Clinical Trial Update: EPI-743 in Cobalamin C Defect: Effects on Visual and Neurological Impairment [Phase 2]

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Summary

Phase 2 trial of EPI-743 in cblC-type methylmalonic acidemia with homocystinuria evaluated effects on visual and neurologic impairment; the study has completed.

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Diseases / indications

• Methylmalonic Acidemia with Homocystinuria Cblc Type
• Retinopathy

Drugs mentioned

• EPI-743

Snippet

Sponsor: Bambino Gesù Hospital and Research Institute | Conditions: Methylmalonic Aciduria and Homocystinuria,Cblc Type, Genetic Disease | Phase 2 | Status: COMPLETED