Muscular dystrophies are rare, complex, and often misunderstood. For patients and families, awareness isn’t a “nice to have” - it’s the beginning of everything that follows: earlier recognition of symptoms, faster referrals, access to genetic testing and clinical trials, better care coordination, and stronger support networks. Put simply, the more clearly we see muscular dystrophy, the more effectively we can act.
Why awareness matters (and how it moves the needle)
- Faster paths to answers: Awareness helps clinicians and communities recognize warning signs sooner and understand when to order genetic testing or refer to neuromuscular specialists.
- Access to options: An informed community is more likely to find clinical trials, emerging therapies, and multidisciplinary care.
- Support for families: Visibility reduces isolation, connects caregivers to resources, and helps schools and employers understand accommodations.
- Momentum for research and policy: Public understanding drives funding, advocacy, and sustained investment in science.
How Genomenon shows up for the MD community
For years, Genomenon has shown up for the MD community in two complementary ways: raising awareness and equipping researchers and clinicians with better evidence.
- Awareness & education. We regularly publish explainers, interviews, and campaign content that make complex genomics understandable - spotlighting patient and caregiver voices, clarifying testing pathways, and pointing readers to trusted resources and advocacy organizations. Our aim is to put reliable, plain-language information in more hands, more often.
- Free MD gene resources for researchers. To accelerate discovery, in the light of awareness month, we provide free MD gene resources (selected genes and evidence) to academic scientists at no cost - so labs can explore mechanisms, prioritize variants, and generate hypotheses faster without sacrificing evidence quality.
- Support the work of other organizations. We’re proud to support the work of the Muscular Dystrophy Association (MDA) and other community groups whose advocacy, services, and research leadership improve lives. Our role is to listen, amplify, and contribute the evidence and tools we do best.
Muscular Dystrophy Awareness: A Q&A with MDA’s VP Paul Melmeyer
The Muscular Dystrophy Association (MDA) has long been a cornerstone for the MD community - advocating for patients, advancing research, and supporting care across the neuromuscular landscape. Their work helps turn awareness into action and momentum into measurable progress.
In this conversation, Paul Melmeyer, Executive Vice President, Public Policy and Advocacy, Muscular Dystrophy Association, shares his perspective on why awareness months matter, what’s changing in research and patient support, and how collaboration can accelerate impact.
Q1. In your experience, why are awareness months critical in driving both public understanding and research progress in muscular dystrophy?
Paul: Awareness months concentrate attention, resources, and momentum in a way day-to-day activity rarely can. Muscular Dystrophy Awareness Month gives families a platform to share their stories and is a part of our #MDAstrong Campaign. It helps clinicians and researchers amplify discoveries and creates a moment for coordinated fundraising and advocacy.
This year’s #MDAstrong campaign includes Muscular Dystrophy Awareness Month but goes beyond that to embrace the entire neuromuscular community, including people living with ALS and over 300 rare conditions. And as we mark MDA’s 75th anniversary, we are reflecting on the progress made, but also the urgent need to act now to protect it. That’s why we launched the MDA Strong Policy Initiative, mobilizing advocates to push back against proposed NIH funding cuts. Every breakthrough we’ve ever celebrated started with research, and research starts with funding. Awareness is important, but translating awareness into action, advocacy, and policy change is how we ensure that progress continues year after year.
Q2. From your perspective at MDA, what progress or changes have you seen over the years in research and patient support?
Paul: The progress has been remarkable. We’ve gone from a time when there were no treatment options to today, when more than 25 FDA-approved therapies exist, with many more in development. Families now have access to MDA’s nationwide multidisciplinary clinics, advanced genetic testing, newborn screening programs, telehealth services, and strong clinical trial networks.
Equally critical are the supports around the neuromuscular community including MDA Community Education programming, MDA Resource Center, and MDA Summer Camp, to name a few. Our Advocacy efforts help families face everyday challenges, like ensuring wheelchairs damaged during travel are repaired quickly and without burden, access to caregiving, independent living, access to education and employment, diagnostic codes for insurance, and so much more. None of this happened overnight, it’s the result of decades of sustained advocacy. The new treatments available are due to decades of NIH investment, innovation from researchers and industry, and relentless advocacy by families and organizations like the Muscular Dystrophy Association. Protecting NIH funding is essential if we are to build on this momentum.
Q3. How can partnerships between patient advocacy groups, researchers, and industry accelerate progress for families affected by muscular dystrophy?
Paul: Partnerships are the engine of progress. Advocacy groups bring urgency and the patient voice; researchers bring scientific rigor; and industry has the capacity to develop therapies at scale. When these partners come together, at venues like the 2026 MDA Clinical & Scientific Conference, we see faster translation of discovery into real-world treatments.
Practical collaboration, like co-designing consensus guidelines for the delivery of gene therapy, clinical trials, and comparing advocacy priorities around funding and access and aligning where we can, shortens the path from discovery to treatment. At the Muscular Dystrophy Association, our role is to convene these stakeholders and ensure our community's priorities guide the work, from advancing therapies to protecting NIH funding, expanding caregiver support, and strengthening protections for mobility equipment. Partnerships rooted in advocacy, science, and lived experience are how we make the next wave of breakthroughs possible.
Q4. What message would you most like to share with the community during Muscular Dystrophy Awareness Month?
Paul: Your story matters, and your action matters just as much. #MDAstrong is about celebrating all forms of strength, but it’s also about channeling that strength into action. Whether you’re raising funds, sharing your story publicly, or advocating with lawmakers, you’re helping power research, care, and policy change that impacts millions.
This September, as we celebrate MDA’s 75-year legacy of progress, we’re also mobilizing to protect that progress. Proposed cuts to NIH funding put discovery at risk, and that means putting lives at risk. That’s why I’m asking everyone in our community to join us in advocacy. Visit MDA.org/Advocacy, share how NIH research has impacted your family, and tell Congress to keep NIH strong. Together, we can protect research, strengthen caregiver support, and ensure families have access to the equipment and services they need. Every voice matters, and when we advocate together, we are unstoppable. Stay tuned for MDA on the Hill in early November!
How you can help - today
- Share verified information from MDA and clinical centers; amplify patient and caregiver voices responsibly.
- Support research by connecting scientists to open resources
- Encourage evaluation when symptoms arise; normalize genetic testing and referrals to neuromuscular specialists.
- Sustain the signal past awareness months - advocacy, volunteering, fundraising, and informed conversations matter year-round.
Awareness is the first step that makes every other step possible.
To patients, families, clinicians, researchers, and advocates: thank you.
We’re proud to stand with you - and to keep building the evidence and tools that move care forward.
