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Spinal Muscular Atrophy: full-pipeline trial registry
6 of 23 indexed Spinal Muscular Atrophy trials are currently recruiting.
Corpus placement: #724 of 28,707 conditions by registered trial count.
23 US clinical trials · 6 currently recruiting · #724 of 28,707 conditions by registered trial count
Spinal Muscular Atrophy: 23 US clinical trials tracked, 6 recruiting.
Spinal Muscular Atrophy is the subject of 23 registered US clinical trials on ClinicalTrials.gov, 6 of them currently open to new participants. 6 are in Phase 3-4 (later-stage) and 4 in Phase 1-2 (earlier-stage). The most active sponsor is Scholar Rock, running 4 of these trials.
Enrollment posture brief
Registry condition spinal-muscular-atrophy
OPEN 6 | RECR 26% | LATE 6 | EARLY 4 | RANK #724 | SPON 4
Volume-matched condition peers
Nearest open-share peer: Parkinson's Disease (26% recruiting · 161 trials)
Spinal Muscular Atrophy lists 6 open slots (26% of 23 indexed trials). Peers are matched on registered volume, not therapeutic-area browse lists. How peer matching works →
Key findings
What ClinicalTrials.gov does not surface for Spinal Muscular Atrophy on its own pages , computed from the registry mirror as of 2026-08-08. Each line carries its own denominator so it can be quoted as it stands.
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Spinal Muscular Atrophy is recruiting below its size-band peers.
6 of 23 indexed trials (26.1%) are open to enrollment, against a 39.7% average across 762 conditions with 20-99 trials. The peer set uses the same ClinicalTrials.gov-derived counts as this page.
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One sponsor concentrates Spinal Muscular Atrophy research.
Scholar Rock accounts for 4 of 23 trials (17.4%). 17 distinct sponsors appear on at least one study for this condition label.
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Most Spinal Muscular Atrophy trials plan to enrol fewer than 100 people.
Median target enrolment is 54 among the 23 trials that report a genuine target (sentinel values excluded). 14 of those (60.9%) plan for fewer than 100 participants.
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Spinal Muscular Atrophy splits between interventional and observational designs.
15 interventional and 8 observational studies make up this index (65.2% interventional). Study type is sponsor-reported on ClinicalTrials.gov.
Open and recent trials
23 total, page 1 of 1
Study of an Intrathecal Port and Catheter System for Subjects With Spinal Muscular Atrophy
Alcyone Therapeutics
NCT05866419
Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy
NMD Pharma A/S
NCT05794139
A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy
Scholar Rock
NCT07047144
Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease
University of Missouri-Columbia
NCT07478172
Characterizing Perceived Physical Fatigability in Nusinersen-treated SMA
Columbia University
NCT06955897
Exploring the Physiologic, Pharmacodynamic, and Clinical Responses of Skeletal Muscle in Patients With Spinal Muscular Atrophy Treated With SMN-Directed Therapies
St. Jude Children's Research Hospital
NCT06532474
Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA)
Genentech
NCT05232929
A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy
Biohaven Pharmaceuticals
NCT05337553
Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab
Scholar Rock
NCT05626855
Spinraza in Adult Spinal Muscular Atrophy
Washington University School of Medicine
NCT03709784
A Study to Learn About the Effect of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Risdiplam (ASCEND)
Biogen
NCT05067790
Risdiplam in Patients With Spinal Muscular Atrophy Previously Treated With Nusinersen
Clinic for Special Children
NCT05522361
COMMAND Early Feasibility Study: Implantable BCI to Control a Digital Device for People With Paralysis
Synchron Medical
NCT05035823
Pediatric Powered Wheelchair Standing Devices: An Exploratory Study
Grand Valley State University
NCT05117827
Spinal Cord Stimulation for the Treatment of Motor Deficits in People With Spinal Muscular Atrophy - Upper Limb
Marco Capogrosso
NCT06300996
Genetics of Pediatric-Onset Motor Neuron and Neuromuscular Diseases
Nemours Children's Clinic
NCT02532244
Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam
Scholar Rock
NCT05156320
A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy
Hoffmann-La Roche
NCT03032172
A Pilot Study of Biomarkers for Spinal Muscular Atrophy
Carelon Research
NCT00756821
Clinical Study of Spinal Muscular Atrophy
Columbia University
NCT00443066
An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular Atrophy
Scholar Rock
NCT03921528
Mechanisms and Treatment of Exercise Intolerance and Persistent Fatigue in Spinal Muscular Atrophy
Columbia University
NCT05518773
Mechanisms of Cell Death in Spinal Muscular Atrophy
Nemours Children's Clinic
NCT01754441
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 2 | 4 |
| Phase 3 | 4 |
| Phase 4 | 2 |
Top Sponsors
Named interventions
Most-linked intervention names on Spinal Muscular Atrophy records (sponsor-reported on ClinicalTrials.gov). Counts are trial links, not unique products.
US sites by state
Facility locations filed on Spinal Muscular Atrophy trials. A trial with sites in several states counts in each.
Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.
Related
What to do with this Spinal Muscular Atrophy page
23 registered trials, 6 currently recruiting, is a starting point for a conversation, not a diagnosis or a recommendation.
- 6 Spinal Muscular Atrophy trials are currently recruiting, check eligibility criteria before contacting a site. Browse recruiting trials
- Scholar Rock sponsors the most Spinal Muscular Atrophy trials on record, review their full trial history before evaluating a specific study. See Scholar Rock's trials
- Bring the specific trial ID (NCT number) to your own doctor before enrolling, this page is a directory, not medical guidance. Read the trial-finding guide
Trial and recruiting counts reflect ClinicalTrials.gov registry status as of the data vintage above; individual trial eligibility and enrollment status can change.
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This Spinal Muscular Atrophy condition card totals 23 registered trials; most-linked intervention Risdiplam; top US site state New York. See our editorial standards & corrections policy, the methodology behind these numbers, the data changelog, or report a data error.