Carglumic Acid in Methylmalonic Acidemia and Propionic Acidemia

计划入组 33 人 · 首次发布于 2015 年 4 月 27 日

Anaplerotic Therapy in Propionic Acidemia

计划入组 3 人 · 首次发布于 2008 年 3 月 28 日

Natural History Study of Patients With Methylmalonic Acidemia and Propionic Acidemia

计划入组 11 人 · 首次发布于 2022 年 6 月 30 日

PCCA gene therapy (AAV, propionic acidemia)

Methylmalonic acidemia(Genespire)

Propanoic acid

C₃H₆O₂

Foundation Medicine, Inc.

United States

VCS FOUNDATION LTD

Australia

FLINDERS FOUNDATION

Australia

NIH Awards $15.6 Million to Advance Research on Rare Bone and Metabolic Disorders

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