How Biotechnology and Advocacy Are Shaping the Future of Neuromuscular Care: John F. Crowley

WATCH TIME: 3 minutes

“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 several decades, advances in biotechnology, genomics, and translational research have reshaped the outlook for patients with rare and neuromuscular diseases. Conditions once managed primarily with supportive or palliative care are increasingly becoming targets for disease modifying therapies, including enzyme replacement, gene therapies, and precision medicines.¹ These developments reflect the growing convergence of academic discovery, biotechnology entrepreneurship, and patient driven advocacy, an ecosystem that has helped accelerate therapeutic development across the rare disease landscape.²

At the 2026 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, held March 8-11 in Dallas, Texas, John F. Crowley, President and CEO of the Biotechnology Innovation Organization (BIO), delivered a keynote address titled Driving the Future of Innovation, Policy, and Patient Impact. His presentation focused on the collaborative network of stakeholders including academic institutions, biotechnology startups, advocacy organizations, and industry partners that are required to translate early scientific discoveries into approved therapies for patients living with rare and neuromuscular disorders.

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