246. メチルマロン酸血症
［臨床試験数：14，薬物数：22（DrugBank：2），標的遺伝子数：1，標的パスウェイ数：6］

Searched query = "Methylmalonic acidemia"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

Drug = 291844; ATALUREN; Ataluren; Ataluren 1000mg; Ataluren 125mg; Ataluren 250mg; Carbaglu; Carglumic Acid; Carglumic Acid (Carbaglu®); Carglumic acid; Compound 1a; HLB-001; MRNA-3704; N-Carbamylglutamate; N-carbamylglutamate; Other: Standard of Care; PTC-0161480; PTC-124; PTC-C124; PTC124; RPS 2505; Standard of Care Treatment