Organization
Jerry Vockley, MD, PhD
2 clinical trials
Clinical trial
A Phase II, Escalating Dose, Open Label Study to Evaluate the Safety of Triheptanoin for the Prevention of Hypoglycemia in Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)Status: Recruiting, Estimated PCD: 2025-01-01
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) MutationStatus: Recruiting, Estimated PCD: 2025-06-01