Spinal Muscular Atrophy
153
25
42
75
Key Insights
Highlights
Success Rate
94% trial completion (above average)
Published Results
21 trials with published results (14%)
Research Maturity
75 completed trials (49% of total)
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 40/100
3.3%
5 terminated out of 153 trials
93.8%
+7.2% vs benchmark
10%
16 trials in Phase 3/4
28%
21 of 75 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.6%
Based on 75 completed trials
Clinical Trials (153)
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
A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy
A Registry of Cases of Spinal Muscular Atrophy
A Real-World Medical Chart Review of Spinal Muscular Atrophy Patients Treated With Onasemnogene Abeparvovec in Saudi Arabia
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 of the Safety and Effectiveness of Onasemnogene Abeparvovec (Zolgensma) Intrathecal Injection in Spinal Muscular Atrophy Patients
Acceptability, Feasibility, Safety and Efficacy of a Optimized Rehabilitation Program for Treated Patients With Spinal Muscular Atrophy (SMA).
Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA)
COMMAND Early Feasibility Study: Implantable BCI to Control a Digital Device for People With Paralysis
Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab
UK SMA Patient Registry
Study of an Intrathecal Port and Catheter System for Subjects With Spinal Muscular Atrophy
Equine-Assisted Physiotherapy for Spinal Muscular Atrophy
Spinal Muscular Atrophy and School Transitions
Effects of Developmental Support for Mothers of Infants With Spinal Muscular Atrophy
Exploring the Physiologic, Pharmacodynamic, and Clinical Responses of Skeletal Muscle in Patients With Spinal Muscular Atrophy Treated With SMN-Directed Therapies
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