The above story appeared earlier this week on The Today Show, which has helped raise awareness of many families battling rare diseases including Sanfilippo.
Foundation President Glenn O’Neill shared the clip on Facebook with the following message:
People we know! Check out this inspiring story of a child (now adult) with a related MPS disease (MPS I, Hurler-Sheie) on The Today Show!
Check out this video story and the new book, Saving Ryan.
This is a success story. This is what we hope, thanks to the thousands of generous people who have supported our families and this cause, can be achieved in Sanfilippo Syndrome. Where children get to thrive into their teen and adult years.
As many of us know all too well, not all stories have such positive outcomes. This type of success doesn’t happen overnight. It happens because of years of research, science, clinical trials, and tests. It happens because of sacrifice. It happens because of learnings taken from the setbacks and challenges. We honor and remember children and families with Sanfilippo who have been part of helping advance the understanding by participating in clinical trials, and even more heartbreaking those who never got a chance to participate in a trial. Stories like this remind us why the work being done in Sanfilippo is so important. One day … one day.
Congratulations to Ryan Dant, Dad Mark Dant & family, and Dr. Emil Kakkis for this coverage and amazing story! These three rare disease champions want nothing more than to see these types of success stories for all MPS disorders (including Sanfilippo Syndrome, MPS III), and we are so fortunate to have them in our corner.
Dr. Kakkis leads Ultragenyx, which acquired global rights to AAV Gene Therapy ABO-102 for Sanfilippo Syndrome Type A (MPS IIIA) from Abeona Therapeutics earlier this year.