SRPT - Sarepta Therapeutics' Next-Gen Duchenne Muscular Therapy Shows Efficacy At Less Frequent Dosing | Benzinga
Sarepta Therapeutics Inc (NASDAQ:SRPT) announced data from Part B of the MOMENTUM study (Study SRP-5051-201) Phase 2, multi-ascending dose trial of SRP-5051 (vesleteplirsen) that enrolled Duchenne muscular dystrophy (DMD) patients aged 8 to 21 years.
SRP-5051 is a next-generation peptide phosphorodiamidate morpholino oligomer (PPMO) treatment for patients with DMD amenable to exon 51 skipping.
SRP-5051 is designed to work similarly to Exondys 51 (eteplirsen), an older Exon 51-skipping therapy developed by Sarepta
Data from Part B of MOMENTUM found that at the higher target dose, approximately 30 mg/kg dosed every four weeks, SRP-5051 resulted in mean dystrophin expression of ...