The Primary Catalyst

In any conversation about patient advocacy for Duchenne muscular dystrophy, Pat Furlong’s name is bound to come up.

Pat is the driving force behind Parent Project Muscular Dystrophy (PPMD), a patient advocacy organization founded by Pat and a group of parents of children with Duchenne in 1994. Since its founding, PPMD has played a pivotal role in the fight against Duchenne, by funding and advancing research, improving care and advocating for and connecting the community.

When her sons were diagnosed with Duchenne in 1984, the doctors told Pat there was no hope – there were no treatments, no clinical trials and no funding for research. This devastating news galvanized her, and she set out to learn everything she possibly could about the disorder: its pathology, the type and extent of research done, the people involved in the research community and what their plans were for the future. She began traveling around the world to medical centers, immersing herself in the Duchenne landscape. Pat found that unfortunately, few physicians were willing to speak to her, and not much research was being done.

Over the course of all her travels and countless conversations, Pat realized that she needed to start a patient advocacy organization in order to raise significant funds to push research forward and convince the scientific community to get involved. She reached out to a group of parents with whom she’d met throughout her travels, and PPMD was formed. They had a single goal: fight to end Duchenne. As the organization grew and began its work in earnest, in 2001, Pat helped lobby Congress to pass legislation that allocated more funding into research of muscular dystrophy, with a significant portion earmarked specifically for Duchenne. They also began holding annual meetings to share the latest in the fight against Duchenne and connect the community.

It was at one of the annual meetings where Pat and PPMD first met PTC, through Dr. Lee Sweeney. Dr. Sweeney, who eventually became PPMD’s Senior Scientific Advisor, was collaborating with PTC on a potential treatment for Duchenne – he suggested to Pat that she meet PTC. It was in their first conversations that Pat knew the company was committed to Duchenne. It was first time PPMD had met a company that knew Duchenne and was committed to finding a treatment. They wanted to hear her story and discuss how PTC could help aid PPMD in their mission. The collaboration between PPMD and PTC began.

PTC worked with Pat and her team to find ways to approach a treatment for Duchenne and invited her to their offices to share her moving story. Hearing directly from Pat and meeting PPMD and Duchenne patients was the catalyst that helped convert the company to a cause – it helped cement PTC’s mission to find a treatment. Over the years, as the partnership flourished, the team at PTC would meet Duchenne patients and then see them year after year. The team at PTC knew there were people waiting for them to develop a treatment, and this drove the team to act with urgency. PTC has continued to fight for Duchenne patients, and Pat feels that the company persevered when other companies would have given up and moved on.

PPMD is a powerhouse patient advocacy organization that has made an astonishing impact in the Duchenne community. Since the group’s advocacy efforts began in 2000, it has invested more than $50 million in Duchenne research and therapy development, leveraged $600 million in federal funding into Duchenne research and supported five U.S. drug approvals.

Pat is considered one of the foremost authorities on Duchenne in the world and leads PPMD to this day. She is committed to the fight against Duchenne – fighting in her sons’ honor and for everyone in the Duchenne community.