The Next Era of Neuromuscular Medicine: Innovation, Access, and Policy

The landscape of rare disease and neuromuscular medicine has evolved dramatically over the past several decades, shifting from largely supportive care toward an era defined by targeted therapies, genetic medicines, and patient-driven innovation. As biotechnology advances and new therapeutic platforms emerge, stakeholders across academia, industry, and advocacy organizations continue working to accelerate the development of treatments while improving access for patients living with rare diseases.

At the 2026 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, held March 8-11 in Orlando, Florida, John F. Crowley, president and chief executive officer of the Biotechnology Innovation Organization (BIO), delivered a keynote titled “Driving the Future of Innovation, Policy, and Patient Impact.” In the address, Crowley reflected on the evolution of the neuromuscular research ecosystem and emphasized the growing role of biotechnology startups, patient advocacy, and emerging technologies in shaping the future of rare disease care.

Following his keynote, Crowley spoke with NeurologyLive®about the progress made in neuromuscular medicine, the collaborative ecosystem required to translate scientific discoveries into approved therapies, and the policy and access challenges that remain as the field enters what he described as a “golden age of medicine.”

NeurologyLive: Could you provide a brief overview of your keynote presentation for those who may have missed it?

John Crowley: It was a privilege to be here at the conference. I came to the MDA science and clinical conference about 25 years ago. It was much smaller then. It was only researchers. There were no biotech companies, no patients, no patient advocates, and no caregivers.

Seeing everyone in that ecosystem come together today, the scale of it, but also the hope that is here, is remarkable. Years ago, when we were introduced to the world of science and medicine in neuromuscular disease, there was very little hope. It was mostly palliative care, even in Pompe disease, where our two children were diagnosed in the late 1990s. Megan was diagnosed at 15 months old and Patrick at just a few months old. After Megan’s diagnosis, we were told there was nothing that could be done and that we should go home and enjoy the time we had with our children.

Increasingly, those days are behind us. Now we have not just hope. For years people said we were on the cusp of a golden age of medicine. I believe we are now in that age. We are in the age of biology, and it is reflected here at this meeting.

For me, leading BIO, which is the world’s leading advocacy and policy organization for biotechnology and biomedical research, is a great privilege. It also gives me the opportunity to be an evangelist and remind people of everything and everyone it takes to make newer and better medicines.

The foundation of this innovation starts in academic research centers, medical institutions, hospitals, and the NIH. Small startup companies are incredibly important as well. In fact, about 70% of all medicines originate in startup biotech companies, often using technology spun out of academic research.

We need to preserve, protect, and advance that system. That is a big part of what we do at BIO. At the same time, we need to recognize the importance of medium-sized and larger companies that bring the financial resources, manufacturing capabilities, and global reach necessary to actually develop and distribute medicines.

One of the hardest things you can do in the world, and certainly in business, is to go from an idea to a drug. Turning an experimental molecule into an approved medicine is incredibly difficult and incredibly rare. And even when we succeed, it is often not a cure. It is something that meaningfully extends a person’s life.

Part of what I wanted to convey at the convention is not only the number of people required to create this virtuous circle of innovation, but also just how hard this work really is. The other important point is that making great medicines is not enough. We need to make sure that everyone who needs them has access to them.

We need to ask why people go without their medicines or why they are denied access. In many cases, the issue is the complexity of the system. There are too many middlemen involved in drug distribution, and there are insurance practices that create barriers.

Increasingly, patients face copays, deductibles, coinsurance, and other out-of-pocket costs. For rare disease medicines, those costs can reach thousands of dollars. If you are living with a rare disease or neuromuscular disorder, you already have enough burden. We believe there should be no coinsurance or deductibles for rare disease therapies.

We also need to examine insurance practices that create perverse incentives, where an insurance company may benefit financially from delaying or denying access to a medicine.

In situations where a patient is diagnosed with a neuromuscular disease and a physician prescribes an FDA-approved therapy, there should be no additional barriers. There should not be prior authorization or prolonged reviews by insurers. Patients have already gone through the necessary steps, and they should be able to access those treatments.

Simplifying the system, reducing out-of-pocket costs, eliminating unnecessary middlemen, and addressing insurance barriers are all critical steps. Too often, delays or denials of treatment simply prolong suffering and, in some cases, lead to death.

Neuromuscular medicine has advanced tremendously in recent years. Where do you see the field heading next?

I think the future is incredibly exciting. There are so many technologies advancing at once. Gene therapies, CRISPR-Cas9 gene editing, and small molecule precision medicines are all transforming how we approach treatment.

Increasingly, we will be developing therapies tailored to the individual based on their own genetic sequence. That is where medicine is going. Precision medicine will become more and more central to how we care for patients.

Artificial intelligence will also play a major role. AI can help shorten or even eliminate the diagnostic odyssey that many patients with rare diseases experience. It can support physicians in determining the best treatment strategies for their patients.

When we think about the future, we also have to think about access. How do we simplify the system, eliminate redundancies, and reduce bureaucracy so that patients can receive the treatments they need?

Technology can help us there as well. If we combine scientific innovation with smarter systems for delivering care, we can dramatically improve outcomes for patients living with rare and neuromuscular diseases.

Transcript edited for clarity. Click here for more MDA 2026 coverage.