Nice to see you again. Thank you so much for following my mission & vision and being the kindest person. I think about patients with Wolfram syndrome and their families and friends every morning. That’s one of the first things I do every day at 4:30 am. I would like to know their challenges and help them overcome these challenges. Three things are always on my mind: 1. Improve Clinical Care, 2. Raise Awareness, and 3. Provide a Cure. We are clearly making progress in #1 and #2. How about #3? Is it possible? If so, how long?
Here is my answer. “We are making progress. We are this close.”
This close = my head size. I have all the strategies and ideas for developing cutting-edge treatments for Wolfram syndrome in my head. My challenge is to realize these ideas. There are technical roadblocks for developing gene therapy. There are regulatory issues to bring new drugs from bench (lab) to bedside (patients). There are financial constraints. These are not so easy to overcome, but these are much smaller challenges than those our patients have been experiencing. I would like to articulate my strategies again. There are three steps. Step 1: Drug Therapy for halting progression. Step 2: Regenerative therapy for protecting and regrow remaining eye and brain cells. Step 3: Gene therapy for replacing pathogenic genes.
As always, please feel free to contact me with any questions or concerns (firstname.lastname@example.org). I would like to know what you think and how you feel. Thank you again for your support. Our potential is limitless. We have superpower to overcome this challenge.