John F. Crowley, JD, MBA, on the Future of Neuromuscular Care

This interview was originally published on our sister site, NeurologyLive®.

“For years people said we were on the cusp of a golden age of medicine. I believe we are living in it now. What you see at meetings like this is the entire ecosystem of scientists, biotech companies, advocates, and patients working together to make that possible.”

Over the past few decades, progress in biotechnology, genomics, and translational science has significantly altered the therapeutic landscape for individuals with rare and neuromuscular disorders. Diseases that were historically managed largely through supportive or palliative interventions are increasingly being addressed with disease-modifying approaches, including enzyme replacement therapies, gene therapies, and other precision medicine strategies.¹ These advances reflect the growing intersection of academic research, biotechnology innovation, and patient advocacy, a collaborative ecosystem that has played an important role in accelerating treatment development across the rare disease field.²

In an interview with CGTLive®’s sister site NeurologyLive® at the 2026 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, held March 8–11 in Dallas, Texas, John F. Crowley, JD, MBA, the president and chief executive officer of the Biotechnology Innovation Organization (BIO), gave a keynote presentation entitled Driving the Future of Innovation, Policy, and Patient Impact. In his remarks, Crowley emphasized the interconnected roles of stakeholders—including academic researchers, emerging biotechnology companies, patient advocacy groups, and industry collaborators—in advancing early scientific discoveries into approved treatments for patients affected by rare and neuromuscular diseases.

Crowley’s perspective is informed not only by his leadership role at BIO, the world’s largest biotechnology advocacy organization, but also by his personal experience as a parent of children diagnosed with Pompe disease. His keynote reflected on how the field has evolved over the past two decades and highlighted the growing role of biotechnology innovation in transforming the rare disease landscape.

Following the keynote session, Crowley spoke with NeurologyLive® about the themes of his presentation and the broader forces shaping the future of rare disease innovation. In the conversation, he reflects on the expanding ecosystem supporting therapeutic development, the role of biotechnology startups in bringing new medicines to patients, and the policy and access challenges that remain as the field continues to advance.

Click here for more MDA 2026 coverage.

REFERENCES
1. Mendell JR, Al-Zaidy SA, Rodino-Klapac LR, et al. Current and emerging therapies for neuromuscular diseases. Nat Rev Neurol. 2023;19(2):87-104. doi:10.1038/s41582-022-00730-7
2. Austin CP, Cutillo CM, Lau LPL, et al. Future of rare diseases research 2017 to 2027: an IRDiRC perspective. Clin Transl Sci. 2018;11(1):21-27. doi:10.1111/cts.12500