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

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 causative ge...

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Bibliografiske detaljer
Udgivet i:J Transl Med
Main Authors: Pallotta, Maria Teresa, Tascini, Giorgia, Crispoldi, Roberta, Orabona, Ciriana, Mondanelli, Giada, Grohmann, Ursula, Esposito, Susanna
Format: Artigo
Sprog:Inglês
Udgivet: BioMed Central 2019
Fag:
Review
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6651977/
https://ncbi.nlm.nih.gov/pubmed/31337416
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s12967-019-1993-1
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