Thank you Ace.
Race to Yes Update: March Madness Begins!
It’s been one year since 106,000+ people signed The Race to Yes petition urging the FDA to implement a faster and more efficient regulatory approach when evaluating new drugs for Duchenne. The White House responded with a statement confirming its commitment to faster drug approvals for this rare and fatal disease, but too many kids with Duchenne are still waiting. We need to let the FDA and Congress know that we will not turn down the heat until we are satisfied with their level of urgency. Please share this infographic with everyone you know. Let’s let the FDA and the drug companies know that our community is 106,000 strong and growing and that we are watching what they do!
Speaking of watching, we’re excited to let you know that To the Edge of the Sky, a documentary film chronicling our fight to get new treatments for kids with Duchenne, is a wrap! To raise the money needed to complete the editing and release the film, a kickstarter campaign will launch on March 25th. Stay tuned for details.
Matt’s Promise is dedicated to making a difference in the lives of young people affected by terminal illnesses. Our commitment, through select funding of treatment, research, education, and support programs, is to advance the fight against these diseases and to assure the most comprehensive, innovative, and compassionate care.
In the Lab:
Charley’s Skin Cells Get to Work
Would you believe that Charley’s skin cells are participating in their own experiment? That’s what’s happening right now in a genetics lab at Stanford University! Dr. Michele Calos is using the skin cells of boys with Duchenne to test a cutting edge therapeutic strategy. As you read this, the skin cells are being converted into iPSCs (induced pluripotent stem cells), a type of stem cell with the potential to be grown into a muscle cell. The IPSCs are then “corrected” to fix the mutation that causes Duchenne and converted into muscle cells producing healthy dystrophin. The goal is ultimately to return those healthy cells to boys with Duchenne. Co-funding this exciting research with us is the Nash Avery Foundation. (continues…)