Methylmalonic Acidemia - 2 Studies Found
| Completed |
: Selective Screening of Children for Hereditary Metabolic Diseases by Tandem Mass Spectrometry in Kazakhstan : : 2025-08-02 : Neonatal blood samples will be taken from healthy infants no earlier than 3 hours after feeding by heel prick with a heel stick.Five drops of wh |
| COMPLETED |
: Long-term Efficacy of Carglumic Acid in Organic Acidemia. : Propionic Acidemia (PA) , Methylmalonic Acidemia (MMA) : 2025-08-02 : A. Study period: 36 months; treatment period: 12 months.B. Patients will receive Carglumic Acid (Carbaglu�) 50 mg/kg/day in additi |