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 Novartis' SMA gene therapy cleared for wider use in US A new FDA approval means Novartis now has a gene therapy option for almost everyone with spinal muscular atrophy (SMA), regardless of their age.
News Sarepta shares slide as DMD treatment woes pile up The ESSENCE trial of Sarepta's Vyondys 53 and Amondys 45 failed to hit its objective, placing their FDA accelerated approvals at risk.
News Novartis makes $12bn wager on RNA drugs with Avidity buy Novartis is poised to make one of its largest takeover deals in its history after agreeing to buy RNA specialist Avidity Biosciences for $12bn.
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 Rumour mill says Novartis may want to buy Avidity Novartis has made an offer to acquire RNA therapeutic specialist Avidity Biosciences, according to an unconfirmed report.
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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