Title : Evaluation of long-term effectiveness of the use of carglumic acid in patients with propionic acidemia (PA) or methylmalonic acidemia (MMA): study protocol for a randomized controlled trial.

Pub. Date : 2019 Jun 13

PMID : 31196016






4 Functional Relationships(s)
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1 Evaluation of long-term effectiveness of the use of carglumic acid in patients with propionic acidemia (PA) or methylmalonic acidemia (MMA): study protocol for a randomized controlled trial. carglumic acid monocyte to macrophage differentiation associated Homo sapiens
2 Here we report the rationale and design of a phase IIIb trial that is aimed at determining the long-term efficacy and safety of carglumic acid in the management of PA and MMA. carglumic acid monocyte to macrophage differentiation associated Homo sapiens
3 Although retrospective studies have suggested that long-term carglumic acid may be beneficial in the management of PA and MMA, current literature lacks evidence for this indication. carglumic acid monocyte to macrophage differentiation associated Homo sapiens
4 This clinical trial will determine the long-term safety and efficacy of carglumic acid in the management of PA and MMA. carglumic acid monocyte to macrophage differentiation associated Homo sapiens