Glycogen Storage Disease
18
4
6
10
Key Insights
Highlights
Success Rate
91% trial completion (above average)
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 50/100
5.6%
1 terminated out of 18 trials
90.9%
+4.4% vs benchmark
0%
0 trials in Phase 3/4
0%
0 of 10 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 10 completed trials
Clinical Trials (18)
Assessment of Safety and Acute Effects of a Lower-limb Powered Dermoskeleton in Patients With Neuromuscular Disorders
Assessment of Safety and Acute Effects of a Knee-hip Powered Soft Exoskeleton in Patients With Neuromuscular Disorders
Precision Diagnosis and Risk Stratification of Rare Cardiomyopathies Based on Novel Cardiac Magnetic Resonance Techniques
Identification and Characterization of Novel Non-Coding Variants That Contribute to Genetic Disorders
Rare Glycogen Storage Diseases Natural History Study
A Study of mRNA-3745 in Adult and Pediatric Participants With Glycogen Storage Disease Type 1a (GSD1a)
Baby Detect : Genomic Newborn Screening
Carbon-13 Magnetic Resonance Spectroscopy in Glycogen Storage Diseases
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Characterization of Patients With Cardiomyopathy to Identify Critical Patients Candidates for Cardiac Transplantation
Pompe Lactation Sub-Registry
Glycosade v UCCS in the Dietary Management of Hepatic GSD
Biomarker for Glycogen Storage Diseases (BioGlycogen)
McArdle Disease Treatment by Ketogenic Diet
Lingual Muscle Training in Late-Onset Pompe Disease (LOPD)
Study of Glycogen Storage Disease Expression in Carriers
Clinical Evaluation of a Non-Invasive Hypoglycemia Detector in a Glycogen Storage Disease Population
Study of Glycogen Storage Disease and Associated Disorders