EPI-743 in Cobalamin C Defect: Effects on Visual and Neurological Impairment

NCT01793090 · clinicaltrials.gov ↗

PHASE2

Phase

COMPLETED

Status

30

Enrollment

OTHER

Sponsor class

Conditions

Methylmalonic Aciduria and Homocystinuria,Cblc Type
Genetic Disease
Retinopathy

Interventions

DRUG: Epi-743
OTHER: Placebo supplementation

Sponsor

Bambino Gesù Hospital and Research Institute

Collaborators

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