No slam dunk, but DMD data could secure future for Translarna
After repeated failures to register its Duchenne muscular dystrophy (DMD) drug in the USA, new data suggest PTC Therapeutics (Nasdaq: PTCT) might be about ready to try again.
The New Jersey, USA-based rare disease drug developer has long had conditional marketing approval in Europe for ataluren, a novel protein restoration remedy which the company markets as Translarna.
The US Food and Drug Administration has remained unpersuaded, however, rejecting a submission from PTC for the third time in late 2017.
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