Rare diseases may start from relatively common symptoms, such as diabetes. In rare diseases, another symptom (or symptoms) which seems unrelated often starts to manifest later. In the case of Wolfram, this is optic nerve atrophy, diabetes insipidus, or hearing impairment. These symptoms seem unrelated, but they are related. The same molecular wiring altered in Wolfram causes different problems in different organs. This may sound complicated, but I see this as an opportunity. In theory, a drug that can correct the abnormal molecular wiring in one organ should be able to correct molecular wiring in other organs. This is the reason we need to dissect out the molecular wiring in Wolfram syndrome.
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