Propionic Acidemia - 2 Studies Found
| Completed |
: A Retrospective Study to Characterize Participants With Propionic Acidemia : Propionic Acidemia : 2025-07-31 |
| COMPLETED |
: Long-term Efficacy of Carglumic Acid in Organic Acidemia. : Propionic Acidemia (PA) , Methylmalonic Acidemia (MMA) : 2025-07-31 : A. Study period: 36 months; treatment period: 12 months.B. Patients will receive Carglumic Acid (Carbaglu�) 50 mg/kg/day in additi |