A Promising Small Molecule for Vanishing White Matter Disease

Divakar S Mithal; Jennifer P Rubin

doi:10.15844/pedneurbriefs-32-5

A Promising Small Molecule for Vanishing White Matter Disease

Pediatric Neurology Briefs

Volume 32 (2018): Issue 0

By: Divakar S Mithal and Jennifer P Rubin

Open Access

|Aug 2018

Abstract

Investigators from Calico Life Sciences LLC and AbbVie report the effects of a novel drug targeting the genetic basis of Vanishing White Matter Disease (VWMD).

References

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Wong YL LeBon L Edalji R Lim HB Sun C Sidrauski C The small molecule ISRIB rescues the stability and activity of Vanishing White Matter Disease eIF2B mutant complexes eLife 2018 Feb 7 e32733 https://doi.org/10.7554/eLife.32733 29489452
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van der Knaap MS Barth PG Gabreëls FJ Franzoni E Begeer JH Stroink H A new leukoencephalopathy with vanishing white matter Neurology 1997 Apr 48 4 845 55 https://doi.org/10.1212/WNL.48.4.845 9109866
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Articles in this issue

DOI: https://doi.org/10.15844/pedneurbriefs-32-5 | Journal eISSN: 2166-6482

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Language: English

Page range: 5 - 5

Submitted on: May 25, 2018

Accepted on: Jun 5, 2018

Published on: Aug 28, 2018

Published by: Pediatric Neurology Briefs Publishers

In partnership with: Paradigm Publishing Services

Keywords:

Vanishing White Matter Disease,

Leukoencephalopathy,

Leukodystrophy,

Integrated Stress Response,

Eif2b,

ISRIB

© 2018 Divakar S Mithal, Jennifer P Rubin, published by Pediatric Neurology Briefs Publishers
This work is licensed under the Creative Commons Attribution 4.0 License.

Volume 32 (2018): Issue 0