June 2, 2004
Question from Istanbul, Turkey, USA:
Both of my cousins, a boy, age 12, and a girl, age nine, have type 1 diabetes. Both got this after they became three years old. The boy has had optic atrophy for more than five years and started having urinary problems two years ago. The girl didn't have these problems, but lately she is losing sight and, after research, the doctors said that both children have Wolfram's syndrome. Has there been any research into a cure for this syndrome? If yes, how far are they and is there any hope in the near future that a cure is possible? The two also have a younger sister, age 8, and a brother, age 6. What is the risk now, and in the future, that they will have the same disease?
Wolfram is a rare syndrome including diabetes insipidus, diabetes mellitus, optic atrophy and nerve deafness. Not all parts of the syndrome have to be present. We currently believe this is a genetically determined problem and so, it is commonly seen in family clusters. There is no known cure, but treatment for the diabetes insipidus with pituitary hormone preparations is available although expensive. Treatment for the diabetes mellitus is the same as other types of insulin dependent diabetes. Optic atrophy is not treatable. Deafness sometimes responds to hearing aids, but this depends upon the degree of deafness present. There has been some research work on Wolfram’s syndrome and specific information is available using PubMed search to review this research literature.