Genetic Diseases, X-Linked
13
1
2
8
Key Insights
Highlights
Success Rate
80% trial completion
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 39/100
15.4%
2 terminated out of 13 trials
80.0%
-6.5% vs benchmark
31%
4 trials in Phase 3/4
63%
5 of 8 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 8 completed trials
Clinical Trials (13)
Managed Access Program for Del-zota in Participants With DMD Mutations Amenable to Exon 44 Skipping
Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy
A Trial of Metformin in Individuals With Fragile X Syndrome (Met)
A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy
A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy
A Trial of Metformin in Individuals With Fragile X Syndrome
Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors
Lead-in Study to Collect Prospective Efficacy and Safety Data of Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Participants
Rett Syndrome Registry
Kidney Information Network for Disease Research and Education
An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy
Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
Genetic Studies of X-linked Lymphoproliferative Disease