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Spinal Muscular Atrophy clinical trials recruiting in the U.S.
23 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
- New York City, NY9 studies
- Mountain View, CA8 studies
- Philadelphia, PA7 studies
- Norfolk, VA6 studies
- Chicago, IL6 studies
- Grand Rapids, MI6 studies
- Houston, TX5 studies
- Boston, MA5 studies
- Aurora, CO4 studies
- Little Rock, AR4 studies
- Memphis, TN4 studies
- Phoenix, AZ4 studies
- Lewisville, TX4 studies
- Columbia, MO3 studies
- Durham, NC3 studies
- Harrisburg, PA3 studies
- Cedar Rapids, IA3 studies
- Charlottesville, VA3 studies
- Fort Worth, TX3 studies
- Minneapolis, MN3 studies
- Portland, OR3 studies
- Orange, CA3 studies
- Orlando, FL3 studies
- Dallas, TX3 studies
- Gainesville, FL3 studies
- Atlanta, GA3 studies
By state
- Arizona4 studies
- Arkansas4 studies
- California10 studies
- Colorado4 studies
- Florida8 studies
- Georgia3 studies
- Illinois6 studies
- Iowa3 studies
- Massachusetts6 studies
- Michigan6 studies
- Minnesota3 studies
- Missouri4 studies
- New York10 studies
- North Carolina3 studies
- Oregon3 studies
- Pennsylvania8 studies
- Tennessee4 studies
- Texas10 studies
- Virginia7 studies
Recently updated studies
ASsessing 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 Sep 18, 2026 · NCT07448610A 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 Sep 14, 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 Sep 14, 2026 · NCT07047144A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy
RecruitingPhase 4
3 Months – 24 Months · All sexes · Updated Sep 10, 2026 · NCT05861999A Registry of Cases of Spinal Muscular Atrophy
RecruitingObservational study
All ages · All sexes · Updated Sep 8, 2026 · NCT07805278A 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 · NCT06555419A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy
RecruitingPhase 4
3 Months – 24 Months · All sexes · Updated Sep 3, 2026 · NCT05861986Interfacing With NeuroTechnology to Expand Neural Throughput (INTENT)
Recruiting
Ages 18 to 80 · All sexes · Updated Sep 2, 2026 · NCT07521930Study of an Intrathecal Port and Catheter System for Subjects With Spinal Muscular Atrophy
Recruiting
Ages 3 and older · All sexes · Updated Aug 19, 2026 · NCT05866419A 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 Aug 18, 2026 · NCT07221669A 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 Aug 11, 2026 · NCT07444450Exploring 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 · NCT05966038Characterizing Perceived Physical Fatigability in Nusinersen-treated SMA
RecruitingObservational study
Ages 12 and older · All sexes · Updated May 14, 2026 · NCT06955897A 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 Apr 21, 2026 · NCT07444476Establishing 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 · NCT06839469HABIT-ILE + FST in Children With SMA: Preliminary Effectiveness
Recruiting
Ages 5 to 17 · All sexes · Updated Mar 23, 2026 · NCT07488338Gait and Bone Health in SMA
RecruitingObservational study
Ages 8 to 50 · All sexes · Updated Mar 18, 2026 · NCT07400198Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease
Recruiting
Ages 18 and older · All sexes · Updated Mar 17, 2026 · NCT07478172A 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 Mar 9, 2026 · NCT05789758Diagnostic 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 Sep 29, 2026. Each study sets its own eligibility; the study team decides who can join.