You probably already know that Charley’s Fund is supporting the development of several new medicines to slow or halt Duchenne’s deadly progression. Our goal is to see a cocktail of medications become available over the next few years that will work together to manage the disease.
As we have supported and closely monitored the development of new drugs, we have recognized hurdles that slow progress. To end Duchenne, it’s not enough to fund the best research; we also have to knock down hurdles that stand in the way of efficient drug development. Hence, The Impact Initiative!This program identifies roadblocks and then forges and implements solutions. We are currently tackling two key areas: improving the gating process for determining which molecules should advance to clinical trials and validating more effective outcome measures that can better signal whether a new treatment is achieving the desired effect. These are hefty challenges. Coming up with and implementing solutions requires significant brain power and funding. We are grateful to have support from fellow nonprofits the Nash Avery Foundation, Zubin’s Wish, Team Joseph, Hope for Gus, and Michael’s Cause. If you run a nonprofit and want to get involved in the Impact Initiative, contact Laura@charleysfund.org.
Speaking of clearing hurdles that slow access to new medicines, here’s some good news that can impact patients right now: Marathon Pharmaceuticals is now making deflazacort, an alternative to prednisone, available to patients in the US through an expanded access program! Click here to learn more.