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Methylmalonic Acidemia clinical trials

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NCT ID: NCT05842837 Enrolling by invitation - Propionic Acidemia Clinical Trials

A Non-Interventional Post-Authorization Study of Carbaglu for the Treatment of Hyperammonemia Due to MMA and PA

Start date: September 15, 2022
Phase:
Study type: Observational

The objective of this study is to obtain short- and long-term clinical safety data in pediatric and adult patients with PA and MMA treated with Carbaglu, including pregnancy and fetal outcomes. This is an observational/non-interventional study. Patients will be treated per the prescribing information and routine medical practice. Only available data will be collected as part of the study.