There has been so much news about Duchenne in the last month…and it’s good news. Next month will be the 10th anniversary of our son’s diagnosis of this dreadful disease. At that time, we searched the internet and found just a few research projects that were in early stages. Today, we have Glaxo Smith Kline/Prosensa with a drug in a phase III pivotal trial around the world, Sarepta Therapeutics has shown remarkable results in their phase II trial and the Duchenne community is mobilizing to address the FDA with our need for rapid approval for these potential life saving drugs.
Today, Cincinnati Children’s Medical Center has announced what is believed to be the first in the nation to have a device implanted into a Duchenne patient to help his heart pump blood to the body long-term. Cardiac failure is a leading cause of death in Duchenne…this could be a major advance, although it’s very new and we must be cautious.
CureDuchenne was early in it’s support of exon skipping, and we are actively involved in working with the biotech/pharmaceutical companies and the FDA to get these drugs to all patients as soon as possible. The news from CCMC today gives us hope that cardiac issues can be treated as we hopefully improve the skeletal muscle strength in Duchenne boys.
Now is the time to double down with our efforts to bring treatments to all of our sons. Please know that we need everyones help, cooperation and enthusiasm. Everyone has something to contribute. Finally, the science is ready for us to be bold, please help us to turn the science into treatments.