Like many disorders that have benefited from research innovation and improved care, the life expectancy for individuals diagnosed with Duchenne muscular dystrophy has increased by more than a decade. This means that a disease that used to be considered a ‘pediatric disease’ is now more commonly referred to as a ‘pediatric-onset’ disease.
While this is great news overall, there exist significant societal, policy, healthcare, insurance, and systemic barriers for teens and adults transitioning from a pediatric to an adult settings – they are transitioning from life with parents to more independent living options. Simply put, a new population of adults exists.
For this reason, in 2015 Parent Project Muscular Dystrophy (PPMD), the world’s leading Duchenne-specific organization, convened the PPMD Adult Advisory Committee (PAAC). Comprised of adults with Duchenne and Becker muscular dystrophy, the PAAC serves to ensure that the voices of adults living with Duchenne & Becker muscular dystrophy are reflected in national legislative, policy, and program activities.
Since establishment, PAAC members have helped to shift the paradigm of how Congressional leaders, federal agency partners, clinical experts, and biopharmaceutical industry view individuals living with Duchenne. Representatives of the PAAC serve as special government employees to the Food and Drug Administration (FDA), on National Institutes of Health (NIH) Councils, have served as experts and authors on publications shaping Transition care guidelines, and serve on data safety monitoring boards for industry sponsors.
PAAC members also serve as critical conveners of the young adult-Duchenne community and lead an Annual Transition Meeting each June. The PAAC has helped provide important resources and tools for families seeking support and resources to navigate complex systems. Topics at the Meetings have included everything from healthcare, to benefit eligibility, to managing personal care attendants, to sex & dating. Throughout the year the PAAC sponsors informational webinars – and launched a social media forum last spring that has drawn membership across the globe.
The PAAC has also been very active in legislative efforts and policy through outreach on both the state and federal level. Each year, the PAAC meets for an Annual Leadership Workshop in Washington, DC and then heads to Capitol Hill to meet with congressional champions.
PPMD’s Adult Advisory Committee is paving new pathways for themselves and future generations of teens and adults with Duchenne – and demonstrating that individuals with pediatric-onset diseases such as Duchenne are focused on a lifetime of happiness, and not just a happy childhood.