A Phase 2, Open-label, Fixed-dose Study to Assess the Efficacy of Sodium Phenylbutyrate (ACER-001) in Treating Pediatric and Adult Patients with Medium Chain Acyl-CoA Dehydrogenase (MCAD) Deficiency Resulting from the Prevalent ACADM C.985 A>G (K304E) Mutation
Latest Information Update: 29 Jan 2025
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At a glance
- Drugs Sodium phenylbutyrate (Primary)
- Indications Medium chain acyl CoA dehydrogenase deficiency
- Focus Adverse reactions
- 21 Jan 2025 New trial record