Spinal Muscular Atrophy
151
22
40
74
Key Insights
Highlights
Success Rate
94% trial completion (above average)
Published Results
21 trials with published results (14%)
Research Maturity
74 completed trials (49% of total)
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 40/100
3.3%
5 terminated out of 151 trials
93.7%
+7.1% vs benchmark
11%
16 trials in Phase 3/4
28%
21 of 74 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.6%
Based on 74 completed trials
Clinical Trials (151)
A Real-World Medical Chart Review of Spinal Muscular Atrophy Patients Treated With Onasemnogene Abeparvovec in Saudi Arabia
Equine-Assisted Physiotherapy for Spinal Muscular Atrophy
Spinal Muscular Atrophy and School Transitions
Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA)
Exploring the Physiologic, Pharmacodynamic, and Clinical Responses of Skeletal Muscle in Patients With Spinal Muscular Atrophy Treated With SMN-Directed Therapies
Effects of Developmental Support for Mothers of Infants With Spinal Muscular Atrophy
Early Check: Expanded Screening in Newborns
An Open Label Study of Gene Therapy Product (Vesemnogene Lantuparvovec) in Spinal Muscular Atrophy
Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy
A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With 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)
A Study to Learn About the Effect of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Risdiplam (ASCEND)
Characterizing Perceived Physical Fatigability in Nusinersen-treated SMA
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
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
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
Investigating NMJ Defects in SMA Following Central and Peripheral SMN Restoration
Motor Unit Number Estimation (MUNE) in Adults With Spinal Muscular Atrophy (SMA)