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
Scholar Rock is the first company to show clinical proof of concept for a muscle-targeted treatment in spinal muscular atrophy (SMA). Patients and caregivers have said they want new therapies to address unmet needs including increasing muscle strength, stabilizing or gaining motor function, reducing fatigue and improving daily activities.
Apitegromab is our investigational medicine with the potential to transform the standard of care for people living with SMA. We reported positive topline results of our Phase 3 pivotal SAPPHIRE clinical trial in Q4 2024. Pending regulatory approval, we anticipate launching apitegromab in the United States and Europe in 2025, and then exploring subsequent access in other countries.
Existing therapies for SMA address motor neuron degeneration, but they do not directly address progressive muscle atrophy and weakness. Apitegromab is designed to be used with background SMN treatments to potentially sustain and improve motor function.
Learn more about apitegromab’s progress through our ongoing clinical trials
Apitegromab aims to help people with SMA by selectively inhibiting myostatin, a protein that regulates muscle growth.
“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.
September 30, 2024
Caregiver perspectives on creating possibilities for loved ones living with SMA
Read More about Caregiver perspectives on creating possibilities for loved ones living with SMA
October 16, 2024
What do new possibilities mean to those living with SMA?
Read More about What do new possibilities mean to those living with SMA?
Patient Resources
We are proud to work with the SMA community to learn from their everyday experiences and inform what we do every day.
