Real-World Experience of Carglumic Acid for Methylmalonic and Propionic Acidurias: An Interim Analysis of the Multicentre Observational PROTECT Study

ConclusionsIn this group of patients with MMA and PA, treatment with carglumic acid for at least 1 year reduced peak plasma ammonia levels in the total patient population and reduced the frequency of metabolic decompensation events, as well as the duration of inpatient stay due to metabolic decompensations in a subset of patients.Clinical Trial RegistrationClinicalTrials.gov, NCT04176523. Registered 25 November, 2019, retrospectively registered,https://clinicaltrials.gov/ct2/show/NCT04176523.
Source: Drugs in R&D - Category: Drugs & Pharmacology Source Type: research