Lysosomal Storage Diseases
20
2
5
6
Key Insights
Highlights
Success Rate
60% trial completion
Clinical Risk Assessment
Based on trial outcomes
High Risk
Score: 85/100
20.0%
4 terminated out of 20 trials
60.0%
-26.5% vs benchmark
5%
1 trials in Phase 3/4
0%
0 of 6 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 6 completed trials
Clinical Trials (20)
Longitudinal Study of Neurodegenerative Disorders
Identification and Characterization of Novel Non-Coding Variants That Contribute to Genetic Disorders
Stem Cell Gene Therapy for Cystinosis
A Pilot Study for Systematic Neonatal Screening for Lysosomal Storage Diseases Using Tandem Mass Spectrometry
OTL-200 in Patients With Late Juvenile Metachromatic Leukodystrophy (MLD)
A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)
Fabry Disease in High-risk Patients With Left Ventricular Hypertrophy: Prevalence and Implementation of a Clinical Score
A Long Term Follow-Up Study of Fabry Disease Subjects Treated With FLT190
Nutritional Therapy in Late-onset Pompe Disease
A Fabry Disease Gene Therapy Study
Neutralizing Antibody Seroprevalence Study With a Retrospective Component in Participants With Late-Onset Pompe Disease
A Natural History Study of Aspartylglucosaminuria
Complement Activation in the Lysosomal Storage Disorders
Lyso-Gb1 as a Long-term Prognostic Biomarker in Gaucher Disease
Diet and Exercise in Pompe Disease
Natural History Study of Children With Metachromatic Leukodystrophy
Cellular Pharmacodynamics of Small Molecules in Lysosomal Storage Disorders
Screening of Lysosomal Storage Disorders Diseases in Minority Groups
Fetal Umbilical Cord Blood (UCB) Transplant for Lysosomal Storage Diseases
Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis (MPS) I