Substrate reduction therapy for Krabbe disease and metachromatic leukodystrophy using a novel ceramide galactosyltransferase inhibitor.

Summary
Title
Substrate reduction therapy for Krabbe disease and metachromatic leukodystrophy using a novel ceramide galactosyltransferase inhibitor.
Authors
  • Babcock MC
  • Mikulka CR
  • Wang B
  • Chandriani S
  • Chandra S
  • Xu Y
  • Webster K
  • Feng Y
  • Nelvagal HR
  • Giaramita A
  • Yip BK
  • Lo M
  • Jiang X
  • Chao Q
  • Woloszynek JC
  • Shen Y
  • Bhagwat S
  • Sands MS
  • Crawford BE
Source
Sci Rep
Publication Date
2021 Jul 14
PubMed ID
34262084

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Acknowledgements

Supported by JST NBDC Grant Number JPMJND2204

Partly supported by NIH Common Fund Grant #1U01GM125267-01


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