Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2

计划入组 226 人 · 首次发布于 2020 年 7 月 14 日

Pharmacokinetics and Tolerability of Zavesca® (Miglustat) In Patients With Juvenile GM2 Gangliosidosis

计划入组 5 人 · 首次发布于 2007 年 1 月 5 日

Pharmacokinetics, Safety and Tolerability of Zavesca (Miglustat) in Patients With Infantile Onset Gangliosidosis: Single and Steady State Oral Doses

计划入组 10 人 · 首次发布于 2008 年 5 月 6 日

GM2-n

GM2-KLH vaccine(NCI)

Anti-GM2 humanised antibody (Kyowa Kirin)

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

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

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