Sarepta Therapeutics to Present New Long-Term and Safety Data Across Gene Therapy and Exon-Skipping Programs at 2026 Muscular Dystrophy Association Clinical & Scientific Congress

Key Terms

dystrophin medical

Dystrophin is a large protein that acts like a structural support beam inside muscle cells, helping them withstand the stress of repeated use. It matters to investors because loss or shortage of dystrophin causes serious muscle-wasting diseases, so companies developing tests or treatments that restore or replace this protein can change patient outcomes and create significant commercial and regulatory value — but they also face scientific and approval risks.

exon skipping medical

A laboratory method that alters how a cell reads a gene so it ignores (or “skips”) a faulty segment, allowing the rest of the gene to produce a shorter but working version of a protein. Think of it like skipping a damaged chapter in a manual so the appliance can still function. Investors care because exon skipping is a targeted drug approach that can create new therapies, influence clinical trial outcomes, regulatory decisions, and future revenue potential.

phase 3 medical

Phase 3 is the late-stage clinical testing step for a new drug or medical treatment, where the product is given to large groups of patients to confirm effectiveness, monitor side effects, and compare it to standard care. Successful Phase 3 results are often the final scientific hurdle before regulators decide on approval and market launch—like passing a final exam before graduation—and can sharply change a company's valuation and future revenue prospects.

phase 4 medical

Phase 4 is the stage after a drug or vaccine has been approved and is sold to the public, where regulators and companies keep watching how it performs in the real world to detect rare side effects, long‑term effects, or differences in effectiveness across different groups. Think of it as ongoing quality control for a product already on shelves; results can prompt label changes, safety warnings, sales impacts or recalls, all of which matter to investors evaluating risk and future revenue.

observational study medical

An observational study is a type of research where investigators watch and record what happens to people who are already using a treatment or experiencing a condition, without assigning who gets what. Think of it like monitoring shoppers in a store rather than giving some a special product and others not. For investors, these studies provide real‑world evidence about safety, effectiveness and market use that can influence regulatory decisions, sales forecasts and perceived risk.

pharmacokinetics medical

Pharmacokinetics is the study of how a substance, such as a drug or chemical, moves through and is processed by the body over time. It tracks how it is absorbed, distributed, broken down, and eventually eliminated. For investors, understanding pharmacokinetics helps gauge the effectiveness, safety, and potential risks of new medications or treatments, which can influence a company’s success and valuation in the healthcare industry.

loss of ambulation medical

Loss of ambulation is the partial or complete inability to walk or move independently without assistance or devices. It matters to investors because it is a clear, measurable outcome used in drug and device trials, influences regulatory approval and reimbursement decisions, and shapes the size and urgency of markets for treatments — like a key milestone that signals a major change in patients’ daily needs and healthcare costs.

real-world medical

Real-world describes information or outcomes gathered outside controlled experiments or trials, coming from everyday settings like routine clinical care, customer behavior, or actual market use. Investors care because it shows how a product, treatment, or strategy performs in normal conditions — like a car test driven on real roads instead of a closed track — helping estimate actual demand, risks, long-term value, and regulatory or adoption hurdles.

• Several abstracts, including a late-breaking podium presentation and posters, bring forward accumulating long-term efficacy, safety and caregiver-reported insights that deepen understanding of dystrophin restoration and its impact in Duchenne

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, will present at the Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, taking place March 8 - 11, 2026, in Orlando, Florida.

At MDA 2026, Sarepta will present new and ongoing evidence across its rare neuromuscular portfolio, including commercially available gene therapy and exon-skipping treatments in Duchenne muscular dystrophy. Presentations include a late-breaking oral presentation on delandistrogene moxeparvovec gene therapy from the Phase 3 EMBARK study (Part 1) up to three years post-infusion compared with a matched external control. Another abstract will feature caregiver-reported impressions from the Phase 3 EMBARK study through two years of follow-up, offering complementary perspectives on treatment impact beyond clinician-reported and performance-based outcomes. Additionally, a safety analysis of several delandistrogene moxeparvovec clinical studies with up to 7.5 years of patient follow-up will be presented; pooled data will include treatment-related adverse events that most commonly occurred within the first 60 days post-infusion.

Sarepta will also present data from across its exon skipping franchise, including Phase 3 results from ESSENCE for golodirsen and casimersen. Also, a new real-world analysis that explores survival in patients treated with exon skipping medicines will be presented, as well as interim real-world findings from the Phase 4 EVOLVE study describing long-term safety and loss of ambulation as observed in clinical practice.

“At MDA we’re sharing data that reinforce dystrophin restoration as a foundational therapy and its ability to slow Duchenne disease progression over time,” said Louise Rodino-Klapac, Ph.D., president of research & development and technical operations, Sarepta. “We want to bring forward a data-driven view of treatment experience in clinical and real-world settings, including longer-term functional outcomes, pooled safety learnings, and caregiver-reporter perspectives that provide complementary insights into treatment effect in a subset of patients treated in EMBARK. Our goal is to ensure clinicians and families have the information they need to make treatment decisions with confidence.”

Sarepta Podium Presentation:

480LB: Delandistrogene Moxeparvovec in Duchenne Muscular Dystrophy: EMBARK Functional Outcomes and Safety up to 3 Years Post-Infusion (late-breaker)

Poster: March 10 10:30 a.m. – 1:30 p.m. ET 4 – 4:30 p.m. ET 6 – 8 p.m. ET Oral: March 11 2:30 – 2:45 p.m. ET

Sarepta Poster Presentations (*Denotes encore presentation):

22 S: Efficacy and Safety of Golodirsen and Casimersen Compared with Placebo in Duchenne Muscular Dystrophy (ESSENCE): Phase 3 Topline Results		March 8 6 – 8 p.m. ET
25 S: 2025 Interim Analysis of EVOLVE: A Long-Term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice		March 8 6 – 8 p.m. ET
71 S: A Real-World Target Trial Emulation of Eteplirsen, Golodirsen, and Casimersen to Evaluate Survival Among Patients with Duchenne Muscular Dystrophy		March 8 6 – 8 p.m. ET
77 S: Caregiver Global Impressions of Delandistrogene Moxeparvovec in Patients with Duchenne Muscular Dystrophy: Findings from EMBARK 2-Year Follow-Up		March 8 6 – 8 p.m. ET
56 S: Model-Based Evaluation of Delandistrogene Moxeparvovec Adeno-Associated Virus Pharmacokinetics and Safety Implications*		March 8 6 – 8 p.m. ET
478LB: Pooled Safety Analysis from Phase 1 to Phase 3 Clinical Trials of Delandistrogene Moxeparvovec in Duchenne Muscular Dystrophy (late-breaker)		March 10 10:30 – 1:30 p.m. ET 4 – 4:30 p.m. ET 6 – 8 p.m. ET
272 T: Quantitation of Dystrophin Expression in Patients with Duchenne Muscular Dystrophy by Western Blot Analysis Adjusted for Muscle Content		March 10 Poster Reception: 10:30 – 1:30 p.m. ET 4 – 4:30 p.m. ET 6 – 8 p.m. ET
319 T: Expression of SGCA and Safety Following Treatment with Patidistrogene Bexoparvovec in Patients with LGMD2D/R3: Results from a Phase 1b Study		March 10 Poster Reception: 10:30 – 1:30 p.m. ET 4 – 4:30 p.m. ET 6 – 8 p.m. ET

The full MDA 2026 program is available here: https://www.mdaconference.org. Sarepta abstracts and presentations will be available on Sarepta.com in the Events & Presentations section following the MDA embargo.

About Sarepta Therapeutics
Sarepta is on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold a leadership position in Duchenne muscular dystrophy (Duchenne) and are building a robust portfolio of programs across muscle, central nervous system, and cardiac diseases. For more information, please visit www.sarepta.com or follow us on LinkedIn, X, 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 at www.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us.

Forward-Looking Statements
This press release contains forward-looking statements. Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "will," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements related to our research and development programs, clinical trials, technologies, scientific approaches, products and product candidates; and expected plans and milestones, including presenting certain data and findings at MDA.

These forward-looking statements involve risks and uncertainties, many of which are beyond Sarepta’s control. Known risk factors include, among others: success in preclinical and clinical trials, especially if based on a small patient sample, does not ensure that later clinical trials will be successful, and the results of future research may not be consistent with past positive results or may fail to meet regulatory approval requirements for the safety and efficacy of product candidates; certain programs may never advance in the clinic or may be discontinued for a number of reasons, including regulators imposing a clinical hold and us suspending or terminating clinical research or trials; we may not be able to execute on our business plans, including meeting expected or planned regulatory milestones and timelines, clinical development plans, and bringing products to markets for various reasons including possible limitations of financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, and regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover our product candidates; and those risks identified under the heading “Risk Factors” in Sarepta’s most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company which you are encouraged to review.

Any of the foregoing risks could materially and adversely affect the Company’s business, results of operations and the trading price of Sarepta’s common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof, except as required by law.

 

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Investor Contacts:

Ian Estepan, 617-274-4052

iestepan@sarepta.com

Ryan Wong, 617-800-4112

rwong@sarepta.com

Tam Thornton, 617-803-3825

tthornton@sarepta.com

Media Contacts:

Tracy Sorrentino, 617-301-8566

tsorrentino@sarepta.com

Kara Hoeger, 617-710-3898

khoeger@sarepta.com

Source: Sarepta Therapeutics, Inc.