Key Highlights

Risk & Performance

Pipeline Risk Assessment

Based on historical performance

High Risk

Score: 60/100

Failure Rate

14.3%

1 terminated/withdrawn out of 7 trials

Success Rate

75.0%

-11.5% vs industry average

Late-Stage Pipeline

14%

1 trials in Phase 3/4

Results Transparency

33%

1 of 3 completed trials have results

Key Signals

2 recruiting1 with results

Enrollment Performance

Analytics

Phase 2

1(50.0%)

Phase 4

1(50.0%)

2Total

Phase 2(1)

Phase 4(1)

Activity Timeline

Global Presence

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Clinical Trials (7)

Showing 7 of 7 trials

NCT00668187Recruiting

A Natural History Study of the Gangliosidoses

Role: collaborator

NCT02583672Phase 2Completed

Role of Oxidative Stress and Inflammation in Type 1 Gaucher Disease (GD1)

Role: collaborator

NCT02993796Recruiting

Krabbe Disease Global Patient Registry

Role: collaborator

NCT02030015Phase 4Terminated

Synergistic Enteral Regimen for Treatment of the Gangliosidoses

Role: collaborator

NCT03576729Completed

MRS to Determine Neuroinflammation and Oxidative Stress in MPS I

Role: collaborator

NCT01870375Completed

Longitudinal Studies of Brain Structure and Function in MPS Disorders

Role: collaborator

NCT02994303Unknown

Podocyturia - Predictor of Renal Dysfunction in Fabry Nephropathy

Role: collaborator

All 7 trials loaded