Key Highlights

Risk & Performance

Pipeline Risk Assessment

Pipeline Risk Assessment

Based on historical performance

High Risk

Score: 90/100

Failure Rate

33.3%

1 terminated/withdrawn out of 3 trials

Success Rate

66.7%

-19.8% vs industry average

Late-Stage Pipeline

0%

0 trials in Phase 3/4

Results Transparency

0%

0 of 2 completed trials have results

Key Signals

Enrollment Performance

Analytics

Phase 1
1(100.0%)
1Total
Phase 1(1)

Activity Timeline

Global Presence

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Clinical Trials (3)

Showing 3 of 3 trials
NCT02898753Phase 1Terminated

VAL-1221 Delivered Intravenously in Ambulatory and Ventilator-free Participants With Late-Onset Pompe Disease

Role: lead

NCT02057705Completed

Prospective, Longitudinal Study of the Natural History and Functional Status of Patients With Myotubular Myopathy (MTM)

Role: lead

NCT01817946Completed

Myotubular Myopathy Genetic Testing Study

Role: collaborator

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