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Case series reveals poor prognosis and treatment strategies in Wolfram Syndrome, suggesting need for early intervention.
Wolfram Syndrome (WS), also known as DIDMOAD (Diabetes Insipidus, Diabetes Mellitus, Optic Atrophy, and Deafness), is a rare neurodegenerative disorder [1, 2]. This condition features symptoms that typically appear progressively from childhood through adulthood [3, 4, 5]. Diagnosis is based on specific clinical criteria and confirmed by genetic testing for mutations in the WFS1 gene [6, 7]. Insulin therapy remains the mainstay treatment for diabetes mellitus, but incretin analogs may provide benefit if initiated early [8, 9]. Prognosis is generally poor, with a mean life expectancy of 39 years, often due to central respiratory complications [3, 10]. Gene therapyiscurrentlyexperimental but promising [11].
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Gorgi et al. (2025) studied this question.
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