Patients & Families
Creating new possibilities for patients
Our work is grounded in a commitment to address unmet needs that can make a meaningful difference for people living with serious diseases. From developing new therapies to engaging with patient communities, everything we do focuses on what matters most to patients and their families.
Focus on the Patient
We champion what matters to patients and their families.
Spinal Muscular Atrophy
Patients and caregivers have said they want new therapies to address unmet needs including increasing muscle strength, stabilizing or gaining motor function. Spinal muscular atrophy (SMA) is a rare, severe, genetic neuromuscular disease. The disease is characterized by the irreversible loss of motor neurons, atrophy of the voluntary muscles of the limbs and trunk, and progressive muscle wasting that causes continuous motor function decline throughout life and can diminish the independence of both children and adults.
- Patients and caregivers have said they want new therapies to address unmet needs including increasing muscle strength, stabilizing or gaining motor function.
- Other treatment approaches were designed to address the motor neuron component of the disease and do not directly address the muscle. Despite advances in SMN-targeted treatments, challenges remain in preserving motor function over time, demonstrating how muscle remains at the center of unresolved needs in SMA.
We are committed to advancing fundamentally different treatment approaches with the potential to change the disease trajectory for people living with SMA around the world.
“We all work hard at Scholar Rock. However, no one works harder than the participants in our studies. It is them and their families’ resilience and strength that continues to motivate me.”
Erin Barnobi
Clinical Operations
Clinical Trials
We focus on the discovery and development of safe, effective medicines.
How Clinical Trials Work
A clinical trial is part of the research process involving volunteers (also called participants) that follows strict guidelines to test whether an investigational medicine is safe and effective for humans. Results from these trials are then submitted to regulatory agencies like the U.S. Food & Drug Administration (FDA) to gain approval of the investigational medicine.
Clinical trials are usually conducted in a series of rounds (also called phases), each of which is designed to answer specific scientific questions:
Phase 1:
Testing in healthy volunteers.
Phase 1 tests for the safest investigational medicine dose level in a small number of healthy volunteers. If an investigational medicine is found reasonably safe during Phase 1 trials, it will move on to Phase 2.
Phase 2:
Testing for safety and effectiveness in patients with the disease or condition being studied.
Phase 2 involves another round of tests to measure potential beneficial results and potential side effects of the investigational medicine.
Phase 3:
Broadening the test group and raising the standard for success.
Phase 3 requires the largest number of participants. An investigational medicine that successfully passes Phase 3 can be submitted to regulatory agencies for approval to be marketed to the public.
Phase 4:
Post-approval monitoring.
Phase 4 refers to the period after regulatory approval, when patients can be prescribed a medicine without having to join a clinical trial. After approval, the medicine is studied for safety surveillance, to understand the medicine’s real-world use, and outcomes over longer periods of time.
October 13, 2025
Update on Third Party Manufacturing Site
September 22, 2025
Believe in What is Possible
Patient Resources
We are proud to work with the SMA community to learn from their everyday experiences and inform what we do every day.
