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 Sarepta rises after FDA adds warning label to Elevidys Uncertainty ends Sarepta's Duchenne gene therapy Elevidys ends after an FDA label update, with the company's shares on the up despite restrictions.
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 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 FDA gives the go-ahead to restart Elevidys shipments Sarepta has restarted distribution of its Duchenne muscular dystrophy gene therapy Elevidys, but only for patients who can walk.
News Sarepta backs down and pauses Elevidys shipments The FDA seems to have won its staring contest with Sarepta over shipments of Elevidys, as the company blinks and agrees to a pause from today.
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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