A Study to Evaluate the Tolerability, Safety and Efficacy of VGM-R02b

计划入组 12 人 · 首次发布于 2024 年 1 月 23 日

Nitric Oxide Supplementation in Argininosuccinic Aciduria

计划入组 12 人 · 首次发布于 2014 年 9 月 30 日

Combined Malonic and Methylmalonic Aciduria (CMAMMA): Gene Identification and Outcome Study

计划入组 6 人 · 首次发布于 2011 年 2 月 3 日

glutaric aciduria type 1 therapeutic

Glutaric aciduria type 1(Novel Pharma)

GCDH gene therapy (AAV9, glutaric aciduria)

Breakthrough Gene Editing Therapy Successfully Treats Infant with Rare Metabolic Disorder

Genetic Study Reveals Elevated Risk of Rare Inherited Diseases in Scottish Traveller Founder Population

Hypoxia Therapy Dramatically Extends Lifespan in Mouse Model of HTRA2-Linked Mitochondrial Disease