FDA approves Viltepso (viltolarsen) injection for treatment of Duchenne muscular dystrophy (DMD) in patients who have confirmed mutation of DMD gene that is amenable to exon 53 skipping

Viltepso was evaluated in 2 studies (n=32), 1 of which demonstrated an increase in dystrophin production (from 0.6% of normal at baseline to 5.9% of normal at week 25), which FDA concluded is reasonably likely to predict clinical benefit, but confirmatory trial will be required.


Biospace Inc.