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

N-Acetyl-L-Leucine for GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease)

计划入组 30 人 · 首次发布于 2018 年 11 月 30 日

AAV gene therapy (Tay Sachs) (Amicus Therapeutics)

Madalena association

Canada Association

NATIONAL INSTITUTE OF CARDIOVASCULAR DISEASES

Bangladesh

NATIONAL CENTRE FOR INFECTIOUS DISEASES

Singapore

NATIONAL INSTITUTE OF INFECTIOUS DISEASES

Japan

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

Myrtelle's rAAV-Olig001-ASPA Gene Therapy Shows Promise in Canavan Disease Trial

BridgeBio's BBP-812 Receives FDA RMAT Designation for Canavan Disease Gene Therapy