Genetic Disease
113
44
55
31
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
91.2%
+4.7% vs benchmark
2%
2 trials in Phase 3/4
10%
3 of 31 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 31 completed trials
Clinical Trials (113)
Insights Into Microbiome and Environmental Contributions to Sickle Cell Disease and Leg Ulcers Study (INSIGHTS Study)
Penn Medicine Biobank Return of Results Program
Genetic Studies in the Amish and Mennonites
Clinical and Genetic Evaluation of Individuals With Undiagnosed Disorders Through the Undiagnosed Diseases Network
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
Natural History of Type 1 Interferonopathies: Insights From a European Cohort
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)
Identification and Characterization of Novel Non-Coding Variants That Contribute to Genetic Disorders
Genome Sequencing in the Intensive Care Unit Population
Biocollection of Rare Pediatric-onset of Autoimmune and Autoinflammatory Diseases
Educational Video for Genetic Testing
Using a Speech-Generating Device to Support Communication in Childhood Dementia
STXBP1 and SYNGAP1 Related Disorders Natural History Study
An Open-label, Single Center, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for TUBB4A-related Leukodystrophy