Rare Diseases
94
35
40
19
Key Insights
Highlights
Success Rate
83% trial completion
Clinical Risk Assessment
Based on trial outcomes
High Risk
Score: 62/100
4.3%
4 terminated out of 94 trials
82.6%
-3.9% vs benchmark
2%
2 trials in Phase 3/4
21%
4 of 19 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 19 completed trials
Clinical Trials (94)
Longitudinal Studies of Patient With FPDMM
Genetic Analysis of Uncommon Disease Presentations in Non-US Populations
AI-assisted Rare Disease Diagnosis
Clinical Trial of the Dual Vector Base Editor for the Treatment of the CHD3-R1025W Mutation
Epidemiological Study of Treatment Approaches on AQP4-IgG Positive NMOSD in Russia
The Mayo Clinic Rare and Undiagnosed Disease Hackathon
Epidemiological Study of Treatment Approaches in AChR-Antibody Positive Generalized Myasthenia Gravis in Russia
Identification of Cellular Biomarkers of Rare Eye Diseases in Adults
Marfan Syndrome and Quality of Life of Pediatric Patients
Functional Study to Indentify Genetic Etiology of Rare Diseases - ORIGIN
Ascending Doses of Crofelemer Powder for Oral Solution in Pediatric Microvillus Inclusion Disease (MVID)
Artificial Intelligence for Rare Disease Diagnosis
Baker Gordon Syndrome Natural History Study
UW Undiagnosed Genetic Diseases Program
Fibroblasts and Thoracic Aortic Aneurysms: in Vitro Characterization in With Marfan Syndrome and Genetic Aortic Diseases
Intact Cord Resuscitation in CDH
Diagnostic Value of Exome/ Genome Sequencing, Conventional Methods in Rare Diseases and Familial Tumor Syndromes
A Multi-Dimensional Model of cAre and transItion for Patients With cOmplex RAre Diseases
Evaluation of Socio-professional Inclusion for Young Adults Aged 15-25 Living With a Rare Genetic Disability
Using Social Robots in Children With Rare Diseases and Their Parents: A Feasibility Study