Gene Therapy for Tay-Sachs Disease

计划入组 4 人 · 首次发布于 2013 年 6 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

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

AAV gene therapy (Tay Sachs) (Amicus Therapeutics)

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

TAY-018

TAS

TAC

Canada

Day, Robert

United States

UMass Chan Gene Therapy Shows Biochemical Correction in GM2 Gangliosidosis Phase I/II Trial

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

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