American biotech PTC Therapeutics (Nasdaq: PTCT) has submitted to the US regulator for approval to market Upstaza (eladocagene exuparvovec).
The gene therapy has been developed for the severe and fatal genetic disorder aromatic L-amino acid decarboxylase (AADC) deficiency.
In another boost to the company’s prospects, PTC said it would now also resubmit for approval for Translarna (ataluren), for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD).
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