Anaplerotic Therapy in Propionic Acidemia

计划入组 3 人 · 首次发布于 2008 年 3 月 28 日

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

AAVrh10-PCCA Gene Therapy for Propionic Acidemia

计划入组 9 人 · 首次发布于 2026 年 6 月 12 日

PCCA gene therapy (AAV, propionic acidemia)

Methylmalonic acidemia(Genespire)

Propanoic acid

C₃H₆O₂

ACADEMIA CEARENSE DE ODONTOLOGIA

Brazil

ACADEMIA NACIONAL DE MEDICINA

Brazil

Ono Academic College

Israel

Advancing Rare Disease Research: How mRNA Technology and Synthetic Control Arms Are Transforming Clinical Trials

FDA Grants Rare Pediatric Disease and Orphan Drug Designations to Six Gene Therapies

Moderna Restructures Leadership, Appoints New CCO Ahead of Up to Three Product Launches in 2027–2028