Long-Term Follow-Up of Subjects Treated With AXO-AAV-GM2 for Tay-Sachs or Sandhoff Disease

计划入组 7 人 · 首次发布于 2024 年 9 月 26 日

A Dose-escalation and Safety & Efficacy Study of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease

计划入组 9 人 · 首次发布于 2020 年 12 月 17 日

Biomarker for Gangliosidosis: BioGM1/BioGM2 (BioGM1/GM2)

首次发布于 2014 年 11 月 24 日

GM2-n

GM2-KLH vaccine(NCI)

ALS-AAV9

GMG ArcData LLC

United States

GMP Endotherapeutics Inc

Parsolex Gmp Center, Inc.

United States

Andelyn Biosciences Partners with Queen's University to Manufacture AAV9-GM2 Gene Therapy for GM2 Gangliosidoses

MEDIPAL Holdings and JCR Pharmaceuticals Partner on Novel GM2 Gangliosidosis Therapy JR-479

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