I have been privileged to know Pat Furlong for the past several years and work alongside her on the board of the National Organization for Rare Disorders (NORD). In 1984, doctors diagnosed her two sons, Christopher and Patrick, with Duchenne Muscular Dystrophy (Duchenne), a fatal genetic disorder that slowly robs young men of their muscle strength. Pat didn’t accept the doctor’s advice that “there’s no hope and little help.” Instead, she immersed herself in Duchenne, working to understand the pathology of the disorder, the extent of research investment, and the mechanisms for optimal care. Her sons lost their battle with Duchenne in their teenage years, but she continues to fight—in their honor and for all families affected by Duchenne.
In 1994, Pat, together with other parents of young men with Duchenne, founded Parent Project Muscular Dystrophy (PPMD) to change the course of Duchenne and, ultimately, to find a cure. PPMD is the largest non-profit organization in the United States solely focused on Duchenne. Today, Pat continues to lead the organization and is considered one of the foremost authorities on Duchenne in the world.
Duchenne Central
I give this background to explain why the team at Siren Interactive was so happy to collaborate with PPMD on a mobile app titled Duchenne Central. The app, which is now available for free download for Apple and Android devices, is an easy-to-use and on-the-go tool to locate clinical trials and clinics and view and share the latest Duchenne news.
“Currently, there are no FDA-approved treatments for Duchenne; however, there are several potential therapies in development and currently in clinical trials,” says Pat. “It’s essential that patients and their families have ongoing access to information on the latest clinical trials. We’re proud to bring this new resource to the Duchenne community.”
Especially for rare disorders, enrolling enough patients in a clinical trial can be challenging. With the explosive growth of mobile devices, people are using their smartphones to access information 24/7 whenever and wherever they need it. This app will make it easier for Duchenne families to find the right resources. We’re pleased to help provide the app to the Duchenne community and to be able to support the amazing work of PPMD.
