I appreciate my teams’ multiple efforts to develop novel treatment for Wolfram syndrome. A dose-escalation study for dantrolene is ongoing. We are developing AAV-based gene therapy, new drug screening methods, genome-editing-based therapy, and stem-cell therapy. At the same time, I need to secure funds for clinical trials. For each effort, there are obstacles, but I will try and try and try again to make these work.
I have three things always on my mind.
1. A cure for Wolfram syndrome
2. Provide better clinical care for Wolfram syndrome
3. Raise awareness of Wolfram syndrome
Again, for accomplishing each aim, there are many roadblocks to overcome. I sometimes want to cry out for help, but I will try and try and try again.
Thank you for your support and encouragement. I appreciate it from the bottom of my heart.