Sarepta Therapeutics

Are you familiar with the North Star Ambulatory Assessment (NSAA)? Sarepta’s Senior Director of Global Medical Affairs, Rachel Salazar, PT, DPT, PhD, offers a clear explanation of the clinical outcome measurement tool designed specifically for people living with #Duchenne. By rating 17 different motor skill activities, doctors can keep an eye on how a child’s functional performance changes over time due to disease progression. As Rachel shares, kids typically reach their peak score and performance around 6 years of age before entering a decline in their functional skills. “If we look at 9-year-old with Duchenne, no change or even a one-point decline is rather unexpected because this child would be typically in the steep decline phase of his disease. And if the North Star holds steady rather than declining each year, then he may be retaining abilities that he was expected to lose due to his disease.” Watch the video to learn more and view Sarepta’s fact sheet outlining the complete list of 17 measured skills. https://bit.ly/3YjVFuG #raredisease

Did you see the news that we announced the recipients of the 2024 Limb-girdle muscular dystrophy (LGMD) Grant Award Program? Created by Sarepta to help accelerate the LGMD diagnostic journey and enhance participation in genetic testing programs, this year we are awarding more than $100,000 in grant funding. Learn more about the program and read about the work of a past LGMD Grant Award Program recipient in this Newsroom article: https://bit.ly/3GKFP3D

We’re leaving #WMS2024 feeling energized by our collective progress in addressing neuromuscular disorders. Connecting with attendees – from industry peers to clinicians, scientists and caregivers – is always mission-affirming. See you next year!

We look forward to convening with industry peers this week at the 29th Annual Congress of the World Muscle Society in Prague from October 8-12. Stop by Booth #1 to say hello! #WMS2024 #Duchenne #LGMD

We’re lighting up green for limb-girdle muscular dystrophy! Today, our Genetic Therapies Center of Excellence in Columbus, Ohio is glowing with green lights for LGMD Awareness Day. We’re shining bright to show our support for the LGMD community. #LGMD2024

Today is Limb-Girdle Muscular Dystrophy Awareness Day, and we’re wearing lime green to show our support and raise awareness.💚 This year, we’re more committed than ever to supporting the LGMD community and advancing research on these rare neuromuscular diseases. Join us in going green and showing your support. #LGMDAwarenessDay #LimeGreen4LGMD

We are excited to announce the recipients of the 2024 Limb-girdle muscular dystrophy (LGMD) Grant Award Program! Created by Sarepta to help accelerate the LGMD diagnostic journey and enhance participation in genetic testing programs, this year we are awarding more than $100,000 in grant funding. Open to non-governmental and patient advocacy organizations worldwide, this year’s recipients are based in Argentina and Italy. Check out our news release for more details about the program and the recipients. https://bit.ly/4dxyKlc #LgmdDay2024

Duchenne affects the entire family and siblings are an integral part of the #Duchenne community. For that reason, we extend our annual Route 79 Scholarship program to the siblings of individuals with Duchenne. We’re happy to acknowledge Mateo, who is studying Business at the University of North Carolina Chapel Hill. Learn more about the Route 79 Scholarship program and this year’s recipients: https://bit.ly/3TmweXI

We are excited to share that Sarepta was honored with the Transformational Therapy Award at the #BiotechWeekBoston Awards this week. The award, given for our newest therapy for Duchenne muscular dystrophy, recognizes innovation in the rare disease space and efforts to bring much-needed therapies to patients. Thank you, #BWBAwards!

We're continuing our spotlighting of recipients of Sarepta’s 2024-2025 Route 79 Scholarship and are happy to introduce you to Bradon. Bradon is pursuing his passion for entomology at the University of Florida. Every year, through the Route 79 Scholarship program, we acknowledge and support individuals with Duchenne who are taking charge and mapping out their future via educational pursuits. Learn more about the Route 79 Scholarship program and meet the additional recipients: https://bit.ly/3TmweXI