Fucosidosis
8
0
1
5
Key Insights
Highlights
Success Rate
83% trial completion
Clinical Risk Assessment
Based on trial outcomes
High Risk
Score: 59/100
12.5%
1 terminated out of 8 trials
83.3%
-3.2% vs benchmark
0%
0 trials in Phase 3/4
60%
3 of 5 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 5 completed trials
Clinical Trials (8)
MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
Longitudinal Studies of the Glycoproteinoses
Human Placental-Derived Stem Cell Transplantation
Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders
Stem Cell Transplant for Inborn Errors of Metabolism
Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism
Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells
Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation