Spinal Muscular Atrophy
149
24
42
71
Key Insights
Highlights
Success Rate
93% trial completion (above average)
Published Results
21 trials with published results (14%)
Research Maturity
71 completed trials (48% of total)
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 40/100
3.4%
5 terminated out of 149 trials
93.4%
+6.9% vs benchmark
11%
16 trials in Phase 3/4
30%
21 of 71 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 71 completed trials
Clinical Trials (149)
A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy
Feasibility of Virtual Reality in Children With Neuromuscular Disease, Effectiveness of Virtual Reality and Biofeedback
Effects of Developmental Support for Mothers of Infants With Spinal Muscular Atrophy
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
Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA)
Study of an Intrathecal Port and Catheter System for Subjects With Spinal Muscular Atrophy
Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy
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)
Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease
Investigating NMJ Defects in SMA Following Central and Peripheral SMN Restoration
Motor Unit Number Estimation (MUNE) in Adults With Spinal Muscular Atrophy (SMA)
Characterizing Perceived Physical Fatigability in Nusinersen-treated SMA
A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy
Characterization of New Phenotypes of Patients With Spinal Muscular Atrophy Treated With SMN Restoring Therapy
Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2
Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy
Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam
Phase IIIb, Open-label, Multi-center Study to Evaluate Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally to Participants With SMA Who Discontinued Treatment With Nusinersen or Risdiplam
Motor Development and Orthoses in Spinal Muscular Atrophy (SMA)
Implementation of FEES in Spinal Muscle Atrophy