Neuromuscular Diseases
149
28
36
72
Key Insights
Highlights
Success Rate
87% trial completion (above average)
Published Results
14 trials with published results (9%)
Research Maturity
72 completed trials (48% of total)
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 50/100
7.4%
11 terminated out of 149 trials
86.7%
+0.2% vs benchmark
9%
14 trials in Phase 3/4
19%
14 of 72 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.6%
Based on 72 completed trials
Clinical Trials (149)
Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients
Managed Access Program for Del-zota in Participants With DMD Mutations Amenable to Exon 44 Skipping
A Prospective Natural History and Outcome Measure Discovery Study of Charcot-Marie-Tooth Disease, Type 4J
CIMR Neuromuscular Research Biobank
Feasibility, Validation and Application of Digital Tools for the Follow-up of Neuromuscular Patient Mobility in Daily Living
Detecting Systemic Carbon Dioxide Levels With a Novel Biosensor
Assessment of Volume-targeted Ventilation in Patients With Neuromuscular Disease
A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy
Optimisation of Mechanical Insufflation:Exsufflation
Investigating Health Related Quality of Life in Patients With Chronic Respiratory Failure
A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy
Does the Coaching Function Improve the Learning of Mechanical In-exsufflation (MI-E) in Paediatric Subjects?
Physical Activity Level at Home in CMT1A Patients: Wearable Sensor Assessment
The Peer Support Study
Soft-robotic Glove Support of High-demand Tasks
Muscle Oxygenation in Effort in Neuromuscular Diseases
A Phase 3 Study of NTLA-2001 in ATTRv-PN
Spastic Paraplegia - Centers of Excellence Research Network
Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy
Measuring Physical Activity With ActivPal