Product

Sodium Phenylbutyrate

1 clinical trial

1 indication

Indication

Medium-chain Acyl-CoA Dehydrogenase Deficiency

Clinical trial

A Phase 2, Open-Label, Fixed Dose Study to Evaluate the Use of Sodium Phenylbutyrate (ACER-001) in the Treatment of Pediatric and Adult Patients With MCAD Deficiency Caused by the Common ACADM c.985 A>G (K304E) Mutation

Status: Recruiting, Estimated PCD: 2025-06-01