Dr. Urano's Blog

NIH’s efforts on drug development for rare diseases

I was interviewed by ABC news on my work on Wolfram syndrome and partnership with the Snow Foundation. Once this is aired, this will be a great opportunity for us to raise awareness of Wolfram syndrome.

During the interview, they asked me about NIH’s efforts on developing treatments for rare diseases. NIH is actively working on expanding their program for developing therapeutics for rare diseases. Dr. Francis Collins, Director of National Institutes of Health (NIH), is a strong¬†advocate for this initiative. Their new institute, National Center for Advancing Translational Sciences (NCATS) led by Dr. Chris Austin, is focusing on developing therapeutics for rare and neglected diseases.

I have been invited to present my drug discovery strategies for Wolfram syndrome to Dr. Austin and his team at NCATS. This is a great opportunity for our patients with Wolfram syndrome. I will do my best.

Thank you so much for your continued support and faith in me. I am grateful and hopeful. I see the light.

Kindest regards,