X-Linked
7
1
1
3
Key Insights
Highlights
Success Rate
100% trial completion (above average)
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 40/100
0.0%
0 terminated out of 7 trials
100.0%
+13.5% vs benchmark
14%
1 trials in Phase 3/4
33%
1 of 3 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 3 completed trials
Clinical Trials (7)
A Phase 3 Study to Evaluate the Safety and Efficacy of AOC 1044 (Also Referred to as Delpacibart Zotadirsen) in Participants With DMD With Gene Mutations Amenable to Exon 44 Skipping
Morphology in Oral Rare Syndromes & Artificial Intelligence for Clinical Diagnosis
Hemophilia A Research Program
Study of RS1 Ocular Gene Transfer for X-linked Retinoschisis
Better Delineation of DDX3X Related Phenotype and Epigenetic Signature.
People s Expectations When Enrolling in a Phase I/II RS1 Ocular Gene Transfer Clinical Trial
Mothers Experiences With X-linked Retinoschisis Compared to Fathers Experiences