News PTC shares soar after SMA drug data impresses market After failing three times to bring their Duchenne therapy to the US market, PTC Therapeutics might be on verge of a breakthrough in the SMA field.
News Pharma collaborates to improve chances of NICE backing Duche... Project will initially focus on DMD but could benefit other rare diseases
News PTC Therapeutics fails to convince FDA panel about DMD drug FDA advisers have voted against approval for PTC Therapeutics’ Duchenne drug Translarna because of doubts about its efficacy.
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 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 Ipsen' Bylvay fluffs its lines in biliary atresia trial Ipsen's IBAT inhibitor Bylvay has failed a trial in biliary atresia, a rare and serious liver disease that affects babies and has limited treatments.
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