Harmonic Ratio in Patients With GLUT1 Deficiency Syndrome

计划入组 32 人 · 首次发布于 2023 年 6 月 5 日

Red Blood Cell Exchange Transfusion as a Novel Treatment for GLUT1 Deficiency Syndrome

计划入组 12 人 · 首次发布于 2019 年 10 月 24 日

Treatment With UX007 for a Single Patient With GLUT1 Deficiency Syndrome

首次发布于 2016 年 11 月 21 日

Leigh syndrome therapeutic(Leigh syndrome)

CDKL5 deficiency disorder therapeutic program (Vyant Bio Inc/Cyclica Inc)

GLUT1 modulators(The Second Military Medical University)

ALPORT SYNDROME FOUNDATION

United States

International Rett Syndrome Foundation

United States

RETT SYNDROME RESEARCH TRUST

United States

Nature Reviews Drug Discovery Review Positions SLC Transporters as Untapped Frontier in CNS Drug Development

UCB's Fenfluramine Achieves Positive Phase 3 Results in Ultra-Rare CDKL5 Deficiency Disorder

FDA Approves Kebilidi, First Gene Therapy for AADC Deficiency