Wolfram syndrome, a rare neurodegenerative disease: from pathogenesis to future treatment perspectives

Wolfram syndrome, a rare neurodegenerative disease: from pathogenesis to future treatment perspectives

Abstract Background Wolfram syndrome (WS), a rare genetic disorder, is considered the best prototype of endoplasmic reticulum (ER) diseases. Classical WS features are childhood-onset diabetes mellitus, optic atrophy, deafness, diabetes insipidus, neurological signs, and other abnormalities. Two caus...

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Bibliographic Details
Main Authors: Maria Teresa Pallotta, Giorgia Tascini, Roberta Crispoldi, Ciriana Orabona, Giada Mondanelli, Ursula Grohmann, Susanna Esposito
Format: Article
Language:English
Published: BMC 2019-07-01
Series:Journal of Translational Medicine
Subjects:
Deafness
Diabetes insipidus
Optic atrophy
Type 1 diabetes
Wolfram syndrome
WFS1
Online Access:http://link.springer.com/article/10.1186/s12967-019-1993-1

Internet

http://link.springer.com/article/10.1186/s12967-019-1993-1

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