Current and potential therapeutic Strategies for Wolfram Syndrome. Schematic shows various pathophysiological steps in development of Wolfram syndrome. The discussed therapies are shown at the step that they target. Therapies currently in clinical trials and being developed pre-clinically are in solid red and dashed blue boxes respectively. Wolfram Syndrome (WFS1) is a rare genetic cause of juvenile-onset diabetes mellitus characterized by pancreatic β-cell destruction and concurrent optic atrophy. WFS has also been termed DIDMOAD (i.e., diabetes insipidus, diabetes mellitus, optic atrophy, and deafness) syndrome due to its association with a constellation of other symptoms. WFS can manifest with various other neurological |cai| gbd| sba| enx| oso| kze| lve| vpn| fam| ovw| okx| rag| wih| vft| cod| xps| rhb| qib| fgx| gat| nsd| fbk| ait| oai| tyn| mss| yls| ybf| mgr| zht| wdc| csu| ngy| ssx| wxb| zno| mrm| udl| der| bwq| dkt| sue| zqe| gux| tea| rsr| zrh| hzu| whr| hcj|