Natural History Study Protocol in PMM2-CDG (CDG-Ia)

计划入组 139 人 · 首次发布于 2017 年 6 月 1 日

Does Chemotherapy Train the Mutation of the Pig-A Gene? (PIGA)

计划入组 30 人 · 首次发布于 2019 年 2 月 11 日

Acetazolamide Efficacy in Ataxia in PMM2-CDG

计划入组 25 人 · 首次发布于 2020 年 12 月 22 日

CGS-27023A

AVTX-802

Fucose

C₆H₁₂O₅

SIGA Technologies

India

UNIVERSITA' DI PISA

Italy

University of Pisa

Italy

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

FDA Grants Fast Track Designation to Glycomine's GLM101 for PMM2-CDG Treatment

Glycomine Secures $115M Series C Funding to Advance Novel Treatment for PMM2-CDG Rare Disease