Spinal Muscular Atrophy (SMA)
28
15
17
2
Key Insights
Highlights
Success Rate
50% trial completion
Clinical Risk Assessment
Based on trial outcomes
High Risk
Score: 62/100
7.1%
2 terminated out of 28 trials
50.0%
-36.5% vs benchmark
7%
2 trials in Phase 3/4
50%
1 of 2 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 2 completed trials
Clinical Trials (28)
A Study to Assess the Safety, Tolerability, Efficacy, Pharmacokinetics, and Immunogenicity of Intravenous Administration of ARGX-119 in Pediatric Participants Aged 5 to Less Than 18 Years With Spinal Muscular Atrophy
An International Federated Model for Wearable-derived Remote Longitudinal Motor Monitoring in Young Children With Spinal Muscular Atrophy Compared With Healthy Controls: Active-NBS Study (UK)
Gut Peptides and Bone Remodeling in Children With Neuromuscular Disorders
HABIT-ILE + FST in Children With SMA: Preliminary Effectiveness
A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophy
Self-Efficacy Enhancement Using a Multicomponent Support Group for Caregivers of Children With DMD/SMA
Observational Study to Observe Variations of Gait Parameters in Patients With Neuromuscular Diseases
Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials
Diagnostic Journey, Patient Experience, and Disparities in the Treatment of Spinal Muscular Atrophy (SMA) in the MedStar Health System
Neuroproprioceptive Equine-Assisted Physiotherapy for Spinal Muscular Atrophy
Long Read Analysis in Spinal Muscular Atrophy - LOREASI
Assessment of a Portable Digital Device for Quantified Analysis of Markerless Walking in Volunteers With Neuromuscular Diseases or Asymptomatic Volunteers
Active NBS Study: Decentralised Monitoring Motor Development in Children With Duchenne Muscular Dystrophy or Spinal Muscular Atrophy Identified by Newborn Screening
Real World Clinical Effectiveness & Safety of Vesemnogene Lantuparvovec for Spinal Muscular Atrophy (SMA) in Low-middle Income Countries (LMIC).
The Effect of a Muscle-mimicking, Fabric-type Shoulder Orthosis on Functional Movements of the Upper Limb in Patients With Neuromuscular Disorder
Psychological Evaluation of the Parental Experience of Newborn Screening for Infantile Spinal Muscular Atrophy in the Grand Est and Nouvelle-Aquitaine Regions
Robot-assisted Training
The Use of Assistive Gait Devices Can Reduce the Risk of Falls in Patients With Neuromuscular Diseases Following a Training Period.
Safety and Tolerability of Low Motoneuron Stimulation Via Transcranial Magnetic Stimulation in Spinal Muscular Atrophy
Adult SMA Research and Clinical Hub