Maple Syrup Urine Disease
9
1
1
5
Key Insights
Highlights
Success Rate
100% trial completion (above average)
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 50/100
0.0%
0 terminated out of 9 trials
100.0%
+13.5% vs benchmark
0%
0 trials in Phase 3/4
20%
1 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 (9)
Evaluation of the Express Plus Range
Baby Detect : Genomic Newborn Screening
MyRareDiet A Novel Diet Tracking Tool
Liquid Valine and Isoleucine in Maple Syrup Urine Disease
Neurocognitive Outcomes and Quality of Life in Adults With Maple Syrup Urine Disease (MSUD)
Systemic Biomarkers of Brain Injury From Hyperammonemia
Selective Screening of Children for Hereditary Metabolic Diseases by Tandem Mass Spectrometry in Kazakhstan
Patient and Observer Reported Outcome Measurements in Inborn Errors of Metabolism
Phenylbutyrate Therapy for Maple Syrup Urine Disease