There are no treatments for the vast majority of rare diseases, including Wolfram syndrome. Our NIH is trying change this. National Center for Advancing Translational Sciences has established a program to accelerate the drug development process for rare diseases. I am trying to be part of it to help our patients.
http://www.ncats.nih.gov/research/rare-diseases/trnd/trnd.html
Thank you for reading this blog. I appreciate emails and phone calls from you. Thank you so much for your faith in me.
Take care,
Fumi Urano