NGLY1 Deficiency: A Prospective Natural History Study

计划入组 29 人 · 首次发布于 2019 年 2 月 8 日

Safety and Efficacy of GS-100 Gene Therapy in Patients With NGLY1 Deficiency

计划入组 10 人 · 首次发布于 2024 年 1 月 10 日

NGLY1 Natural History

计划入组 15 人 · 首次发布于 2023 年 11 月 8 日

NGLY1 Collaboration (Travere Therapeutics)

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

WWOX gene therapy(Mahzi Therapeutics/ Hebrew University)

Rockwell Medical

United States

Ai-Media Technologies Ltd

United States

FDA's START Program Selects First Rare Disease Therapies for Accelerated Development

Drug repurposing screen identifies dopamine signaling and COX inhibition as candidate therapeutic pathways for PIGA-CDG

FDA Approves Kebilidi, First Gene Therapy for AADC Deficiency