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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...
| Autores principales: | , , , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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BioMed Central
2019
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6651977/ https://www.ncbi.nlm.nih.gov/pubmed/31337416 http://dx.doi.org/10.1186/s12967-019-1993-1 |