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

Fields T., Patterson M., Bremova T., Belcher G., Billington I., Churchill GC., Davis W., Evans W., Flint S., Galione A., Granzer U., Greenfield J., Karl R., Kay R., Lewi D., Mathieson T., Meyer T., Pangonis D., Platt FM., Tsang L., Verburg C., Factor M., Strupp M.

DOI

10.1101/2020.03.11.20034256

Type

Preprint

Publication Date

2020

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