Small interfering RNA (siRNA) is a short strand of genetic material that binds to and destroys the messenger RNA that carries instructions for making a specific protein, effectively switching that gene off. Investors care because siRNA is a platform for precise medicines: successful trials or approvals can create high-value drugs, while delivery challenges, manufacturing complexity, patent positions and regulatory risk can sharply affect a biotech company's prospects.
phase 1/2medical
Phase 1/2 is a combined early-stage clinical trial that first tests a new drug or treatment for safety and the right dose, then quickly expands to check if it shows any signs of working in patients. For investors, results from a Phase 1/2 study offer an early read on both risk and potential reward—like a prototype test that both confirms a product won’t harm users and suggests whether it could sell—helping guide valuation and development decisions.
facioscapulohumeral muscular dystrophy type 1medical
A hereditary muscle disorder caused by a specific genetic change that leads to progressive weakness, typically affecting facial muscles, shoulder blades and upper arms. For investors, it matters because the clear genetic cause and measurable progression make it a well-defined target for drug development, clinical trials and potential therapies; understanding the size of the patient group and how fast symptoms progress helps gauge market opportunity and regulatory risk.
myotonic dystrophy type 1medical
A genetic, progressive disorder that causes muscle stiffness, weakness and a range of problems in other body systems (such as breathing, heart rhythm, vision and cognition); symptoms can vary widely and often worsen over time. For investors, it matters because its chronic, multisystem nature shapes the size of the potential patient population, the complexity and cost of clinical trials, regulatory requirements, and long-term demand for any effective treatments — think of it as a condition that requires multiple pieces to be fixed, not just a single part.
CAMBRIDGE, Mass.--(BUSINESS WIRE)--
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that on Wed., March 25, 2026, at 8:30 am Eastern Time, the Company will host a webcast and conference call to present the early clinical results from the Phase 1/2 ascending dose studies of SRP-1001 for facioscapulohumeral muscular dystrophy type 1 (FSHD1) and SRP-1003 for myotonic dystrophy type 1 (DM1).
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 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.
