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

<br><strong>Background<br></strong> The lack of approved treatments for the majority of rare diseases is reflective of the unique challenges of orphan drug development. Novel methodologies, including new functionally relevant endpoints, are needed to render the development pr...

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Bibliographic Details
Main Authors:	Fields, T, Patterson, M, Bremova-Ertl, 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
Format:	Journal article
Language:	English
Published:	BioMed Central 2021

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

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