Genetic Disorders
13
1
1
9
Key Insights
Highlights
Success Rate
100% trial completion (above average)
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 37/100
0.0%
0 terminated out of 13 trials
100.0%
+13.4% vs benchmark
8%
1 trials in Phase 3/4
44%
4 of 9 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.6%
Based on 9 completed trials
Clinical Trials (13)
RNA to the RESCUE: Evaluation to Assess the Clinical Utility of RNA Sequencing in Establishing a Genetic Diagnosis or Adjudicating a Previously Established Genetic Diagnosis.
The Genetics Update
Whole Exome Sequencing and Whole Genome Sequencing for Nonimmune Fetal/Neonatal Hydrops
Intensive Multimodal Neurorehabilitation Targeting Neuroplasticity in Pediatric Neurodevelopmental and Chromosomal Disorders
Clinical Implementation of Carrier Status Using Next Generation Sequencing
A Study of the Safety and Immunogenicity of Repeated rhC1INH Administration
Phase II, Randomized, Placebo-controlled Trial in Patients With Charcot-marie-tooth Disease Type 1A
Face Anthropometric Pattern Recognition Technology for Computer Aided Diagnosis of Human Genetic Disorders.
Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema
Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema
Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema
Allogeneic Bone Marrow Transplantation for the Treatment of Genetic Disorders of Erythropoiesis
Reproducibility of the Array-Based Comparative Genomic Hybridization (aCGH) System Using Whole Blood Samples