What is Wolfram Syndrome?
Wolfram Syndrome is an autosomal recessive genetic
disorder in that the mother and the father each pass
two copies of the gene down to the child. Wolfram
Syndrome is considered a rare disease and a icts
about 1 in 500,000 people. There are around 30,000
patients in the world who have this disease.
In early childhood, kids with Wolfram Syndrome first
develop insulin-dependent diabetes. Unlike common
types of diabetes, these children go on to develop blindness, deafness and other neurologic
disturbances. Wolfram Syndrome can also lead to loss
of sense of smell, problems with balance and
coordination, muscle spasms and seizures, urinary
tract problems, and irregular breathing.
Why Do We Need Research?
There are currently no drug therapies or cures that exist for Wolfram Syndrome. As a result, more than 60% of Wolfram patients die before age 30. It is crucial that we gain a complete understanding of the complexities of Wolfram Syndrome by performing rigorous research, which will serve as the platform to discover and clinically test successful treatment options. Researchers believe that finding a treatment and cure for Wolfram Syndrome may open doors for treating diabetes and other rare genetic diseases such as Parkinson’s and Alzheimer’s.