Rare disease drugmaker · based in Cambridge, United States · ticker SRRK · scholarrock.com
Scholar Rock works on Facioscapulohumeral Muscular Dystrophy and Spinal Muscular Atrophy, 2 of the rare diseases we track. It has 3 active clinical trials, and 2 are looking for volunteers now. It also has 1 FDA-approved rare disease drug.
Scholar Rock is a Cambridge, Massachusetts biotech that spent roughly a decade chasing a target the rest of the industry had largely given up on, and in September 2026 became the first company to make it work. The company was built on research from Harvard Medical School and Boston Children's Hospital into the TGF-beta superfamily, a group of proteins that regulate how tissue grows and repairs itself.
Scholar Rock pipeline
Each row lists the diseases Scholar Rock is studying at that stage, from the ones closest to approval down to the earliest work.
These are Scholar Rock trials for the rare diseases we track, from ClinicalTrials.gov. Statuses there can lag behind company news, so confirm with the study team before you plan around one.
2 trials looking for volunteers now
Looking for volunteersPhase 2Recently updatedNCT07047144
A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy
This double-blind, Phase 2, multiple-dose study will be conducted to evaluate the PK/PD, efficacy, safety, and tolerability of apitegromab in subjects \<2 years old with 5q autosomal recessive SMA who have delayed motor milestones for their age attributed to SMA at the discretion of the Investigator or a Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) score \<55.
The ONYX study is an Open-Label, Multicenter, Extension study that will evaluate the long-term safety and efficacy of Apitegromab in Patients with Type 2 and Type 3 SMA who have completed TOPAZ or SAPPHIRE.
Its central insight is a matter of timing. Most of these growth factors are produced in an inactive precursor form and have to be switched on before they do anything, and Scholar Rock designs antibodies that grab the precursor rather than the finished protein.
Targeting the switch instead of the signal is what allows its drugs to hit one growth factor without disturbing its close relatives, which is where earlier attempts in this field ran aground. The company trades on Nasdaq under SRRK.
The proof is ISEMBYLD (apitegromab), approved by the FDA on September 11, 2026 for spinal muscular atrophy in adults and children 2 and older who are already receiving an SMN2-targeted treatment. It is the first muscle-targeted treatment for SMA and the company's first approved product.
Every SMA drug before it, Spinraza, Zolgensma and Evrysdi, worked on the genetic root of the disease by protecting motor neurons, and none of them addressed muscle that had already wasted away. Apitegromab blocks myostatin, the body's natural brake on muscle growth, which is why it is given alongside an existing SMN therapy rather than in place of one.
Keep reading about Scholar Rock
In the Phase 3 SAPPHIRE trial, patients aged 2 to 12 on the recommended dose gained motor function over a year while those on an SMN drug alone continued to lose it. Drug companies have been trying to drug myostatin since the early 2000s across muscular dystrophy, cachexia and age-related muscle loss, and the record until this approval was a long run of failures.
The same platform is aimed at several other areas. Apitegromab is also being studied in cardiometabolic disease, where preserving muscle during rapid weight loss on GLP-1 drugs is an open question, and the pipeline includes programs in immuno-oncology targeting TGF-beta 1 in solid tumors, plus earlier work in fibrosis and anemia.
For SMA families, the practical significance is narrower and clearer: after a decade in which every new option worked the same way, there is now a second mechanism to add on top.
Where Scholar Rock trials take place
The trials above run at 73 study sites in 9 countries. Tap a country to see its states, cities and hospitals.
United States
38▼
Italy
8▼
Belgium
6▼
France
4▼
Spain
4▼
United Kingdom
4▼
Germany
4▼
Poland
3▼
Netherlands
2▼
Rare diseases Scholar Rock works on
Each disease page lists every open trial, not only Scholar Rock's, plus approved treatments.
Facioscapulohumeral Muscular Dystrophy (FSHD) is an inherited muscular dystrophy characterized by progressive weakness of the facial, shoulder, and upper arm muscles.
How common: About 1 in 20,000 people (MedlinePlus Genetics); one of the most common hereditary muscular dystrophies
Spinal muscular atrophy is an autosomal recessivegenetic disorder affecting motor neurons in the spinal cord, causing progressive muscle weakness and atrophy.
How common: About 1 in 10,000 births; about 1 in 50 people of European or Asian ancestry are carriers of 1 copy of the gene change
Where to get support
Patient groups can explain a diagnosis, point you to specialists and tell you about trials before they open. Scholar Rock's own site is scholarrock.com, and our help paying for treatment page lists drugmaker programs and charity funds.
1 shared disease, including Facioscapulohumeral Muscular Dystrophy
Questions about Scholar Rock
Scholar Rock is a Cambridge, Massachusetts biotech that spent roughly a decade chasing a target the rest of the industry had largely given up on, and in September 2026 became the first company to make it work. The company was built on research from Harvard Medical School and Boston Children's Hospital into the TGF-beta superfamily, a group of proteins that regulate how tissue grows and repairs itself. Its central insight is a matter of timing. Most of these growth factors are produced in an inactive precursor form and have to be switched on before they do anything, and Scholar Rock designs antibodies that grab the precursor rather than the finished protein. Targeting the switch instead of the signal is what allows its drugs to hit one growth factor without disturbing its close relatives, which is where earlier attempts in this field ran aground. The company trades on Nasdaq under SRRK.
The proof is ISEMBYLD (apitegromab), approved by the FDA on September 11, 2026 for spinal muscular atrophy in adults and children 2 and older who are already receiving an SMN2-targeted treatment. It is the first muscle-targeted treatment for SMA and the company's first approved product. Every SMA drug before it, Spinraza, Zolgensma and Evrysdi, worked on the genetic root of the disease by protecting motor neurons, and none of them addressed muscle that had already wasted away. Apitegromab blocks myostatin, the body's natural brake on muscle growth, which is why it is given alongside an existing SMN therapy rather than in place of one. In the Phase 3 SAPPHIRE trial, patients aged 2 to 12 on the recommended dose gained motor function over a year while those on an SMN drug alone continued to lose it. Drug companies have been trying to drug myostatin since the early 2000s across muscular dystrophy, cachexia and age-related muscle loss, and the record until this approval was a long run of failures.
The same platform is aimed at several other areas. Apitegromab is also being studied in cardiometabolic disease, where preserving muscle during rapid weight loss on GLP-1 drugs is an open question, and the pipeline includes programs in immuno-oncology targeting TGF-beta 1 in solid tumors, plus earlier work in fibrosis and anemia. For SMA families, the practical significance is narrower and clearer: after a decade in which every new option worked the same way, there is now a second mechanism to add on top.
Scholar Rock has active programs across 2 rare diseases: Facioscapulohumeral Muscular Dystrophy, Spinal Muscular Atrophy. Each disease page on Trial Friend shows the latest recruiting trials, FDA-approved treatments, and patient resources.
Yes. Scholar Rock is a rare disease specialist headquartered in Cambridge, United States. The company focuses primarily on developing treatments for rare and orphan diseases, with programs targeting Facioscapulohumeral Muscular Dystrophy, Spinal Muscular Atrophy.
The clinical trials section on this page shows active and recruiting rare disease trials sponsored by Scholar Rock, pulled live from ClinicalTrials.gov. Each trial card includes the study phase, recruitment status, conditions being studied, number of trial sites, and a direct link to the full study record. You can also explore individual disease pages to see trials from all sponsors for a specific condition.
To explore enrollment, find a recruiting trial in the clinical trials list on this page and click "View full study" to see the full ClinicalTrials.gov record, which includes eligibility criteria, study locations, and contact information for each trial site. Your doctor can also help determine if a specific trial is appropriate for your condition. Not all trials are open at every location, so check the site list for one near you.
Scholar Rock has 1 FDA-approved drug with a rare disease use that we track, as of November 2026.
Source: the FDA's openFDA label database, matched to the rare diseases on Trial Friend; the list updates automatically and can include older products.
Yes, for 1 of its FDA-approved rare disease medicine that we track, each checked on Scholar Rock's own website.
Isembyld: Scholar Rock Supports, 1-833-777-5444. Offers copay help with private insurance, infusion cost help and insurance help. Checked September 24, 2026.
Medicare and Medicaid: drugmaker copay cards are limited to private insurance, so people with government coverage usually look to a free medicine program or a charity fund, both on the help paying page.
Eligibility is decided by each program, so call the number listed before a prescription is filled.