Article

N-acetyl-L-leucine improves symptoms and functioning in GM2 Gangliosidosis (Tay-Sachs & Sandhoff)

2021-09-27

Abstract excerpt

<h4>Background and Objective</h4> GM2 gangliosidosis (Tay-Sachs and Sandhoff diseases) are rare, inherited neurodegenerative disorders with no available symptomatic or disease modifying treatments. This clinical trial aimed to investigate the safety and efficacy of N-acetyl-L-leucine (NALL) on symptoms of pediatric (≥ 6 years) and adult patients with GM2 gangliosidosis. <h4>Methods</h4> \ We conducted an 8-center,...

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Literature Corpus work: 250fc27a-4ed4-5f46-b5d8-9f424e558763
DOI: 10.1101/2021.09.24.21264020

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