Collaboration between patient advocacy and industry to create a master protocol to investigate the novel therapy acetyl-L-leucine for three ultra-rare neurodegenerative diseases: Niemann-Pick type C, the GM2 gangliosides and ataxia-telangiectasia

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Bibliographic Details
Published in:Molecular genetics and metabolism 2023-02, Vol.138 (2), p.107099, Article 107099
Main Authors: Fields, Taylor, Crowe, Joslyn, Evans, Will, Greenfield, Julie, Hopkin, Justin, Thornton, Jennifer K., Lewi, Daniel, Mathieson, Toni, Pangonis, Diana
Format: Article
Language:English
Online Access:Get full text
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ISSN:1096-7192
1096-7206
DOI:10.1016/j.ymgme.2022.107099