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Sarepta Therapeutics Announces Positive Data from Part B of MOMENTUM, a Phase 2 Study of SRP-5051 in Patients with Duchenne Muscular Dystrophy Amenable to Skipping Exon 51
… in Patients with Duchenne Muscular Dystrophy Amenable to Skipping Exon 51 … At target dose (~30 mg/kg), SRP-5051 dosed monthly …
RNA Platform
… Sarepta has several PMOs in our development pipeline. Our Exon-Skipping Approach Duchenne is caused by a genetic mutation in …
Two-Part Study for Dose Determination of Vesleteplirsen (SRP-5051) (Part A), Then Dose Efficacy (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (MOMENTUM)
… Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (MOMENTUM) …
Community Letter: Update SRP-5051 Program
… or PPMO, to treat people with Duchenne who are amenable to exon 51 skipping. This means dosing in the MOMENTUM study, … option that would build upon our FDA-approved PMO exon skipping therapy. While we were encouraged by the …
SareptAssist
… Forms Enrollment Form (English) Enrollment Form (Spanish) Exon Skipping Enrollment Forms Enrollment (Start) Form (English) …
Treatment Access
… a compassionate use program for any of our investigational exon skipping or gene therapies without jeopardizing our ability …
Duchenne Muscular Dystrophy
… trials for Duchenne underway in both gene therapy and RNA exon-skipping. Learn more about our Duchenne clinical trials …
Sarepta Therapeutics Announces FDA Approval of VYONDYS 53™ (golodirsen) Injection for the Treatment of Duchenne Muscular Dystrophy (DMD) in Patients Amenable to Skipping Exon 53
… Duchenne Muscular Dystrophy (DMD) in Patients Amenable to Skipping Exon 53 … -- VYONDYS 53 is Sarepta’s second RNA exon-skipping …
Sarepta Therapeutics Completes Submission of New Drug Application Seeking Approval of Casimersen (SRP-4045) for Patients with Duchenne Muscular Dystrophy Amenable to Skipping Exon 45
… for Patients with Duchenne Muscular Dystrophy Amenable to Skipping Exon 45 … -- Casimersen is designed for the treatment of exon …