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-- Ten abstracts, including four podium presentations, reflect Sareptas ongoing commitment to advancing genetic medicine for rare neuromuscular disease and facilitating greater understanding of these devastating conditions --


GlobeNewswire Inc | Mar 15, 2021 08:30AM EDT

March 15, 2021

-- Ten abstracts, including four podium presentations, reflect Sareptas ongoing commitment to advancing genetic medicine for rare neuromuscular disease and facilitating greater understanding of these devastating conditions --

CAMBRIDGE, Mass., March 15, 2021 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, will present at the Muscular Dystrophy Association (MDA) Annual Clinical and Scientific Conference, which will take place virtually March 15-18, 2021. Among the research that will be presented:

-- New, two- and one-year data including expression data from muscle biopsies taken two years post-treatment, from Study 9003-101 of SRP-9003, an investigational gene therapy for the treatment of limb-girdle muscular dystrophy (LGMD) type 2E/R4; -- Data from Part 1 of Study 9001-102, an ongoing clinical trial of SRP-9001, Sareptas investigational gene therapy for Duchenne muscular dystrophy and pre-clinical approaches to the challenge of pre-existing antibodies; and, -- An analysis of time to loss of ambulation in patients taking eteplirsen, a phosphorodiamidate morpholino oligomer (PMO) for the treatment of Duchenne, compared to standard of care.

All posters are available on-demand throughout the Congress beginning on Monday, March 15, 2021 at 6:00 a.m. ET. Podium presentations will take place on Thursday, March 18, 2021. The full MDA 2021 Virtual Congress program is available here: https://mdaconference.org.

Podium Presentations:

Title Program Date, Time Thurs.,Micro-dystrophin Gene Therapy Delivery and Therapeutic SRP-9001 March 18,Plasma Exchange in Non-Human Primates 2021 10:30-10:45 AM ETA Randomized, Double-Blind, Placebo-Controlled, Thurs.,Gene-Delivery Clinical Trial of March 18,rAAVrh74.MHCK7.micro-dystrophin for Duchenne Muscular SRP-9001 2021Dystrophy 3:30-3:45 PM ETSafety,-sarcoglycanExpression, and Functional Thurs.,Outcomes from Systemic Gene Transfer of March 18,rAAVrh74.MHCK7.SGCB in LimbGirdle Muscular Dystrophy SRP-9003 2021Type 2E/R4 4:30-4:45 PM ET Thurs.,Delay in Duchenne Muscular Dystrophy Progression with March 18,Eteplirsen: Longer Time to Loss of Ambulation Versus Eteplirsen 2021Standard of Care 5:45-6:00 PM ET

Poster Presentations:

Poster Title# Biological Efficacy of the Peptide-Conjugated Phosphorodiamidate6 Morpholino Oligomer SRP-5051 in Preclinical Models of Duchenne Muscular Dystrophy Casimersen Treatment in Eligible Patients with Duchenne Muscular54 Dystrophy: Safety, Tolerability, and Pharmacokinetics Over 144 Weeks of Treatment90 Patterns of Clinical Progression Among Patients with Autosomal Recessive Limb-Girdle Muscular Dystrophy (LGMDR): A Systematic Review92 Progression to Loss of Ambulation (LOA) Among Patients with Autosomal Recessive Limb-Girdle Muscular Dystrophy (LGMDR): A Systematic Review104 Health-related quality of life (HRQoL) Associated with Duchenne Muscular Dystrophy (DMD): A Study Using the Health Utilities Index Mark 3 (HUI3) NorthStar Ambulatory Assessment (NSAA) and Health Utilities Index (HUI)113 Scores are Weakly Correlated Among Boys with Duchenne Muscular Dystrophy (DMD)

Presentations will be archived on the events and presentations page in the Investor Relations section of www.sarepta.com for one year following their presentation at MDA.

AboutSarepta TherapeuticsAt Sarepta, we are leading a revolution in precision genetic medicine and every day is an opportunity to change the lives of people living with rare disease. The Company has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophies (LGMDs), mucopolysaccharidosis type IIIA, Charcot-Marie-Tooth (CMT), and other CNS-related disorders, with more than 40 programs in various stages of development. The Companys programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. For more information, please visitwww.sarepta.com or follow us on Twitter, LinkedIn, Instagram and Facebook.

Internet Posting of Information

We routinely post information that may be important to investors in the 'For Investors' section of our website atwww.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us.

Source: Sarepta Therapeutics, Inc.

Sarepta Therapeutics, Inc.

Investors:Ian Estepan, 617-274-4052, iestepan@sarepta.com

Media:Tracy Sorrentino, 617-301-8566, tsorrentino@sarepta.com









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