News Second death in gene therapy trial prompts investigation A second death in a study of Novartis’ spinal muscular atrophy gene therapy Zolgensma (onasemnogene abeparvovec-xioi; AVXS-101) – a potential blockbuster for this rare disease – has prompte
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 Biogen courts controversy with $750k-a-year rare disease dru... Biogen's SMA treatment approved - but price is $125,000 per dose
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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