News FDA reviewers slam PTC's Duchenne drug ahead of meeting FDA staff not persuaded by patchy clinical trial evidence for Translarna.
News Sarepta announces Allergan veteran as next CEO Sarepta has announced former Allergan president Doug Ingram will be its new CEO.
News UK patients gain early access to Duchenne drug Patients to get Santhera's Raxone under EAMS scheme.
News Outrage over Duchenne drug price reinflamed by PTC's $35,000... Families affected by the rare muscle wasting disease Duchenne muscular dystrophy will still have to pay a huge premium for Emflaza, a newly-approved drug based on an old steroid that helps
News Sarepta files controversial Duchenne drug in EU US biotech seeks conditional approval for eteplirsen in EU.
News Neurogene drops high-dose arm in Rett study after death The patient affected by a serious adverse event in Neurogene's clinical trial of Rett syndrome gene therapy has now died
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