Press release
Sarepta Therapeutics’ SRP-9001 Shows Sustained Functional Improvements in Multiple Studies of Patients with Duchenne
Results presented at ‘Micro-dystrophin Day’ highlight breadth, depth and strength of the clinical evidence to date for SRP-9001 in treating Duchenne muscular
About this update from Sarepta Therapeutics, Inc.
[{"type":"text","content":"Results presented at ‘Micro-dystrophin Day’ highlight breadth, depth and strength of the clinical evidence to date for SRP-9001 in treating Duchenne muscular dystrophy; tolerability profile remains consistent across treated patients Results from Study SRP-9001-101 found that SRP-9001-treated participants (n=4, ages 4 to 7 years) improved 8.6 points on the North Star Ambulatory Assessment (NSAA) compared to a matched natural history cohort three years after treatment Results from Study SRP-9001-102 found that SRP-9001-treated participants (n=12, ages 6 to 7) had a positive 2.9-point difference on NSAA compared to a matched natural history cohort one year after treatmentFunctional results from Study SRP-9001-103 Cohort 1 (n=11, ages 4-7) found participants improved 3.0 points on NSAA six months after treatment EMBARK, the first global Phase 3 pivotal double-blind gene therapy trial in Duchenne to enroll 120 patients in the US, Europe and Asia, conducted in partnership with Roche CAMBRIDGE, Mass., Oct. 11, 2021 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today shared new analyses and functional data from its SRP-9001 (rAAVrh74.MHCK7.micro-dystrophin) development program and details of Study SRP-9001-301, known as EMBARK, its global pivotal Phase 3 trial of SRP-9001 for the treatment of Duchenne muscular dystrophy. SRP-9001, being developed in partnership with Roche, is an investigational gene transfer therapy intended to deliver its micro-dystrophin-encoding gene to muscle tissue for the targeted production of the micro-dystrophin protein. In new analyses presented at “SRP-9001 Micro-dystrophin Day,” results from participants treated with SRP-9001 in Study SRP-9001-101 (n=4, ages 4 to 7) found that participants in Study 101 improved 8.6 points on the North Star Ambulatory Assessment (NSAA)* compared to a matched natural history cohort three years following a single administration of SRP-9001 (p","length":2690,"tagName":"div"}]