Investing.com - Sarepta Therapeutics Inc (NASDAQ:SRPT) shares jumped on Wednesday after the company reported positive results from a Phase 1/2 study for golodirsen (SRP-4053) treatment for Duchenne muscular dystrophy (DMD).
All 25 boys in Part 2 of the study experienced an increase in skipping exon 53 over baseline levels at week 48. DMD is a rare, and often deadly genetic disease. Patients with DMD lack dystrophin, a protein that helps keep muscle cells intact. Symptom onset is in early childhood, usually between ages 3 and 5. The disease primarily affects boys.
Sarepta's share jumped Wednesday, as these results sparked hopes for an accelerated approval for the drug. The latest study comes just a year after the company was granted approval for another DMD drug, Eteplirsen. Eteplirsen was the first U.S. drug approved for DMD, but its approval was marred by significant controversy and a great deal of disagreement at the Food and Drug Administration.
Complete study results will be submitted for presentation at a future medical conference. Sarepta's shares were recently up over 14%.