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PTC Therapeutics Reports Mixed Results from Dystrophy Trial

PTC Therapeutics Inc. PTCT 4.60 % reported that its treatment for a form of Duchenne muscular dystrophy failed to show a statistically significant benefit to the primary goal of the late-stage clinical trial.

However, for a certain group of pre-specified patients, PTC’s treatment—called Translarna—showed a highly significant benefit. As a result, PTC plans to complete its new drug application with the U.S. Food and Drug Administration by year’s end.

Translarna is PTC Therapeutics’ lead product candidate and has conditional marketing authorization in the European Union.

Muscular dystrophy is an umbrella term for more than 30 inherited diseases that cause muscles to weaken and wither away, and that currently have no cure. Duchenne muscular dystrophy patients, which are mostly boys and...


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