Long-term Efficacy of Carglumic Acid in Organic Acidemia.
Status:
Enrolling by invitation
Trial end date:
2021-12-01
Target enrollment:
Participant gender:
Summary
Evaluate the Long Term Effectiveness & Safety of the use of Carglumic Acid (Carbaglu®) in
Patients with Propionic Acidemia (PA) or Methylmalonic Acidemia (MMA).