Gene Therapy for Tay-Sachs Disease

计划入组 4 人 · 首次发布于 2013 年 6 月 5 日

A Natural History of Late Onset Tay-Sachs Disease

计划入组 10 人 · 首次发布于 2016 年 8 月 2 日

A Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, and Safety of Venglustat in Late-onset GM2

计划入组 75 人 · 首次发布于 2020 年 1 月 9 日

AAV9-HEXA/HEXB gene therapy (intravenous, Tay Sachs disease/Sandhoff disease)

AAV gene therapy (Tay Sachs) (Amicus Therapeutics)

TAY-R1

Rare Disease Research, LLC

CURE RARE DISEASE, INC.

United States

CASTLEMAN DISEASE COLLABORATIVE NETWORK

United States

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

Sanofi Pivots R&D Strategy, Discontinues Diabetes Research to Focus on Novel Therapeutics

Azafaros to Present Phase 3 Nizubaglustat Data for Rare Lysosomal Storage Disorders at WORLDSymposium 2026