Clinical Trial Update: Efficacy and Safety of the Treatment of Pyruvate Dehydrogenase Deficiency Patients With Glycerol Phenylbutyrate (RAVICTI) [Phase 2]

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Summary

Phase 2 trial evaluating the efficacy and safety of glycerol phenylbutyrate (RAVICTI) in patients with Pyruvate Dehydrogenase Deficiency; currently recruiting.

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Diseases / indications

• Pyruvate Dehydrogenase Deficiency

Drugs mentioned

• Ravicti (glycerol phenylbutyrate)

Snippet

Sponsor: Assistance Publique - Hôpitaux de Paris | Conditions: Pyruvate Dehydrogenase Complex Deficiency Disease | Phase 2 | Status: RECRUITING