3. It probably works. Maybe.

The first drug therapy for Duchenne muscular dystrophy (DMD), eteplirsen, is now on the accelerated approval pathway by the FDA. It’s a gene therapy that allows 13% of DMD patients to partially produce an otherwise absent protein, which slows the progression of DMD. At least, that’s what the supposed clinical benefit is. Trials run by Sarepta Therapeutics, the drug’s manufacturer, do show an increase in the otherwise missing protein, but haven’t effectively linked those increased levels to a clinical benefit yet. The trials performed so far weren’t randomized, which our readers may recognize as… not the best way to collect unbiased data. But the increased protein levels seem promising enough for the FDA. The accelerated approval also comes with priority review and an orphan drug designation.