GlobeNewswire Inc | Sep 14, 2020 08:30AM EDT

September 14, 2020

--Webcast conference call to be held on Monday, Sept. 28, 2020 at 8:30 a.m. Eastern Time --

-- Additional poster presentations at WMS will highlight data from Sareptas RNA and gene therapy programs --

CAMBRIDGE, Mass., Sept. 14, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that new data from its most advanced gene therapy programs will be presented at the WMS25 Virtual Congress, the 25th International Annual Congress of the World Muscle Society, being held Sept. 28 Oct. 2.

Sarepta will host a webcast and conference call on Monday, Sept. 28, 2020 at 8:30 a.m. ET, to discuss the results, which include two-year functional data from Study 101 of SRP-9001 for Duchenne muscular dystrophy and 18-month functional results from Cohort 1 in the study of SRP-9003 for Limb-girdle muscular dystrophy Type 2E.

This will be webcast live under the investor relations section of Sarepta's website at https://investorrelations.sarepta.com/events-presentationsand will be archived there following the call for one year. Please connect to Sarepta's website several minutes prior to the start of the broadcast to ensure adequate time for any software download that may be necessary. The conference call may be accessed by dialing (844) 534-7313 for domestic callers and (574) 990-1451 for international callers. The passcode for the call is 6793650. Please specify to the operator that you would like to join the "Long-term Functional Data from Sareptas Gene Therapy Programs call.

In total, Sarepta will present 16 abstracts at this years meeting. All posters will be available on-demand throughout the Congress beginning on Monday, Sept. 28 at 7:00 a.m. EST. The full WMS25 Virtual Congress program is available here: https://www.wms2020.com/programme/.

Gene Therapy:

TITLE PROGRAM POSTER #Treatment of Aged Mice and Long-term Durability of SRP-9003,AAV-Mediated Gene Therapy in Two Mouse Models of Limb Girdle SRP-9004 P.137Muscular DystrophyExpression-Functional Correlation and Validation of a SRP-9003 P.139Surrogate Marker for DAPC Restoration in LGMD2E Mouse ModelSystemic Gene Transfer with rAAVrh74.MHCK7.SGCB Increased-sarcoglycan Expression in Patients with Limb Girdle SRP-9003 P.140Muscular Dystrophy Type 2EEvaluation of the Lipid-Binding and Stability Properties of SRP-9001 P.206Recombinant Dystrophin Spectrin-Like Repeat ConstructsSystemic Gene Transfer with rAAVrh74.MHCK7.micro-dystrophin in SRP-9001 P.280Patients with Duchenne Muscular DystrophySystemic Dose-Finding Study with AAV-Mediated -Sarcoglycan SRP-9005 P.138Gene Therapy for Treatment of Muscle Deficits in LGMD2C Mice

RNA Platform:

TITLE PROGRAM POSTER #Long-term Safety and Efficacy of Golodirsen in Male Patients Golodirsen P.283with Duchenne Muscular Dystrophy Amenable to Exon 53 SkippingCasimersen Treatment in Eligible Patients with DuchenneMuscular Dystrophy: Safety, Tolerability, and Casimersen P.288Pharmacokinetics Over 144 Weeks of TreatmentOpen-Label Evaluation of Eteplirsen in Patients With DuchenneMuscular Dystrophy Amenable to Exon 51 Skipping: PROMOVI Eteplirsen P.289TrialDelay in Duchenne Muscular Dystrophy Progression withEteplirsen: Attenuation of Pulmonary Decline and Projected Eteplirsen P.290Freedom from Continuous VentilationReal-world Evidence of Eteplirsen Treatment Effects onDuchenne Muscular Dystrophy Related Health Outcomes Using Eteplirsen P.291Claims Data in the United States

Natural history and other presentations:

TITLE POSTER #Development of Cardiomyopathy, Respiratory Insufficiency and Loss of P.41Ambulation in Becker Muscular Dystrophy: A Systematic Literature ReviewDisease Attributes Most Important from a Societal Perspective: A Case P.56Study Involving Duchenne Muscular DystrophyIdentification of Disease Progression Stages in Patients with Duchenne P.119Muscular Dystrophy Using Administrative Claims Data in the United StatesThe Age at Loss of Ambulation Among Patients with Limb-Girdle Muscular P.142Dystrophy (LGMD) Subtype 2: A Systematic ReviewRasch Analysis of the Pediatric Quality of Life Inventory 4.0 Generic P.345Core Scales Administered to Patients with Duchenne Muscular Dystrophy

Presentations will be archived under the events and presentations section of the Sarepta Therapeutics website at www.sarepta.comforone year following their presentation at WMS25.

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