Spinal Muscular Atrophy (SMA) clinical trials
23 active trials name this condition (recorded in the registry as “Spinal Muscular Atrophy”) . Describe your situation to see which ones you may be eligible for.
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- A 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
- A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy
- A 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)
- 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
- HABIT-ILE + FST in Children With SMA: Preliminary Effectiveness
- A 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
- A 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)
- A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy
- PT for Montana Children With NMD Using Hippotherapy and the Equine Environment
- Study of an Intrathecal Port and Catheter System for Subjects With Spinal Muscular Atrophy
- Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease
- Exploring the Physiologic, Pharmacodynamic, and Clinical Responses of Skeletal Muscle in Patients With Spinal Muscular Atrophy Treated With SMN-Directed Therapies
- Characterizing Perceived Physical Fatigability in Nusinersen-treated SMA
- Gait and Bone Health in SMA
- Diagnostic Journey, Patient Experience, and Disparities in the Treatment of Spinal Muscular Atrophy (SMA) in the MedStar Health System
- Establishing Walking-related Digital Biomarkers in Rare Childhood Onset Progressive Neuromuscular Disorders
- Assessing the Fertility Status of Men With Spinal Muscular Atrophy (SMA)
- A 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
- Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)
- Families Filming Infants Learning Movement
- ALS/MND Natural History Study Data Repository
- Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials
- Early Check: Expanded Screening in Newborns
Guides for people searching for a trial
- Why people get turned down — and what to do nextWhat eligibility criteria are for, what keeps people out, and where to go when the first door closes.
- How your biomarkers decide which trials you qualify forEGFR, HER2, BRAF, PD-L1 and the rest — what they are and why they increasingly gate access.
- How to search for a trial without losing your mindWhere to look, what to gather first, and how to read what you find.
- What Phase 1, 2 and 3 actually mean for youWhat each phase is testing, and how to weigh an early-phase trial against a later one.
- 10 questions to ask before you say yesTreatment, time, cost, safety and your rights. Worth bringing to a screening visit.
- You matched with a trial. What happens now?Phone screens, the screening visit, informed consent, and day one.
Showing active trials that list “Spinal Muscular Atrophy” as a condition. Eligibility shown on each trial is an estimate — the trial's study team makes the final decision.