Article

A Phase 1b/2a Clinical Trial of Dantrolene Sodium in Patients with Wolfram Syndrome

2020-10-12

Abstract excerpt

<h4>Background</h4> Wolfram syndrome is a rare endoplasmic reticulum disorder characterized by insulin-dependent diabetes mellitus, optic nerve atrophy, and progressive neurodegeneration. Although there is currently no treatment to delay, halt, or reverse the progression of Wolfram syndrome, preclinical studies in cell and rodent models suggest that therapeutic strategies targeting endoplasmic reticulum calcium ho...

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Literature Corpus work: 3c5f20ef-51b0-5e4e-9370-be4e05ec5714
DOI: 10.1101/2020.10.07.20208694

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