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

Open-Label Study of Uridine Triacetate in Pediatric Patients With Hereditary Orotic Aciduria

计划入组 4 人 · 首次发布于 2014 年 4 月 10 日

glutaric aciduria type 1 therapeutic

ASL gene therapy (AAV, argininosuccinic aciduria)

Glutaric aciduria type 1(Novel Pharma)

3-D Matrix Ltd.

Japan

River 3 Renal Corp.

United States

Immuno Cure 3 Limited

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

Breakthrough Gene Editing Therapy Successfully Treats Infant with Rare Metabolic Disorder

Sanofi's Rilzabrutinib Achieves Primary Endpoint in Phase 3 ITP Trial