245. プロピオン酸血症 Propionic acidemia Clinical trials / Disease details


臨床試験数 : 13 薬物数 : 16 - (DrugBank : 3) / 標的遺伝子数 : 1 - 標的パスウェイ数 : 6

  
1 trial found
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1NCT04284917
(ClinicalTrials.gov)
November 28, 201924/2/2020Long-term Efficacy of Carglumic Acid in Organic Acidemia.Evaluate the Long Term Effectiveness & Safety of the Use of Carglumic Acid (Carbaglu®) in Patients With Propionic Acidemia (PA) or Methylmalonic Acidemia (MMA).Propionic Acidemia (PA) , Methylmalonic Acidemia (MMA)Drug: Carglumic Acid (Carbaglu®)National Taiwan University HospitalNULLEnrolling by invitation18 YearsN/AAll5N/ATaiwan