Wolman Disease
13
4
4
7
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%
+13.5% vs benchmark
0%
0 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 (13)
Registry of Patients Diagnosed With Lysosomal Storage Diseases
PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)
Lysosomal Acid Lipase (LAL) Deficiency Registry
Breakfast for Young Women
The Effect of Kegel Exercise and Ba Duan Jin Applications on Premenopausal Women With Urinary Incontinence
Human Placental-Derived Stem Cell Transplantation
Prevalence and Risk Factors of Women Mental Health Disorders
HSCT for High Risk Inherited Inborn Errors
Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency
Stem Cell Transplant for Inborn Errors of Metabolism
A Retrospective Natural History Study of Patients With Lysosomal Acid Lipase Deficiency/Wolman Phenotype
Wolman/CESD Natural History Chart Review and Longitudinal Follow-Up
Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation