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ArticleTrials2021

A master protocol to investigate a novel therapy acetyl-L-leucine for three ultra-rare neurodegenerative diseases: Niemann-Pick type C, the GM2 gangliosidoses, and ataxia telangiectasia.

T Fields et al.PubMed ↗Full text ↗Publisher ↗

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20 papers cite it

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this papertrialcites it

3 trials

Full record →Abstract, authors, funding and every citing paper · PMID 33482890