Hi Everyone!
I am pumped up this morning! We have just wrapped up our two day live meeting with the principal investigators and clinical coordinators of the CRC-SCA natural history study. The pharma company members of our NAF Drug Development Collaborative were also present. This is the first time this group has been able to meet in person since 2019, and first off, it was just great to be together! Deep discussions were held regarding the current status of the study (patient recruitment, follow-up, study protocols, etc.) and how it can be even more impactful going forward. Natural history data is a critical component of drug development and regulatory submissions, and we talked about how to work most effectively with FDA going forward. While there is much work to do, the energy in the room and the unquestionable commitment to doing what’s right for the patient is contagious. Thank you to all who traveled to the Twin Cities for this important gathering. And now we turn our attention to our annual Hill Day next week where Ataxia advocates from around the country will be holding meetings with members of congress and their aides, pushing hard for increased funding for Ataxia research and other causes meaningful to our community.
So much going on at NAF, and it’s time to get back to work!
Andrew