Genetic Diseases
22
7
8
7
Key Insights
Highlights
Success Rate
78% trial completion
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 49/100
9.1%
2 terminated out of 22 trials
77.8%
-8.8% vs benchmark
9%
2 trials in Phase 3/4
43%
3 of 7 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.6%
Based on 7 completed trials
Clinical Trials (22)
The Mayo Clinic Rare and Undiagnosed Disease Hackathon
A Phase 3 Study to Evaluate the Safety and Efficacy of AOC 1044 (Also Referred to as Delpacibart Zotadirsen) in Participants With DMD With Gene Mutations Amenable to Exon 44 Skipping
Familial Mediterranean Fever and Related Disorders: Genetics and Disease Characteristics
Impaired Type I IFN Immunity Due to Autoantibodies or a Genetic Defect: a Prospective National Cohort
The Genetics Update
Decoding Developmental Disorders in Humams
Genetic and Metabolic Disease in Children
BAG3-DCM Natural History Study
PKP2-ACM Natural History Study
Genotype-phenotype Characterization Study on Genetic Diseases With Immune and Neurological Dysfunctions
The Alfred Step Test Exercise Protocol (A-STEP), for Adults With Cystic Fibrosis.
Perinatal Precision Medicine
Rady Children's Institute Genomic Biorepository
The Pilot Study of High-throughput Sequencing in Neonatal Birth Defects
Rapid Whole Genome Sequencing Study
Safety, Tolerability, Pharmacokinetics of ELX-02 in Healthy Adult Volunteers
Combining Lovastatin and a Parent-Implemented Language Intervention for Fragile X Syndrome
Prenatal Microarray Follow-Up Study
Mutation Exploration in Non-acquired, Genetic Disorders and Its Impact on Health Economy and Life Quality
Related Hematopoietic Stem Cell Transplantation (HSCT) for Genetic Diseases of Blood Cells