From Father's Quest to Biotech Beacon: A New Era for Rare Disease

NEW YORK, NY – December 08, 2025 – The path from a devastating diagnosis to a potential cure is often measured in decades of painstaking research. But for John F. Crowley, that path was a personal sprint, a father’s desperate race against time that ultimately helped reshape the biotechnology landscape. Now, as the President and CEO of the Biotechnology Innovation Organization (BIO), Crowley is set to bring his unique perspective to the global stage, delivering the keynote address at the Muscular Dystrophy Association’s (MDA) 2026 Clinical & Scientific Conference.

The announcement places a figure whose life embodies the intersection of patient advocacy and corporate innovation at the heart of the world’s largest neuromuscular disease gathering. For a community grappling with over 300 different conditions, Crowley’s journey from parent to biotech pioneer offers a powerful testament to the theme of his upcoming address: “Driving the Future of Innovation, Policy, and Patient Impact.” His presence signals a pivotal moment, underscoring a new era where the human layer—the patient, the caregiver, the advocate—is not merely a recipient of innovation but its most powerful catalyst.

A Personal Quest that Reshaped an Industry

John Crowley’s story is the stuff of modern medical legend, chronicled in a Pulitzer Prize-winning book and immortalized in the film Extraordinary Measures. In 1998, his world was upended when two of his young children, Megan and Patrick, were diagnosed with Pompe disease, a rare and progressive neuromuscular disorder that was almost always fatal in its infantile-onset form. Faced with a grim prognosis, Crowley left his career at Bristol-Myers Squibb and poured his life into a singular mission: saving his children.

He co-founded Novazyme Pharmaceuticals in 2000, a startup laser-focused on developing an enzyme replacement therapy for Pompe. This audacious move pitted a father’s resolve against the colossal challenges of drug development. The company was acquired by Genzyme Corporation just a year later, with Crowley staying on to lead the Pompe program. The resulting therapy, now credited with saving the lives of his children and countless others, stands as a landmark achievement in rare disease treatment. His daughter Megan is now a college graduate working for the Make-A-Wish Foundation, a living symbol of what determined, patient-centered innovation can achieve.

His journey didn't stop there. Crowley went on to lead Amicus Therapeutics for nearly two decades, building it into a global company dedicated to therapies for rare metabolic diseases, before taking the helm at BIO in 2024. His career is a blueprint for how personal experience can fuel industry-wide change, embedding a deep-seated empathy into the corporate and policy frameworks that govern medical progress.

The Convergence of Advocacy, Policy, and Innovation

The MDA conference, scheduled for March 8–11, 2026, in Orlando, Florida, has long served as a critical nexus for the neuromuscular disease community. It is a place where lab researchers, frontline clinicians, pharmaceutical giants, and patient advocates converge to share data, forge partnerships, and chart the course for future treatments. Crowley’s keynote is poised to crystallize the importance of this collaborative ecosystem.

As he stated, “We are living in an extraordinary era for biotechnology—one where innovation, data-driven insights, and patient partnerships are transforming what’s possible for people living with rare diseases.” This statement reflects a core priority for BIO under his leadership: fostering an environment where policy, investment, and patient advocacy work in concert.

This synergy is essential for overcoming the immense hurdles in rare disease research. The development of novel therapies requires not only scientific brilliance but also supportive regulatory pathways, sustainable funding models, and the invaluable insights that only patients and their families can provide. The conference itself, backed by premier sponsors like Biogen, Novartis, and argenx—all major players in the neuromuscular space—is a microcosm of this ecosystem in action. These companies are not just funding an event; they are investing in the collaborative engine that fuels their own pipelines and, ultimately, delivers hope to patients.

An 'Extraordinary Era' for Neuromuscular Disease

Crowley’s optimism is well-founded. The field of neuromuscular disease is undergoing a profound transformation, driven by breakthroughs that were once confined to the realm of science fiction. Gene therapies like Novartis’s Zolgensma for spinal muscular atrophy (SMA) offer the potential for a one-time, life-altering intervention. RNA-based treatments, such as Biogen’s Spinraza, have fundamentally changed the standard of care for SMA. Other conference sponsors are at the vanguard of this movement; Scholar Rock is developing novel muscle-targeting therapies for SMA, while argenx has brought new hope to patients with generalized myasthenia gravis.

However, this era of unprecedented opportunity is not without its challenges. Patients still face grueling “diagnostic odysseys” that can last for years. The high cost of these transformative therapies raises critical questions about equitable access and reimbursement. Navigating the regulatory landscape for small patient populations remains a complex endeavor. Crowley's leadership at BIO places him at the center of these debates, advocating for policies that streamline development and ensure that breakthroughs in the lab can reach the patients who need them most.

His keynote is expected to address this duality—celebrating the remarkable scientific progress while confronting the systemic barriers that still stand in the way of universal access and care.

MDA at 75: A Legacy of Impact and a Vision for the Future

The timing of the 2026 conference is especially significant, as it follows the Muscular Dystrophy Association's 75th anniversary in 2025. For three-quarters of a century, MDA has been a relentless force, funding pivotal research, building a network of world-class clinics, and championing the rights of the neuromuscular community. The selection of Crowley as a keynote speaker is a powerful reflection of the organization's forward-looking vision.

As Dr. Sharon Hesterlee, MDA’s Interim President and CEO, noted, “John Crowley’s extraordinary journey, from father and entrepreneur to global leader in biotechnology, perfectly reflects the Muscular Dystrophy Association’s enduring mission.” That mission has always been to empower those it serves, and Crowley's story is the ultimate embodiment of that empowerment.

His address will not just be a speech; it will be a call to action for the thousands of attendees—a challenge to continue pushing boundaries and a reminder that behind every data point, every clinical trial, and every policy debate is a human life. The convergence of Crowley’s personal history with MDA’s institutional legacy in Orlando will mark a defining moment, setting a tone of determined optimism for the next chapter in the fight against neuromuscular disease.