Compassionate Use of Triheptanoin (C7) for Inherited Disorders of Energy Metabolism

首次发布于 2011 年 10 月 28 日

Study of Glycogen Storage Disease and Associated Disorders

计划入组 20 人 · 首次发布于 2002 年 12 月 10 日

Glycogen Storage Disease Breath Test Study

计划入组 14 人 · 首次发布于 2017 年 7 月 17 日

glycogen synthase inhibitors (glycogen storage diseases)

Glycogen Storage Disease 1b (Prime Medicine)

mini-GDE gene therapy (AAV9, glycogen storage disease type III)

Self Storage Group Inc

United States

Institute For Neurodegenerative Disorders

United States

Ascendis Pharma Growth Disorders A/S

Denmark

Glycogen Storage Disorders: Competitive Landscape, Pipeline Assessment, and Market Dynamics

Optoacoustic Imaging Shows Promise for Non-Invasive Pompe Disease Monitoring

Ultragenyx Completes BLA Submission for First Gene Therapy Targeting Glycogen Storage Disease Type Ia