Genetic Disease
113
44
56
32
Key Insights
Highlights
Success Rate
91% trial completion (above average)
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 50/100
2.7%
3 terminated out of 113 trials
91.4%
+4.9% vs benchmark
2%
2 trials in Phase 3/4
13%
4 of 32 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 32 completed trials
Clinical Trials (113)
Clinical and Genetic Evaluation of Individuals With Undiagnosed Disorders Through the Undiagnosed Diseases Network
Insights Into Microbiome and Environmental Contributions to Sickle Cell Disease and Leg Ulcers Study (INSIGHTS Study)
Rett Syndrome Registry
Functional Study to Indentify Genetic Etiology of Rare Diseases - ORIGIN
Rifampin in CYP24A1-related Hypercalcemia and Hypercalciuria
Educational Video for Genetic Testing
Genetic Studies in the Amish and Mennonites
Application of a Systematic Developmental Assessment to a Novel Population: Infants With Rare Genetic Disorders
UW Undiagnosed Genetic Diseases Program
Using a Speech-Generating Device to Support Communication in Rare Genetic Conditions
Penn Medicine Biobank Return of Results Program
Developing Protocols for Modelling of Genetic Diseases Using Induced Pluripotent Stem Cells
Natural History of Type 1 Interferonopathies: Insights From a European Cohort
Genetic Disorders of Obesity Program Database
Adaptive Optics Retinal Imaging in Inherited and Acquired Retinal Disorders
The Natural History of Mitochondrial Diseases
Molecular Diagnosis of Systemic Autoinflammatory Diseases
Ensuring Patients' Informed Access to Noninvasive Prenatal Testing
Single Participant Study of an Experimental ASO Treatment for TUBB4A-related Leukodystrophy
CUHK Stroke Biobank