Scholar Rock

Our BLA for our investigational SMA treatment has been accepted by the FDA with a PDUFA action date of September 30, 2026. Scholar Rock is prepared for the U.S. apitegromab launch immediately upon FDA approval, which may be granted at any time through September 30, 2026. Read more in our Q1 release here: https://bit.ly/3OROzNw

Scholar Rock is looking forward to attending the Bank of America (BofA) Securities 2026 Health Care Conference in Las Vegas, NV, on May 12. A live webcast of the event will be available within the Investors section of the Scholar Rock website. Read the full press release here: https://bit.ly/3QvAOVf

We have resubmitted our BLA to the FDA for our investigational, muscle-targeted treatment, moving closer to delivering this potential new therapy to children and adults living with SMA. Our progress reaffirms the promise of myostatin biology as we continue to prioritize scientific advancement and the needs of the SMA community. Read more in our latest press release here: https://bit.ly/4sIxX9x

We’re headed to Budapest, Hungary for the 5th SMA Europe International Scientific Congress on Spinal Muscular Atrophy. We look forward to connecting with the global multidisciplinary community of scientists, researchers, and experts to exchange insights and foster discussions on the evolving SMA landscape. In addition to data presentations, we will participate in a symposium, join in on an interactive workshop, and connect with you at our booth. For more information on the congress, visit https://lnkd.in/eGXTBYrS. 

Scholar Rock is looking forward to participating in fireside chats at both Leerink Partners 2026 Global Healthcare Conference (Mar 10) and Barclays 28th Annual Global Healthcare Conference (Mar 12). Read the full press release: https://bit.ly/4rgR8FP 

On March 3 we reported Q4 and full year 2025 financial results and recent business updates. 2026 is poised to be a transformative year for Scholar Rock as we prepare to usher in the next phase of innovation for people living with SMA. Read the full earnings release: https://bit.ly/3Pprz8a

We’re attending the 2026 Muscular Dystrophy Association Clinical & Scientific Conference! Find our data presentations: - Alicia Henriquez, MD, Director of Neuromuscular Gene Therapies, Seattle Children's, will speak on Clinical Meaningfulness and the patient voice in SMA on Monday, March 9 from 6:00pm to 8:00pm. - Laurent SERVAIS, MD, PhD, Professor of Paediatric Neuromuscular Diseases, MDUK Oxford Neuromuscular Centre, will present post hoc analyses from the Phase 3 SAPPHIRE trial for SMA on Monday, March 9 from 6:00pm to 8:00pm. - John Staropoli, MD, PhD, Scholar Rock, will present on trial design for the Phase 2 FORGE study for FSHD on Tuesday, March 10 from 6:00pm to 8:00pm. - Adam Fogel, PhD, Senior Director, Discovery Biology, Scholar Rock, will present preclinical data of SRK-015 in FSHD models on Tuesday, March 10 from 6:00pm to 8:00pm. In addition to our poster presentations, we invite you to visit us at Booth 413! We’re also excited to take part in a symposium and support a trainee event for the rising stars in the neuromuscular and SMA space. For more details or to register, visit www.mdaconference.org.

On #RareDiseaseDay we are proud to reaffirm our commitment to working alongside the rare disease community as we pursue our mission to discover, develop, and deliver therapies to improve people's lives.

Emily, a parent and caregiver in the #SMA community shares a powerful reminder that speaks to the heart of our #SeeMyAbilities campaign: we must look beyond a diagnosis and see the individual. Because laughter, personality, and joy aren’t defined by physical abilities — they’re expressions of who we are.

1 in 50. That’s the number of people who are genetic carriers for spinal muscular atrophy. The 𝘚𝘔𝘕1 gene, or Survival Motor Neuron 1 gene, is responsible for creating the protein that enables muscle movements. While most people have two functioning copies of the 𝘚𝘔𝘕1 gene, having just one makes you a carrier for #SMA. Discover more about SMA and genetics, carrier testing, and survival motor neuron genes, visit https://lnkd.in/eQNGCYrE.