Thank you for visiting our Wolfram Syndrome website. I hope you and your family are safe, strong, and healthy on a brighter future. I have no doubt better days are ahead, and we can rise about this crisis. Many of you have asked me if I need to assist with COVID-19 research or help clinics related to COVID-19. At the moment, I am solely working on Wolfram syndrome and don’t need to help clinics related to COVID-19. So please don’t worry. I am your Wolframdoc.
My three guiding principles are: Improve clinical care, Raise awareness, and Provide a cutting-edge treatment for Wolfram syndrome. I want to update you on a few things.
We have analyzed the data of our clinical trial of dantrolene sodium in patients with Wolfram syndrome. We plan to make the data publicly available very soon. It takes time to formally publish the data in a scientific journal. Therefore, we are looking into other options. Based on the data we have, it seems like there are patients who respond to dantrolene and don’t respond to dantrolene sodium. My impression is that we need a new medication that can more efficiently target endoplasmic reticulum stress for the treatment of Wolfram syndrome. We are working on it.
My current focus is to develop gene therapy for Wolfram syndrome. Our ultimate goal is to provide a cure using regenerative gene therapy. We have been trying to improve diabetes, visual acuity, and brain functions using viral vectors of a healthy Wolfram gene and a regenerative factor called MANF in mouse models. We are getting encouraging preliminary results and have published two articles recently. We are currently testing two ways to deliver genes through intravitreal (for optic nerve) and retro-orbital (for brain) injections.
As always, please feel free to contact me with any questions (firstname.lastname@example.org). I would like to know what you think and how you feel. Thank you again for your continued support and encouragement. We will go through this challenging period with unusual optimism and courage. We will continue working as one team and change history together.
Happy 4th of July!
Fumi Urano, MD, PhD
July 2, 2020