Scholar Rock

Real people. Real voices. Real Impact. The See My Abilities campaign is an opportunity to listen to and amplify the voices of the #SMA community. We’re inviting those living with the condition to reclaim the acronym beyond medical diagnosis, saying “you get to declare what SMA means to you.”

Muscle function starts with a motor unit: one motor neuron and the muscle fibers it controls. The brain sends a signal, the motor neuron fires, and muscle fibers contract to enable motion. In spinal muscular atrophy, treatments that increase SMN protein help prevent or slow the degeneration of motor neurons. But that’s only half the puzzle. That's why we're exploring directly targeting the muscle itself. Learn more about our science here: https://lnkd.in/ewY-BVpM

Our team is composed of seasoned biopharma leaders, and that includes our Vice President, Head of US Medical Affairs, Erik DeBoer, PhD. With an extensive background in medical affairs, Erik brings deep knowledge and expertise in neuroscience and neuropharmacology to our world-class team of scientists, collaborators, and big-picture thinkers. As we push boundaries with our unique approach to treating disease, we are grateful for the passion and commitment that fuels our work.

This year marks 135 years since spinal muscular atrophy was first described. In that time, we’ve seen extraordinary progress, turning what was once only a diagnosis into real options, real hope, and real change for the SMA community. But there is still more to do. We remain steadfast in our commitment to a better future for individuals living with SMA.

Scholar Rock CEO, David Hallal, presented our strategic priorities for 2026 at the J.P. Morgan Annual Healthcare Conference. Hallal echoed our strong momentum for the year ahead as we focus on building our world-class team, engaging with the SMA community, and establishing reliable access to our investigational, muscle-targeted treatment following approval. Explore our corporate update: https://lnkd.in/eZRnsZzE

Misconceptions about #SMA are common, which is why individuals living with SMA, like Maddy, are committed to changing the narrative. It’s time to use our voices to help those outside the SMA community understand that a diagnosis doesn’t define a person, and that their lives, goals, and identities are not so different from anyone else’s.

Navigating his diagnosis hasn’t been easy, but the support from his parents has been a source of strength. Ben, an individual living with #SMA, is grateful for his family who has always encouraged him to pursue big ideas, embrace new opportunities, and live fully. A diagnosis doesn’t hold him back, and the love and encouragement from those around him has been a powerful reminder of that.

Today we announced that Scholar Rock's management will present at the 44th Annual J.P. Morgan Healthcare Conference on Monday, January 12. CEO, David Hallal will discuss our 2026 strategic priorities. Read the full press release: https://lnkd.in/eJVxxNu6

As we welcome 2026, we’re preparing for a landmark year at Scholar Rock. We’re not only celebrating our 15th anniversary, but we’re also working toward regulatory approvals in the U.S. and EU for our investigational, muscle-targeted treatment for children and adults living with SMA. Here’s to more possibilities in a new year!

One hundred years ago, the concept of the motor unit transformed our understanding of movement. By defining the motor unit as a single motor neuron and its associated muscle fibers, Edward Liddell and Charles Sherrington revealed that muscle function is driven primarily by neuromuscular signaling—not muscle mass alone. Today, while current therapies for the treatment of #SMA focus on the neuronal component of the motor unit, our investigational approach targets the muscle fibers themselves. A century after the foundational discovery that reshaped neuroscience and muscle biology, we look ahead to the next 100 years—driven by continued innovation to better serve people living with rare, severe neuromuscular diseases.