For the past seven years, with the help of Matt’s Promise we have been funding medical research in the hope of finding a treatment that could save Charley’s life and the lives of thousands of children like him who have been struck by Duchenne muscular dystrophy. Now, we have arrived at a crucial turning point. Human clinical trials are finally underway. Additional trials will be starting in 2012 and 2013 as long as we continue to fund the development of these promising medications.

During the past year, we initiated critical work to ensure the success of these trials. We formed a clinical trial network with a cadre of Duchenne doctors who are eager to test new medicines for their patients. We began preparing for a clinical trial to test halofuginone (HT-100), a potent antifibrotic that could prevent and possibly even reverse muscle fibrosis. We committed to support the development of exon-skipping drugs so more children can get access to these promising medications on a faster timeline than would be possible without our help. We teamed up with a group of foundations to support the reformulation of a promising new molecule that needs to achieve better bioavailability.

Seven years ago, we didn’t dare dream that within such a short time frame we would have multiple clinical trials underway for kids with Duchenne. But, thanks to you, we are here. Now here’s the catch: This is the expensive part.
Human clinical testing costs millions of dollars. That’s why we are leveraging your donations by teaming up with other foundations, pharmaceutical and biotechnology companies, the federal government, even venture capital groups to make sure our contributions have the highest possible impact.

With each passing year, as we “de-risk” potential treatments for Duchenne, the odds that we will save Charley’s life get better. We have seen the landscape change before our eyes from a hazy vision in the far-off distance to a clear path right here in front of us. We will not waver from the path until we reach the end, which—for the first time in history—is clearly in sight. What we are accomplishing together is tremendous. It’s amazing. It’s historic. It’s a miracle in the making. And you are a huge part of it.

With special thanks to the supporters of Matt’s Promise,

Tracy Kramer Seckler and Benjamin Seckler, MD