Spinal Muscular Atrophy Type I
11
1
2
6
Key Insights
Highlights
Success Rate
86% trial completion
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 39/100
9.1%
1 terminated out of 11 trials
85.7%
-0.8% vs benchmark
18%
2 trials in Phase 3/4
50%
3 of 6 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 6 completed trials
Clinical Trials (11)
Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular Atrophy Type 1
Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies
Psychological Evaluation of the Parental Experience of Newborn Screening for Infantile Spinal Muscular Atrophy in the Grand Est and Nouvelle-Aquitaine Regions
Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi
Impact of Motor and Oral Motor Function on Quality of Life in Children With SMA
Therapeutic Management and Use of Resources and Costs of Spinal Muscular Atrophy in Spain
Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients with Spinal Muscular Atrophy Type 1
The Effect of Spinal Orthosis on the Development of Scoliosis and Chest Deformity in Type I Spinal Muscular Atrophy
Body Weight Support Harness System in Spinal Muscular Atrophy
CARNIVAL Type I: Valproic Acid and Carnitine in Infants With Spinal Muscular Atrophy (SMA) Type I
Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Type I