Related: Teva wins FDA approval for first generic weight loss GLP-1 Growing pains Despite significant investment and scientific advancement in muscular dystrophy research, it hasn't been all plain sailing. One of the most popular tools in gene therapy, AAV, has recently fallen out of favor over recent safety concerns. Sarepta came under fire when its use of viral vector AAVrh74 was linked to the acute liver failure and eventual death of two non-ambulatory patients. The voluntary hold on Elevidys was then lifted by the US Food and Drug Administration (FDA) for ambulatory patients. Nevertheless, the company has pivoted away from AAV-based drugs to prioritize pipelines focusing on siRNA therapies, triggering a restructure that cut over 500 jobs. Multiple clinical trials from Sarepta were withdrawn or terminated in the process (see here, here, and here for examples). The shift elicited a statement of concern from nonprofit groups supporting LGMD patients.