Sarepta Therapeutics to Report 3-Year Topline Data from EMBARK Study of ELEVIDYS Gene Therapy in Ambulatory Individuals with Duchenne Muscular Dystrophy
Sarepta Therapeutics to Report 3-Year Topline Data from EMBARK Study of ELEVIDYS Gene Therapy in Ambulatory Individuals with Duchenne Muscular Dystrophy
A rare, inherited condition that progressively weakens muscles, Duchenne muscular dystrophy causes the body’s muscle fibers to break down over time, often leading to severe disability. For investors, it matters because the small, well-defined patient population, high unmet medical need and complex regulatory and pricing dynamics mean successes or failures in clinical trials, approvals, or therapies can have outsized effects on a company’s valuation and future revenue prospects.
gene therapymedical
Gene therapy is a medical technique that involves altering or replacing faulty genes in a person's cells to treat or prevent disease. It is considered a promising area of innovation because it has the potential to provide long-term or even permanent solutions to genetic conditions. For investors, advancements in gene therapy can signal opportunities in biotech companies and emerging treatments with significant growth potential.
phase 3medical
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.
randomized placebo-controlledmedical
A randomized placebo-controlled study is a clinical test where people are assigned by chance—like flipping a coin—to receive either the active treatment or a harmless look‑alike (placebo), and often neither participants nor staff know who got which. For investors, these trials are the clearest way to show whether a drug’s effects are real rather than due to luck or expectations; clean, positive results lower development risk and increase the chance of approval and commercial success.
CAMBRIDGE, Mass.--(BUSINESS WIRE)--
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that on Monday, Jan. 26, 2026, at 8:30 am Eastern Time, the Company will host a webcast and conference call to present 3-year topline functional results from patients treated in Part 1 of EMBARK (Study 9001-301), the global, randomized placebo-controlled Phase 3 study evaluating ELEVIDYS (delandistrogene moxeparvovec-rokl) in ambulatory individuals with Duchenne muscular dystrophy who were aged four to seven at time of treatment.
The event will be webcast live under the investor relations section of Sarepta's website at https://investorrelations.sarepta.com/events-presentations and following the event a replay will be archived there for one year. Interested parties participating by phone will need to register using this online form. After registering for dial-in details, all phone participants will receive an auto-generated e-mail containing a link to the dial-in number along with a personal PIN number to use to access the event by phone.
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 leadership positions in Duchenne muscular dystrophy (Duchenne) and are building a robust portfolio of programs across muscle, central nervous system, and cardiac diseases.
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.
