Rare Disease Report

Advocacy Spotlight: Parent Project Muscular Dystrophy

MAY 20, 2016
The mission of Parent Project Muscular Dystrophy (PPMD) is a straightforward one: curing Duchenne muscular dystrophy. The nonprofit organization is devoted to accelerating medical research, advocating in Washington DC, demanding optimal care for DMD patients, and educating and educating the global community. Its family centered approach is central to its mission.

About Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is a genetic muscular disorder that causes progressive muscle weakness. Due to lack of a functional dystrophin protein, muscle cells lose their structural integrity over time. Affected individuals, mostly boys, typically lose the ability to walk by age 10, and life-threatening heart and lung complications occur in the late teens and twenties.
DMD occurs in roughly 1 in 3,500 live male births. However, the disorder can be subdivided into different subsets based on the type of mutation involved. There are currently no treatments approved in the US for DMD, though research is moving forward on a number of different medications, some of which are in phase II or phase III trials. Advocates are frustrated, however, that this research hasn’t yet translated into new available treatments for this generation of affected boys.

About Parent Project Muscular Dystrophy

In 1994 a group of parents and grandparents of children with DMD founded Parent Project Muscular Dystrophy (PPMD). Among them was president and CEO Pat Furlong, the mother of two boys with DMD.
Through a variety of initiatives, PPMD provides many ways for its members to stay involved, including raising funds to support research through grassroots fundraising events.
In the Coach to Cure MD program, the American Football Association is partnering with PPMD to raise awareness for DMD, and community members can host events to further increase awareness. Community members can also participate in the Run for Our Sons Program, which has raised over $5 million for Duchenne in runs across the country.

Where Does the Money Go?

PPMD takes a multifaceted approach to accelerating effective research for DMD. For example, through the GIFTED fund (Global Investment For Therapeutics to End Duchenne), the organization invests in a number of projects specifically designed to improve the quality of drug candidates, decrease the time needed to test drugs, and review drugs already approved by the FDA.
PPMD has invested millions of dollars in direct research. PPMD also indirectly promotes research through its interactions with the pharmaceutical industry, through collaboration with the Duchenne research hub and the European research group TREAT-NMD, and through funding patient registries.
PPMD also actively promotes advocacy through multiple avenues. It provides key information to members about ongoing legislative action items. It has worked to push through a number of key initiatives, including the Muscular Dystrophy Community Assistance, Research, and Education Act, which made possible a number of critical programs promoting DMD research. The organization also recently brought together an advisory committee to make recommendations to help speed responsible access to newly developed therapies under the Food and Safety Administration Safety and Innovation Act.
PPMD additionally sets out to improve current care for all patients with DMD. The organization has worked to certify centers delivering the best Duchenne care. Through the Certified Duchenne Care Center Program, it aims to standardize and improve care to all people with DMD. It also helps fund free genetic testing and family outreach to all families coping with DMD.
Lastly, PPMD provides extensive educational materials and opportunities for patients, clinicians, and researchers. For example, through the early identification of child weakness project, PPMD is helping clinicians diagnose DMD earlier. PPMD also hosts the largest international conference focused exclusively on Duchenne. Through apps, website information, newsletters, webinars, social media, conferences, and more, PPMD leads the way in disseminating information to those who need it most.
These are only some of the many program initiatives pursued by PPMD. On the PPMD website, President Pat Fulong says, “PPMD is a community of parents, because parents will do anything to save their children.” She adds, “PPMD has seeded every single drug and every single strategy that is currently coming into trial.” The breadth and impact made by PPMD are truly breathtaking and heartening.

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