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Spinal Muscular Atrophy clinical trials recruiting in the U.S.
20 studies for Spinal Muscular Atrophy are recruiting participants at sites across the United States.
Spinal muscular atrophy (SMA) attacks nerve cells in the spinal cord, weakening voluntary muscles. Read about the genetics, types, and what may help.
Cities with the most studies
- Mountain View, CA9 studies
- New York City, NY8 studies
- Boston, MA7 studies
- Norfolk, VA6 studies
- Philadelphia, PA6 studies
- Houston, TX5 studies
- Chicago, IL5 studies
- Phoenix, AZ4 studies
- Grand Rapids, MI4 studies
- Lewisville, TX4 studies
- Baltimore, MD3 studies
- Charlottesville, VA3 studies
- Harrisburg, PA3 studies
- Orlando, FL3 studies
- Orange, CA3 studies
- Little Rock, AR3 studies
- Cedar Rapids, IA3 studies
- Durham, NC3 studies
- Memphis, TN3 studies
- Minneapolis, MN3 studies
- Portland, OR3 studies
By state
- Arizona4 studies
- Arkansas3 studies
- California11 studies
- Florida6 studies
- Illinois5 studies
- Iowa3 studies
- Maryland3 studies
- Massachusetts8 studies
- Michigan4 studies
- Minnesota3 studies
- Missouri3 studies
- New York9 studies
- North Carolina3 studies
- Ohio3 studies
- Oregon3 studies
- Pennsylvania7 studies
- Tennessee3 studies
- Texas8 studies
- Virginia7 studies
Recently updated studies
A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety in Participants Aged 15 to 60 Years With Spinal Muscular Atrophy (SMA) Who Are Either New to SMA Treatment or Were Previously Treated With Risdiplam
RecruitingPhase 3
Ages 15 to 60 · All sexes · Updated Nov 13, 2026 · NCT07444476A Study to Learn How Nusinersen (Spinraza) Affects Participants With Spinal Muscular Atrophy (SMA) Who Took it Before or During Pregnancy And About The Health of Their Babies
RecruitingObservational study
Ages 18 to 54 · Women · Updated Nov 12, 2026 · NCT05789758Study of an Intrathecal Port and Catheter System for Subjects With Spinal Muscular Atrophy
Recruiting
Ages 3 and older · All sexes · Updated Nov 9, 2026 · NCT05866419ASsessing The REAl-world Safety & Effectiveness of Spinal Muscular Atrophy Participants Treated With Intrathecal Onasemnogene Abeparvovec-brve (OAV101B) (ITVISMA®): A U.S. Pragmatic Multicenter Study (STREAM)
RecruitingPhase 4
Ages 2 to 100 · All sexes · Updated Oct 29, 2026 · NCT07448610A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety When Given Before Symptoms Appear in Babies With Genetically Diagnosed Spinal Muscular Atrophy (SMA)
RecruitingPhase 3
0 Days – 42 Days · All sexes · Updated Oct 23, 2026 · NCT07221669A Registry of Cases of Spinal Muscular Atrophy
RecruitingObservational study
All ages · All sexes · Updated Oct 22, 2026 · NCT07805278HABIT-ILE + FST in Children With SMA: Preliminary Effectiveness
Recruiting
Ages 5 to 17 · All sexes · Updated Oct 22, 2026 · NCT07488338A Study to Learn About the Safety and Effects of Salanersen (BIIB115) When Given to Babies With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Onasemnogene Abeparvovec
RecruitingPhase 3
0 Days – 7 Months · All sexes · Updated Oct 15, 2026 · NCT07444450Characterizing Perceived Physical Fatigability in Nusinersen-treated SMA
RecruitingObservational study
Ages 12 and older · All sexes · Updated Oct 14, 2026 · NCT06955897Interfacing With NeuroTechnology to Expand Neural Throughput (INTENT)
Recruiting
Ages 18 to 80 · All sexes · Updated Oct 9, 2026 · NCT07521930A Study to Assess the Safety, Tolerability, Efficacy, Pharmacokinetics, and Immunogenicity of Intravenous Administration of ARGX-119 in Pediatric Participants Aged 5 to Less Than 18 Years With Spinal Muscular Atrophy
RecruitingPhase 2
Ages 5 to 17 · All sexes · Updated Oct 8, 2026 · NCT07287982A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy
RecruitingPhase 2
Up to age 2 · All sexes · Updated Oct 2, 2026 · NCT07047144A Study to Find Out How Nusinersen is Processed in the Body When Given Through the ThecaFlex DRx™ System in Adult and Pediatric Participants With Spinal Muscular Atrophy (PIERRE-PK)
RecruitingPhase 1
Ages 3 and older · All sexes · Updated Sep 3, 2026 · NCT06555419Exploring the Physiologic, Pharmacodynamic, and Clinical Responses of Skeletal Muscle in Patients With Spinal Muscular Atrophy Treated With SMN-Directed Therapies
RecruitingObservational study
Ages 5 to 20 · All sexes · Updated Jul 9, 2026 · NCT06532474Clinical Procedures to Support Research in ALS
RecruitingObservational study
Ages 18 and older · All sexes · Updated Jun 15, 2026 · NCT03489278ALS/MND Natural History Study Data Repository
RecruitingObservational study
Ages 18 to 90 · All sexes · Updated May 20, 2026 · NCT05966038Establishing Walking-related Digital Biomarkers in Rare Childhood Onset Progressive Neuromuscular Disorders
RecruitingObservational studyHealthy volunteers welcome
Ages 5 and older · All sexes · Updated Apr 9, 2026 · NCT06839469Gait and Bone Health in SMA
RecruitingObservational study
Ages 8 to 50 · All sexes · Updated Mar 18, 2026 · NCT07400198Diagnostic Journey, Patient Experience, and Disparities in the Treatment of Spinal Muscular Atrophy (SMA) in the MedStar Health System
RecruitingObservational study
Ages 18 and older · All sexes · Updated Jan 16, 2026 · NCT07223489Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)
RecruitingObservational study
All ages · All sexes · Updated Jan 31, 2025 · NCT04174157From ClinicalTrials.gov, data retrieved Nov 15, 2026. Each study sets its own eligibility; the study team decides who can join.