Article
Collaboration for rare diabetes: understanding new treatment options for Wolfram syndrome.
Endocrine - 1 Mar 2021
Reschke Felix, Rohayem Julia, Maffei Pietro, Dassie Francesca, Schwandt Anke, de Beaufort Carine, Toni Sonia, Szypowska Agnieszka, Cardona-Hernandez Roque, Datz Nicolin, Klee Katharina, Danne Thomas
Abstract excerpt
BACKGROUND: Wolfram Syndrome is a very rare genetic disease causing diabetes mellitus, blindness, deafness, diabetes insipidus, and progressive brainstem degeneration. Neurologic symptoms of affected patients include ataxia, sleep apnea, loss of bladder control, dysphagia, loss of taste, and accompanying psychiatric symptoms as a sign of progressive neurodegeneration. Its genetic cause is mainly biallelic...
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