My current focus is to delay the progression of Wolfram syndrome using small compounds (i.e., pills), especially repurposing currently available FDA-approved drugs. I am aware that this is not good enough because many of our patients have lost tissues producing insulin, maintaining brain functions, and preserving visual acuity.
In collaboration with biomedical engineers and biotechs, I have been developing therapeutics restoring insulin-producing cells and cells transmitting visual images. I plan to use skin cells from patients and a trophic factor produced in our brain cells. I will articulate my strategy for lay audience on September 17th. The title of the event is “REGENERATIVE MEDICINE 101.” I look forward to seeing you there. I plan to post my presentation through our website. We need to RESTORE and REBUILD functional tissues.
Thank you for reading this blog. I hope you have a wonderful Sunday.