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CAMBRIDGE, Mass. —  Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced the U.S. Food and Drug Administration (FDA) has accepted and filed the Company’s efficacy supplement to the Biologics License Application (BLA) for ELEVIDYS (delandistrogene moxeparvovec-rokl) (the “Efficacy Supplement”). The goals of the...
CAMBRIDGE, Mass. – Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced positive results from Part A of the MOMENTUM study (Study 5051-201), a global, Phase 2, multi-ascending dose clinical trial of SRP-5051, its next-generation peptide phosphorodiamidate morpholino oligomer (PPMO) treatment for patients with...
CAMBRIDGE, Mass. —  Sarepta Therapeutics, Inc., the leader in precision genetic medicine for rare diseases, today announced submission of an efficacy supplement to the Biologics License Application (BLA) for ELEVIDYS (delandistrogene moxeparvovec-rokl) to expand its labeled indication as follows “[ELEVIDYS is indicated for] the treatment of Duchenne muscular dystrophy (DMD)...
CAMBRIDGE, Mass. – Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced positive 12-week expression and safety results from the first 11 participants enrolled in Study SRP-9001-103, an open-label study known as ENDEAVOR being conducted in partnership with Roche. In results from the first...
CAMBRIDGE, Mass. – Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today shared new  of SRP-9003 (rAAVrh74.MHCK7.hSGCB), the Company’s investigational gene therapy for limb-girdle muscular dystrophy Type 2E (LGMD2E). In the first look at expression data from biopsies taken two years after a single administration...
TORONTO, Canada — Satellos Bioscience Inc.(TSXV: MSCL) (OTCQB: MSCLF), a public biotech company developing new small molecule therapeutic approaches to improve the treatment of muscle diseases and disorders, announced today positive preliminary data showing SAT-3247 can improve skeletal muscle function in a mouse model of facioscapulohumeral muscular dystrophy (FSHD). Frank...
CHARLESTON, S.C. – A South Carolina mother who has been battling a rare genetic disease for years and lost her two sons to the disease is sharing her story as MUSC works to treat this rare disease. Living in Lake City, Chantel Bass is a South Carolina resident that has...