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1 terminated/withdrawn out of 7 trials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

83.3%

-3.2% vs industry average

Late-Stage Pipeline

14%

1 trials in Phase 3/4

Results Transparency

20%

1 of 5 completed trials have results

Key Signals

1 with results

Enrollment Performance

Analytics

N/A

3(42.9%)

Phase 2

2(28.6%)

Phase 1

1(14.3%)

Phase 4

1(14.3%)

7Total

N/A(3)

Phase 2(2)

Phase 1(1)

Phase 4(1)

Activity Timeline

Global Presence

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Clinical Trials (7)

Showing 7 of 7 trials

NCT01303146Phase 2Completed

Efficacy METAZYM for the Treatment Metachromatic Leukodystrophy Treated With Hematopoietic Stem Cell Transplantation

Role: collaborator

NCT05003648Phase 4Active Not Recruiting

Treating Leg Symptoms in Women With X-linked Adrenoleukodystrophy

Role: collaborator

NCT01801709Phase 1Completed

Intracerebral Gene Therapy for Children With Early Onset Forms of Metachromatic Leukodystrophy

Role: collaborator

NCT01325025Not ApplicableCompleted

Imaging Study of the White Matter Lesions in Children With Metachromatic Leucodystrophy

Role: collaborator

NCT04687007Not ApplicableCompleted

SMART-ALD - A New Lifestyle Intervention to Improve Quality of Life in Women With X-linked Adrenoleukodystrophy (X-ALD)

Role: collaborator

NCT01594853Not ApplicableCompleted

Exercise Study of Function and Pathology for Women With X-linked Adrenoleukodystrophy

Role: collaborator

NCT00657748Phase 2Withdrawn

Lithium and Acetate for Canavan Disease

Role: collaborator

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