Shares of Sarepta Therapeutics rise on early promise for its rare disease drugs[original content]. Another batch of drugs for muscular dystrophy appears safe and effective in early clinical trials, the company reported[original content][1][3]. This is the global MOMENTUM (5051-201), Phase 2 Part A study, which is testing multiple dosing of SRP-5051 in patients with DMD and an appropriate exon 51 skipping mutation[1]. SRP-5051 is a new generation of eteplirsen, it uses PPMO (peptide phosphorodiamidate morpholino oligomer) technology for better penetration into muscle cells[1]. The goal is to verify the maximum tolerated dose[1]. The results indicate higher efficacy with less frequent dosing compared to eteplirsen[1][3]. Sarepta's predictive model shows that at a dose of 30 mg/kg, SRP-5051 achieves more than 10% dystrophin production at monthly dosing[1].