RecruitingPhase 3NCT07723248
Neuromuscular
Spinal Muscular Atrophy
Also known as SMA Type 1 (Werdnig-Hoffmann), SMA Type 2, SMA Type 3 (Kugelberg-Welander), SMA Type 4
Spinal muscular atrophy is caused by deletion or mutation of the SMN1 gene, leading to insufficient SMN protein and progressive loss of motor neurons. Type 1 (the most severe) presents before 6 months and was historically fatal by age 2.
ORPHA:70 ↗Gene SMN1Prevalence 1-9 per 100,000 (Orphanet)Onset All ages (type-dependent)Genetic (autosomal recessive)
80
studies recruiting now
as of 7 Sept 2026
462
studies registered in total
as of 7 Sept 2026
8
countries with a recruiting site
as of 7 Sept 2026
23 Jul 2026
most recent study posted
among recruiting studies
Recruiting trials
RecruitingPhase 4NCT05861986
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy
RecruitingNCT07531719
A Study of Risdiplam in Participants With Type I and Type II Spinal Muscle Atrophy (SMA)
RecruitingPhase 1NCT06555419
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)
RecruitingNot applicableNCT07521930
Interfacing With NeuroTechnology to Expand Neural Throughput (INTENT)
Showing the 5 most recently updated recruiting studies, as recorded 7 Sept 2026. Live status on each study page.
See all 80 recruiting studiesWhere recruiting studies are running
Countries with at least one recruiting site among the studies above, 7 Sept 2026. Tap a country to search trials there.
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About Spinal Muscular Atrophy
Spinal muscular atrophy is caused by deletion or mutation of the SMN1 gene, leading to insufficient SMN protein and progressive loss of motor neurons. Type 1 (the most severe) presents before 6 months and was historically fatal by age 2. Nusinersen (Spinraza), onasemnogene abeparvovec (Zolgensma), and risdiplam (Evrysdi) have transformed outcomes. Newborn screening now allows pre-symptomatic treatment before any motor loss occurs.
Common clinical features
Proximal muscle weaknessSkeletal muscle atrophyIntercostal muscle weaknessAxial muscle weaknessQuadriceps muscle weaknessRecurrent respiratory infectionsDifficulty climbing stairsHypotonia
From Orphanet’s phenotype annotations (CC BY 4.0). Not a complete list.
Before you apply
Things trial teams commonly ask about for Spinal Muscular Atrophy. Not eligibility rules; those are set by each study.
- SMA type (1, 2, 3, or 4) and current treatment status (nusinersen, risdiplam, or gene therapy) are the most important eligibility factors
- SMN2 copy number must typically be confirmed - higher copy number correlates with milder disease and affects trial arms
- Pre-symptomatic infants identified through newborn screening have access to specific early intervention trials
Related conditions
Information, not medical advice. Trial listings are shown as recorded on ClinicalTrials.gov; whether any study is right for you is a decision for you and your clinicians, and eligibility is decided by each research team. Disease information from Orphanet (CC BY 4.0).