Long-term use of carglumic acid in methylmalonic aciduria, propionic aciduria and isovaleric aciduria in Italy: a qualitative survey

Burlina, A; Bettocchi, I; Biasucci, G; Bordugo, A; Gasperini, S; La Spina, L; Maines, E; Meli, C; Menni, F; Paci, S; Procopio, E; Rossi, A; Rubert, L; Spada, M; Tubili, F; Tummolo, A

Burlina, A (通讯作者),Univ Hosp, Dept Diagnost Serv, Div Inherited Metab Dis, Padua, Italy.

EUROPEAN REVIEW FOR MEDICAL AND PHARMACOLOGICAL SCIENCES, 2022; 26 (14): 5136

Abstract

OBJECTIVE: Organic acidurias (OAs) are a group of rare metabolic disorders that disrupt the regular amino acid metabolism. OAs are characterized by re......

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