25 February 2021 - Today, the U.S. FDA granted approval for Amondys 45 (casimersen) injection for the treatment of Duchenne muscular dystrophy in patients who have a confirmed mutation of the DMD gene that is amenable to exon 45 skipping. 

The agency approved Amondys 45 based on an increase in dystrophin (a protein that helps keep muscle cells intact) production in skeletal muscle observed in patients treated with the therapy. 

This is the first FDA approved targeted treatment for patients with this type of mutation.

Read FDA press release