MDA 2026: Keynote speaker to MDA community: ‘Your voice is powerful’
John F. Crowley is CEO of global biotechnology advocacy organization
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At this year’s Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, researchers and clinicians are talking about the central role of patients, families, and caregivers in driving change for people living with neuromuscular diseases.
Muscular Dystrophy News Today sat down with John F. Crowley, president and CEO of the Biotechnology Innovation Organization (BIO) and this year’s keynote speaker, to talk about his perspectives on the challenges and victories in neuromuscular disease research and advocacy over the years — and the key role that families play.
“The role of individuals and families is at the core of everything,” Crowley said. “They’re the ones driving science. They’re the ones convening. They’re the ones at this conference.”
Years ago, Crowley says, only researchers attended the MDA meeting, but that has changed.
“Increasingly now, patient advocates are at the core of driving science, bringing research together, advocating, funding, so that’s really at the vanguard of all of this, and needs to be,” he said.
‘This is kind of where it all began’
Crowley, also a neuromuscular disease parent and longtime MDA collaborator, kicked off the MDA meeting, running this week in Orlando, Florida, with his address, titled “Driving the Future of Innovation, Policy, and Patient Impact.”
When asked what the honor of speaking at the MDA meeting means to him, Crowley replied: “For me, it’s very personal to have been here to see other patients, families, [and] caregivers, but to also look back … and to realize that this is kind of where it all began.”
He’s now a leader in the neuromuscular disease field, but it all started for Crowley more than 25 years ago when his two children, Megan and Patrick, were diagnosed with the rare neuromuscular condition Pompe disease at a very young age.
“Back then, there was little research, and we were told the kids would only live to be a couple of years old, they would get very sick very quickly, and there was nothing we could do,” Crowley said.
The CEO, just a couple of years out of business school and without any scientific or medical background, teamed up with the MDA to connect with the few scientists who were working on Pompe at the time. Crowley, along with family and friends, started raising money through bake sales and other fundraisers.
“That partnership, and some of those early research dollars to those handful of researchers all came through the MDA,” Crowley said. “So I find it inspiring to be part of this conference … informative and also a bit nostalgic.”
‘Golden age of medicine’
Crowley’s early commitment eventually led to the founding of two pharmaceutical companies — Novazyme Pharmaceuticals and Amicus Therapeutics — and multiple approved therapies for Pompe. His goal was always to ensure the patient perspective was central to everything they did.
Now, as the leader of BIO, a global biotechnology advocacy organization whose work spans policy, advocacy, and communication, Crowley says he has the privilege of advocating for all of biotech on a larger scale. He hopes that with this work, he’ll see “hundreds of new Novazymes and Amicuses be formed to tackle thousands of different diseases.”
Recent years have seen massive progress in the treatment of rare and neuromuscular diseases, with technologies such as gene therapy now being used to treat people with muscular dystrophy and spinal muscular atrophy.
Crowley touts this modern era as the “biology revolution” and the “golden age of medicine,” with researchers innovating at an unprecedented rate.
“To think about these early ideas that are being fertilized at our great research academic centers … some of them will go on years, decades from now to be the next generation of cures that we haven’t even yet heard of and sometimes haven’t even yet thought of … that’s what’s exciting,” he said.
‘We just have to be relentless’
However, challenges remain in translating scientific advances into benefits for the patient community. A major recent focus of BIO is ensuring that advances in treatment and technology are actually accessible to people living with neuromuscular diseases.
Crowley points to the complexity of the drug development and insurance systems in the U.S., along with economic factors, as problems that need to be solved.
Copays, coinsurance, and out-of-pocket payments mean families may be asked for “astronomical” amounts of money for a therapy they need, which then “puts that medicine out of touch.”
Also problematic are current insurance practices that delay or deny coverage for treatment, even after a doctor has deemed it medically necessary. Insurance providers sometimes take a stepwise approach, requiring patients to try older, less effective therapies before covering a newer, pricier medication if the person gets sicker.
“I’m firmly of the belief … that if you were diagnosed with one of these diseases, and there’s a new medicine … that’s been approved by the FDA [U.S. Food and Drug Administration], and a doctor writes a prescription for that person to have that medicine, the insurance company must provide that medicine,” Crowley said.
He notes that these older systems were developed so that patients would have shared economic risk, but “if you’ve got a disease and you’re struggling, you’ve got enough skin in the game.”
Crowley has said he and his colleagues spend a lot of time on Capitol Hill advocating for policy changes to help break down those barriers. In his keynote address, he discussed the need for a 21st-century affordability and access act that appropriately addresses modern issues related to accessing advanced, personalized treatments.
“Washington is a difficult environment,” he said. “We just have to be relentless.”
Your voice is powerful. We need you in Washington to educate lawmakers and policymakers. We need you to educate the FDA and advocate with regulators. Every part of that ecosystem, that virtuous circle, needs to be driven by families, caregivers, patients.
The advocate believes the best strategy to mobilize lawmakers is to act as a storyteller, not only for the patients and families “living, sometimes thriving, despite these enormous challenges,” but also the researchers, care providers, and biotech companies working to accelerate progress.
These efforts can lead to major wins. Echoing a recent conversation between Muscular Dystrophy News Today and Sharon Hesterlee, PhD, MDA’s president and CEO, Crowley points to the recent congressional renewal of the Rare Pediatric Disease Priority Review Voucher program as one such victory.
The program is designed to incentivize the development of treatments for rare diseases affecting children that wouldn’t otherwise be financially attractive to pharmaceutical companies. The reauthorization of that program, which had expired at the end of 2024, “are the types of wins we need,” Crowley said.
While large organizations such as BIO help make these victories happen, Crowley emphasized that they don’t come without the support of individuals and families.
The CEO advises families affected by neuromuscular diseases to educate themselves, understand their resources, and advocate for advances in science, technology, and patient care.
“Your voice is powerful,” Crowley said. “We need you in Washington to educate lawmakers and policymakers. We need you to educate the FDA and advocate with regulators. Every part of that ecosystem, that virtuous circle, needs to be driven by families, caregivers, patients.”
Note: The Muscular Dystrophy News Today team is providing live coverage of the 2026 MDA Clinical & Scientific Conference March 8-11 in Orlando, Florida. Go here to see the latest stories from the conference.
