Sarepta Therapeutics

As we head into #RareDiseaseWeek, we’re spotlighting members who show how collaboration across the rare disease community drives real progress. For Sarepta Therapeutics, #OneRareVoice reflects the power of bringing the entire rare disease community together—patients, families, clinicians, advocates, regulators, and industry—around a shared purpose. Rare disease innovation depends on thoughtful partnership across this ecosystem. It’s this shared commitment that strengthens our ability to move life-changing therapies forward for patients.

Today, we celebrate International Day of Women and Girls in Science by highlighting some of the women at Sarepta who are making a difference through their passion for science. They share what first sparked their interest in STEM, the mentors who shaped their paths and what continues to inspire them in their work today—along with advice for the next generation of women in science. Click or swipe to explore their stories. #WomenInScience

Calling all students! Sarepta is now accepting applications for our Summer 2026 Internship Program. We offer paid internships for college and graduate students across STEM and non-science fields. Interns gain hands-on experience with our business, research and culture through real projects and a seminar series showcasing key biotech functions. Explore internship opportunities and apply here: https://bit.ly/45DSylQ (search by Job Type to see all intern positions)

Today we announced positive topline three-year EMBARK results showing that our approved gene therapy significantly slows disease progression on key functional measures in ambulatory Duchenne patients. Read the release at Sarepta.com.

Today at #JPM26, CEO Doug Ingram discussed our preliminary Q4 and full-year 2025 performance and shared an update on corporate developments. Read more: https://bit.ly/49tr2bO

We just announced our preliminary fourth quarter and full-year 2025 net product revenues. Read more: https://bit.ly/49tr2bO

We are excited to connect with you in 2026! Here are just a few of the in-person events our Patient Advocacy team will be attending in the early part of the year. We look forward to seeing you. Email Advocacy@Sarepta.com if you have questions.

Today, Sarepta announced the submission of its CTA for Study SRP-1005-101 to the New Zealand Medicines and Medical Devices Safety Authority. SRP-1005 is an investigational small interfering RNA (siRNA) therapeutic for the treatment of Huntington’s Disease. Read the release: https://bit.ly/45lKgz5

An important milestone: Duchenne muscular dystrophy was added to the Recommended Uniform Screening Panel (RUSP), a critical step toward ensuring early detection and intervention. The RUSP, set by the U.S. Secretary of Health and Human Services, guides states in determining newborn screening panels. While each state sets their own panel, many follow this federal recommendation. We applaud the Department of Health and Human Services for leadership in accelerating access to Duchenne newborn screening, and we recognize Parent Project Muscular Dystrophy (PPMD), Muscular Dystrophy Association (MDA), and the patient advocates and families whose voices drove this progress. Time is muscle, and your advocacy made this possible. Ten states have already passed legislation or approved the addition of Duchenne to their state newborn screening panels, and we look forward to more states now moving quickly to add Duchenne to their panels. Because every baby deserves the chance for early diagnosis and intervention. Read more: https://bit.ly/3XZD1J2 

Our CEO, Doug Ingram, appeared on 60 MINUTES last evening to discuss gene therapies in treating rare diseases, such as Duchenne muscular dystrophy. In his conversation with Scott Pelley, recorded in 2024, Doug talked about the promise and complex work of engineering one-time treatments for rare diseases, and he shared his perspective on what it will take to build on today’s scientific breakthroughs to accelerate innovation and expand what’s possible for many more people living with genetic conditions. Watch here: https://bit.ly/4oTg70M