US FDA approves casimersen injection for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 45 skipping
The approval, the first of a targeted treatment for patients with this type of mutation, was based on a placebo-controlled study (n=43) which found those treated with casimersen had a significantly greater increase in dystrophin protein levels from baseline to week 48.
Source:
US Food and Drug Administration