During this pandemic when the world is so uncertain, Walking Strong has not wavered in helping numerous families that have come to us for assistance as well as donate much-needed funds to our clinic, The Center For Duchenne Muscular Dystrophy at UCLA (CDMD).
Helping families and funding research is what keeps us going and continuing with fundraising. We are so grateful that through our own Duchenne journey, we have been guided on this path to serve others who walk in our shoes, and hopefully change thousands of lives.
Due to the incredible success of our 5th Annual “Night of Strength Gala” last November, Walking Strong has donated $75,000 to the Center For Duchenne Muscular Dystrophy at UCLA to further Duchenne research, education, and supporting clinical trials.
Despite the social distancing, the CDMD has been able to further their work in Duchenne Research and gear up to host the first-ever Gene Therapy trials at UCLA. Alexander is being tested to see if he qualifies for one of these trials that is now expanding into non-ambulatory, older boys. I write this as we are at UCLA getting testing done, which is the first step in assessing his ability to qualify for this clinical trial. Gene Therapy is the ground-breaking therapy that will work to correct the genetic mutation that causes Duchenne. These trials have been underway for the last two years on younger boys, which has proven to be quite effective in gaining physical strength and overall well-being. This is the first time in history that there is hope for a gene-correction and meaningful treatment in Duchenne. The Duchenne Community lies all their hope in Gene Therapy at this time, and Walking Strong is proud to provide funding to assist in its success and offer it to patients.
The CDMD is a world-class multi-disciplinary clinic that is hosting the first-ever Gene Therapy trials by Pfizer and Sarepta, working towards solving the immune response to new therapies, dystrophin quantification, alternate protein therapies, multiple exon-skipping, and exon skipping enhancers, genetic modifiers/outliers for precision medicine, exploration of repurposing approved FDA drugs for the heart, lungs, and skeletal muscle among others, as well providing education to medical students and the community.
Jorge and I are so grateful for all YOUR support, and through your donations, we can support life-saving therapies, change the lives of Duchenne Families through our Assistance Program and create awareness for Duchenne.
YOU KEEP US WALKING STRONG!