Genetic Diseases
20
6
7
7
Key Insights
Highlights
Success Rate
78% trial completion
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 49/100
10.0%
2 terminated out of 20 trials
77.8%
-8.7% vs benchmark
10%
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: 86.5%
Based on 7 completed trials
Clinical Trials (20)
Familial Mediterranean Fever and Related Disorders: Genetics and Disease Characteristics
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
BAG3-DCM Natural History Study
PKP2-ACM Natural History Study
Genotype-phenotype Characterization Study on Genetic Diseases With Immune and Neurological Dysfunctions
Impaired Type I IFN Immunity Due to Autoantibodies or a Genetic Defect: a Prospective National Cohort
The Alfred Step Test Exercise Protocol (A-STEP), for Adults With Cystic Fibrosis.
Genetic and Metabolic Disease in Children
Perinatal Precision Medicine
Decoding Developmental Disorders in Humams
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
Time-Lapse Incubation for Embryo Culture - Morphokinetics and Environmental Stability
Prenatal Cytogenetic Diagnosis by Array-Based Copy Number Analysis