News PTC pulls file for Duchenne therapy on FDA feedback PTC Therapeutics has abandoned its latest attempt to secure FDA approval for Duchenne muscular dystrophy treatment Translarna.
News PTC stung by FDA decision not to approve rare disease drug It looks like PTC will have to carry out another trial of its drug for Friedreich ataxia before the FDA will consider approval.
News Germany will be first launch for PTC's phenylketonuria drug PTC Therapeutics' treatment for the rare disease PKU, Sephience, will launch in its first market next month after getting EU approval.
News PTC's Huntington study was positive. Why did its stock fall? In a potentially pivotal trial, PTC Therapeutics' therapy for Huntington's disease met its primary endpoint, but there are questions about the data.
News Italfarmaco closes on EU okay for Duchenne drug Italfarmaco's oral HDAC inhibitor Duvyzat has been recommended for approval in the EU as a treatment for Duchenne muscular dystrophy (DMD).
News Novartis pays $1bn upfront for Huntington's drug from PTC Novartis has made a return to the Huntington's disease stage by licensing an oral mRNA splicer from PTC Therapeutics for $1 billion upfront.
News NHS "isn't delivering equitable care for rare diseases" A Genetic Alliance UK report claims the NHS is failing to meet the needs of many of the 3.5 million people in the country who live with rare diseases.
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