Azasugar inhibitors as pharmacological chaperones for Krabbe disease

Author:

Hill Chris H.12345,Viuff Agnete H.678,Spratley Samantha J.12345,Salamone Stéphane678,Christensen Stig H.678,Read Randy J.12345,Moriarty Nigel W.9101112,Jensen Henrik H.678,Deane Janet E.12345

Affiliation:

1. Department of Haematology

2. Cambridge Institute for Medical Research

3. University of Cambridge

4. Cambridge CB2 0XY

5. UK

6. Department of Chemistry

7. Aarhus University

8. Denmark

9. Physical Biosciences Division

10. Lawrence Berkeley National Laboratory

11. Berkeley

12. USA

Abstract

Modified azasugar molecules have been synthesized and characterized as excellent pharmacological chaperone candidates to treat the neurodegenerative disorder Krabbe disease.

Funder

Wellcome Trust

Royal Society

Lundbeckfonden

Publisher

Royal Society of Chemistry (RSC)

Subject

General Chemistry

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