Natural History Study of Infantile and Juvenile GM1 Gangliosidosis (GM1) Patients

计划入组 31 人 · 首次发布于 2019 年 8 月 1 日

Interviews and Video Capture in Patients With GM1 Gangliosidosis

计划入组 25 人 · 首次发布于 2020 年 3 月 17 日

A Safety and Efficacy Study of LYS-GM101 Gene Therapy in Patients With GM1 Gangliosidosis

计划入组 5 人 · 首次发布于 2020 年 2 月 18 日

chaperone therapy (oral, GM1 gangliosidosis)

GM1 gangliosidosis therapy（Sharp Therapeutics）

GLB1 gene therapy (AAV, GM1 gangliosidosis)

OM1, Inc.

United States

GMG ArcData LLC

United States

GMP Endotherapeutics Inc

In Utero Gene Therapy Trial Targets GM1 Gangliosidosis Before Birth

Azafaros' Nizubaglustat Gains Orphan Drug Designations, Phase 3 Trials to Start in 2025

Azafaros Publishes Phase 2 RAINBOW Study Data for Nizubaglustat in GM2 Gangliosidosis and Niemann-Pick Type C Disease