While we aggressively advocate for the approval of eteplirsen, we remain active on other medical research fronts. It’s important to remember that eteplirsen helps 13% of kids with Duchenne, Charley not included. And it is only the first of many treatments needed to target all of Duchenne’s symptoms. More drugs are needed before we can call our job done. Our goal is to see a cocktail of medications become available over the next few years that will work together to manage the disease and allow kids like Charley to live long, productive lives. Eteplirsen will be a crucial first step, but significant work remains even if May 26th brings a yes for this first safe and effective new treatment.
Regenerative medicine is one area of our focus. We’re exploring brand new approaches — like CRISPR-Cas9 — that hold tremendous promise for the future. We’re also working on treatment approaches that might not be “sexy” enough for coverage in the New York Times but have a good chance of getting into clinical trials on a relatively short timeline. These drugs have already been tested in humans with other diseases that involve muscle wasting and may benefit kids with Duchenne. One drug we are supporting in that category is a Selective Androgen Receptor Modulator (SARM) being developed by Akashi Therapeutics, and we are exploring others. Our strategy of pursuing a varied portfolio gives us the best chance of a shorter-term “win” while also moving forward longer-term projects that could bring tremendous benefit a few years down the road.