muscular dystrophy
Sarepta Pauses EU Elevidys Trials, Data Monitoring Panel Confirms Favorable Risk-Benefit Profile
The firm said the clinical trial pause is temporary as an independent data monitoring committee finalizes its analysis for submission to regulators.
Epicrispr Biotechnologies Raises $68M in Series B Financing, Prepares to Launch Clinical Trial
The Series B financing will support an upcoming clinical trial in New Zealand of the company's lead program, EPI-321, in facioscapulohumeral muscular dystrophy.
Wave Life Sciences to File NDA for Duchenne Drug Next Year
The firm will seek accelerated approval for WVE-N531 in DMD amenable to exon 53 skipping based on FDA feedback and Phase II data.
Patient Dies After Treatment With Sarepta's Duchenne Muscular Dystrophy Gene Therapy
The firm's stock price dropped 27 percent on Tuesday following the news that the young man died from acute liver failure after getting Elevidys.
The positive results from the second part of the EMBARK trial contrast with findings from one-year post treatment, which did not meet the primary endpoint.