Wolfram syndrome is characterized by juvenile-onset diabetes and optic nerve atrophy. Many patients experience diabetes insipidus, hearing impairment, and ataxia due to neurodegeneration. You can find these symptoms in a text book or review articles.
There are other common symptoms that have not been extensively studied. Here is the list of symptoms based on our database. More than 10% of patients experience the following. You will find how difficult this disorder is. There is an urgent need to develop therapeutics for Wolfram syndrome. We need drugs and stem cell-based therapy in combination with gene therapy.
Neurogenic Bladder: 25.00%
Problems Regulating Temperature: 22.50%
Bladder Incontinence: 17.50%
Dizziness When Standing Up: 15.00%
Postural Instability (this is related to ataxia, but just in case): 12.50%
Constipation/Fecal Impactation: 12.50%
Panic Attacks: 10.00%
Weight Loss: 10.00%
Excessive Sweating : 10.00%
I would like to donate the entire data in our registry database to the patient organizations. I feel patients should own the data collected from patients.
Although these symptoms sound awful, I am still hopeful. We are making steady progress. I believe in the power of genetic medicine. I always appreciate support from our patients and families, patient organizations, our colleagues in St. Louis, US, and Europe. I feel grateful. Thank you, Thank you, Thank you.