… in Patients with Duchenne Muscular Dystrophy Amenable to Skipping Exon 51 … resulted in mean dystrophin expression of 5.17% and mean exon skipping of 11.11% at 28 weeks (n=20) The data support a … in Patients with Duchenne Muscular Dystrophy Amenable to Skipping Exon 51 …
… Sarepta has several PMOs in our development pipeline. Our Exon-Skipping Approach Duchenne is caused by a genetic … in the dystrophin gene. Most commonly, one or more exons (parts of the gene) are missing, causing errors in the … morpholino oligomer (PMO) chemistries is the basis of our exon-skipping therapies for Duchenne muscular dystrophy. This …
… Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (MOMENTUM) …
… 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 …
… Forms Enrollment Form (English) Enrollment Form (Spanish) Exon Skipping Enrollment Forms Enrollment (Start) Form (English) … Forms Enrollment Form (English) Enrollment Form (Spanish) Exon Skipping Enrollment Forms Enrollment (Start) Form …
… a compassionate use program for any of our investigational exon skipping or gene therapies without jeopardizing our ability … a compassionate use program for any of our investigational exon skipping or gene therapies without jeopardizing our …
… trials for Duchenne underway in both gene therapy and RNA exon-skipping. Learn more about our Duchenne clinical trials … trials for Duchenne underway in both gene therapy and RNA exon-skipping. …
… Duchenne Muscular Dystrophy (DMD) in Patients Amenable to Skipping Exon 53 … -- VYONDYS 53 is Sarepta’s second RNA exon-skipping treatment for DMD approved in the U.S. -- -- … Duchenne Muscular Dystrophy (DMD) in Patients Amenable to Skipping Exon 53 …
… for Patients with Duchenne Muscular Dystrophy Amenable to Skipping Exon 45 … -- Casimersen is designed for the treatment of exon 45 amenable patients, approximately eight percent of … patients with Duchenne -- -- Casimersen is the third exon-skipping medicine using the Company’s proprietary PMO …
… in individuals living with Duchenne who are amenable to exon 45 skipping and exon 53 skipping, respectively. SRP-4045 (casimersen) and …
