Rare Genetic Disease
5
2
2
1
Key Insights
Highlights
Success Rate
100% trial completion (above average)
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 50/100
0.0%
0 terminated out of 5 trials
100.0%
+13.4% vs benchmark
0%
0 trials in Phase 3/4
0%
0 of 1 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.6%
Based on 1 completed trials
Clinical Trials (5)
Using a Speech-Generating Device to Support Communication in Rare Genetic Conditions
Transcriptomic Analysis of Fibroblasts and Blood in Patients With Rare Diseases
Role of the Nuclear Pore Component RANBP2 in Inflammatory Responses to Viral Infections
Research for Individualized Therapeutics in Rare Genetic Disease
Realization of Sequencing of All Known Human Genes in Case of Detection of Cerebral, Renal or Ophthalmological Fetal Malformations During Pregnancy in Order to Make an Etiological Diagnosis and to Precise the Fetal Prognosis