has shown that patients on Translarna had a median age of loss of ambulation of 16.5 years of age – up to 5 years later than seen with natural disease progression in untreated children. The data were presented as a late breaker at the 23rd International Annual Congress of the World Muscle Society in Argentina.

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