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
9%
3 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
Genetic Studies in the Amish and Mennonites
Application of a Systematic Developmental Assessment to a Novel Population: Infants With Rare Genetic Disorders
Insights Into Microbiome and Environmental Contributions to Sickle Cell Disease and Leg Ulcers Study (INSIGHTS Study)
UW Undiagnosed Genetic Diseases Program
Penn Medicine Biobank Return of Results Program
Using a Speech-Generating Device to Support Communication in Rare Genetic Conditions
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
Delineating the Molecular Spectrum and the Clinical, Imaging and Neuronal Phenotype of Chopra-Amiel-Gordon Syndrome
Accurate Assessment and Intervention Research on Newborn Whole Genome Sequencing and Genetic Disease Risk
Alpha-1 Antitrypsin Disease Cohort: Longitudinal Biomarker Study of Disease
Improved Diagnosis of Familial Hypercholesterolemia Across the Northland (ID-FH)