Dr. Urano's Blog

Washington DC, NCATS, and World Orphan Drug Congress

I have been in Washington DC this week to visit the National Center for Advancing Translational Sciences (NCATS) of National Institutes of Health and attend the World Orphan Drug Congress.

My trip has been quite fruitful so far. I had a productive discussion with the drug development team at NCATS. We do have new drug candidates for Wolfram syndrome. We have a clear direction for the further development of these drug candidates.

I had a very nice discussion with Dr. Yaffa Rubinstein of NCATS on how to disseminate the data of patient registries and clinical studies for rare diseases.

The World Orphan Drug Congress is ongoing. I discussed our future direction with Mrs. Stephanie Snow Gebel from the Snow Foundation, representatives from multiple patient organizations and biotechs, and doctors working on rare diseases. I came up with new ideas.

Thank you for your continued support.

Warmest regards,

Fumi Urano