Anaplerotic Therapy Using Triheptanoin for Patients With Glycogen Storage Disease Type I

计划入组 4 人 · 首次发布于 2018 年 9 月 11 日

Dietary Treatment Strategies and Metabolic Control in Glycogen Storage Disease Type I

计划入组 20 人 · 首次发布于 2025 年 2 月 28 日

Use of Continuous Glucose Monitoring to Evaluate Postprandial Response to Raw Cornstarch Supplementation in Adult Glycogen Storage Disease Type I

计划入组 8 人 · 首次发布于 2026 年 6 月 12 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

Rare Disease Research, LLC

Universitetet i Oslo

Norway

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

FDA Grants Accelerated Approval to Ultragenyx's GENGLYCOS, First Gene Therapy for Glycogen Storage Disease Type Ia

Pompe Disease Pipeline Shows Promise with Novel Therapies in Development