Genetic Disorder
35
3
6
22
Key Insights
Highlights
Success Rate
92% trial completion (above average)
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 30/100
5.7%
2 terminated out of 35 trials
91.7%
+5.2% vs benchmark
26%
9 trials in Phase 3/4
18%
4 of 22 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 22 completed trials
Clinical Trials (35)
Inherited Reproductive Disorders
Skeletal Muscle Biomarkers in People With Fragile Sarcolemmal Muscular Dystrophy
Evaluation of Association Between Testosterone Levels, Dementia, and Adverse Mental Health Outcomes
DNA Sequencing in Clinical Practice, Mayo Clinic Health Tapestry Study
Pediatric Patients With Metabolic or Other Genetic Disorders
SLC13A5 Deficiency Natural History Study - United States Only
Promoting Stress Management and Resilience Among Individuals With Von Hippel- Lindau Disease
Utility of Gene Test Analysis for Diagnosis, Prognosis and Treatment of Patients With Genetic Arrhythmic Heart Disease: the ARRHYTHMIC GENE-HEART
SLC13A5 Deficiency Natural History Study - Remote Only
Lipid Transport Disorder Italian Genetic Record (LIPIGEN)
Retrospective Observational Study on Efficacy and Safety of Norditropin® in Children With Prader-Willi Syndrome
Genetic Testing of Noonan Subjects Previously Treated With Norditropin®. An Extension to Trial GHNOO-1658
Post Marketing Surveillance on Long-term Use With Norditropin® (Short Stature Due to Noonan Syndrome)
Fludarabine Phosphate, Cyclophosphamide, and Total-Body Irradiation Followed by Donor Bone Marrow Transplant and Cyclophosphamide, Mycophenolate Mofetil, Tacrolimus, and Sirolimus in Treating Patients With Primary Immunodeficiency Disorders or Noncancerous Inherited Disorders
Investigating the Long-term Efficacy and Safety of Two Doses of NN-220 (Somatropin) in Short Stature Due to Noonan Syndrome
Delineation of Novel Monogenic Disorders in the United Arab Emirates Population
Brain-Derived Neurotrophic Factor in Obesity and Brain Function
Studies of Children With Metabolic and Other Genetic Disorders
Pharmacokinetics of Thymoglobulin in Paediatric Haematopoietic Stem-cell Transplants
Observational Prospective Study on Patients Treated With Norditropin®