News FDA probing deaths with DMD gene therapy Elevidys The FDA has launched a formal investigation into two liver failure-related deaths in patients treated with Duchenne gene therapy Elevidys.
News DMD gene therapy Elevidys linked to new liver failure case Another death from liver failure has been seen with Roche and Sarepta's Duchenne muscular dystrophy therapy Elevidys, prompting a pause on treatment.
News Multiple studies of Sarepta's DMD therapy halted after death Three studies of Sarepta's Elevidys Duchenne gene therapy have been placed on hold as the death of a 16-year-old boy is investigated.
News Trial of Boehringer's cystic fibrosis gene therapy imminent Boehringer's cystic fibrosis gene therapy is poised to start clinical testing, hoping to offer a one-shot alternative to costly CFTR modulator drugs.
News New data could widen use of Novartis' SMA therapy Zolgensma Phase 3 results with an intrathecal version of Novartis spinal muscular atrophy (SMA) gene therapy could make older children eligible for treatment.
R&D From promise to reality: Unlocking the true business potenti... The genomic medicine space has seen incredible success, but investors are overlooking it.
