Key Highlights

Risk & Performance

Based on historical performance

High Risk

Score: 65/100

Failure Rate

20.0%

1 terminated/withdrawn out of 5 trials

Success Rate

80.0%

-6.5% vs industry average

Late-Stage Pipeline

0 trials in Phase 3/4

Results Transparency

50%

2 of 4 completed trials have results

Key Signals

2 with results

Phase 2

3(60.0%)

Phase 1

2(40.0%)

5Total

Phase 2(3)

Phase 1(2)

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Showing 5 of 5 trials

Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy

Role: collaborator

Study of Safety and Dosing Effect on SMN Levels of Valproic Acid (VPA) in Patients With Spinal Muscular Atrophy

Role: collaborator

Effect of Low-Dose Celecoxib on SMN2 in Patients With Spinal Muscular Atrophy

Role: collaborator

Valproic Acid in Ambulant Adults With Spinal Muscular Atrophy

Role: collaborator

CARNIVAL Type I: Valproic Acid and Carnitine in Infants With Spinal Muscular Atrophy (SMA) Type I

Role: collaborator

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