My highest priority right now is to repurpose existing drugs for treating our patients with Wolfram syndrome. In parallel, we need a marker that can be easily monitored every week. This marker will tell us if the treatment is working. We also need an assay to monitor the levels of each marker. My team has been developing these markers and assays. These will help us determine the dose of each drug that we prescribe for each patient. Most likely, this step can be achieved by sequencing each patient’s DNA. “Precision Medicine in Wolfram syndrome” is our goal.
Thank you for reading this blog. I hope you have a wonderful weekend. Things are going into the right direction.