Recently we traveled to Washington D.C. to participate in the public RAC (RECOMBINANT DNA ADVISORY COMMITTEE) meeting on behalf of Dr. Kaspar’s Gene Therapy for SMA….We have been supporting, funding and following Dr. Kaspar’s program for just about the last 3 years. We knew this meeting would be step 1 of 3 critical steps that Gene Therapy must go through to move to clinical trials. Once we heard the date announced we quickly contacted the NIH to report that we would like to speak and present during the public comment section of the meeting…..happily we were not the only SMA Families that day…several families traveled from all over the U.S. to participate in the public comment section of the RAC. The meeting was a HUGE success!!! The RAC committee was extremely impressed with The Research teams presentation and moved to tears by the SMA Families who one by one got up and spoke about the impact SMA has had on their families….I am so proud of all the families who came out that day…Im so proud of all the families who supported this program through the years, whether you ran a race, made a bow, had a golf tournament, ran a pasta feed, had a photo shoot, organized a baseball event, made amazing jewelry, made cupcakes, or silently whispered a prayer … “Cure SMA”…it has all made a difference and brought us to this monumental day…..we are not just moving forward… but BULLDOZING FORWARD toward Human Clinical Trials!!!! I am not going to lie, these last three years have been tough, even thought about giving up …but as a Foundation and as a family this has grown beyond “Sophia’s Cure” … but a CURE FOR ALL ……an end to SMA …..for the families who have an SMA child, for the families who lost an SMA child, for the SMA families who wonder if they should have more children and expand their family …. We are in the home stretch, the hour is near………
“It’s like in the great stories, The ones that really mattered. Full of darkness and danger, they were. And sometimes you didn’t want to know the end. Because how could the end be happy? How could the world go back to the way it was when so much bad had happened? But in the end, it’s only a passing thing, this shadow. Even darkness must pass. A new day will come. And when the sun shines it will shine out the clearer. Those were the stories that stayed with you. That meant something, even if you were too small to understand why. But I think, I do understand. I know now. Folks in those stories had lots of chances of turning back, only they didn’t. They kept going. Because they were holding on to something.
…..That there’s some good in this world, ….. and it’s worth fighting for.”
To watch the family portion of the meeting click here .. https://www.youtube.com/watch?v=z1GKTwR2STg
Thank you for your love and support !!!! Wishing you all a very happy holiday season….
All our love The Gaynor Family
Vincent, Catherine, Sophia, & Jackson