For families living with Duchenne and Becker, planning for adulthood should bring possibilities: education, relationships, work, greater independence, and the opportunity to build a life shaped by individual goals and choices. Too often, it also brings a difficult question about who will provide care when moving from pediatric to adult settings.
At PPMD, we believe access to expert, coordinated Duchenne and Becker care should continue throughout life. As more individuals in our community reach adulthood, we are working with clinicians, researchers, and advocacy partners to help build care systems to grow with our community.
PPMD has developed a new Adult Care section on our website, bringing together resources to help individuals and families navigate transition and transfer of care, clinical care, therapies, equipment, independence, college, employment, relationships, community, and more.
Understanding the adult care landscape
We recently completed a review across our 39 Certified Duchenne Care Centers to better understand practices around transition to adulthood, transfer to adult care, including which centers provide care throughout the lifespan and learned that a number of centers have age limits for patients and require transfer of care to an adult provider or associated healthcare system. This work helps us identify where families have a clear path into adult care and where there are gaps.
We are also working to engage with hospital administrators and academic institutions to better understand the challenges behind these decisions . These conversations are an opportunity to bring the needs of our community to the table: continuity of care, experienced providers, and multidisciplinary teams that understand Duchenne and Becker.
Updating care for a changing community
PPMD is updating the Duchenne care standards first published in 2010 and later updated in 2018 with up-to-date consensus recommendations across areas including cardiac and respiratory care, endocrine and bone health, rehabilitation, primary care, digestive and urinary health, emergency care, palliative care, and more. These updated recommendations span the entire dystrophinopathy spectrum across the lifespan, including considerations for Becker and carriers. These recommendations explore newly approved and emerging therapies, including potential use of sequential therapeutic approaches, and considers how these developments should inform care.
Our goal is to develop practical guidance that reflects the needs of the dystrophinopathy community in today’s unique care landscape, including the growing number of adults living with Duchenne and Becker.
Building teams prepared to care for adults
We know that finding adult providers with dystrophinopathy experience—or providers who are willing to develop that expertise—remains a significant challenge.
PPMD is working to strengthen connections between pediatric and adult providers, support the development of coordinated adult care teams, and learn from other communities that have experience building models of adult care. Through these efforts, we aim to help more adults with Duchenne and Becker access knowledgeable providers and coordinated care.
Making coverage and independence part of the solution
A knowledgeable adult care team is essential, but quality adult care also depends on access to insurance coverage, equipment, caregivers, and opportunities to participate fully in community life.
Our advocacy work is focused on three key priorities:
- Protecting access when insurance changes by working with the Muscular Dystrophy Public Health Consortium (MDPHC) to address coverage gaps that can happen when Medicare becomes part of a person’s coverage.
- Exploring better ways to deliver and pay for care by working with the CMS Innovation Center.
- Supporting employment and independence by advocating for policies that provide clear, accurate guidance around benefits, work, and income.
Advancing heart, endocrine, and bone care
PPMD is continuing to strengthen collaborations that address important needs throughout life, including our work with OPTIMIZE DMD on endocrine issues such as bone health, weight management, growth, and puberty and sexuality, and with the ACTION Learning Network on cardiac care.
Through the U.S. Department of Defense’s Congressionally Directed Medical Research Programs (CDMRP), PPMD has also helped secure support for efforts including ACTION and the Duchenne Muscular Dystrophy Cardiac Care Consortium (DMDCCC). We are working with the FDA toward a cardiac meeting to discuss the potential of left ventricular ejection fraction (LVEF) as a primary outcome measure in therapy development, helping make heart health a more central and measurable part of evaluating potential therapies.
Looking ahead
There is meaningful progress to celebrate, but still much to do to ensure that growing older with Duchenne means having access to the care, support, and opportunities people need to live the lives they choose.
This work is about more than extending care beyond childhood. It is about recognizing that adulthood is an important part of the Duchenne journey and making sure our care, resources, and expectations grow with our community.
The post Care Should Grow With You: Building a Better Future for Adults With Duchenne and Becker appeared first on Parent Project Muscular Dystrophy.