Equitable Health Care Access Extends Beyond Costs, Medications

Addressing the cost of gene therapies has remained a prominent challenge as these interventions have become available for patients with spinal muscular atrophy, Duchenne muscular dystrophy (DMD), and other neuromuscular diseases. In this interview, Leigh Maria Ramos-Platt, MD, Children’s Hospital Los Angelas, highlights pertinent nonfinancial barriers that warrant further attention. She stresses the importance of considering geographic location, and abundance of health care resources and specialists, among other obstacles that can limit access to patients who need these therapies. Health care policy can help bridge these gaps, Ramos-Platt contends, because “patient access is not just the medication itself.”

These topics and more were explored at 2025 Muscular Dystrophy Association Clinical & Scientific Conference (MDA) held in Dallas, Texas. To stay up to date with the latest news from this conference, please visit our dedicated MDA page.

This transcript has been lightly edited for clarity; captions were auto-generated.

Transcript

What are some of the prominent nonfinancial challenges patients face access therapies in DMD and SMA, and how can health care policy address these barriers?

We’re in Los Angeles, so we're pretty urban, we're metropolitan. We sometimes complain we don't—but we do—have resources that other states don't have, that especially smaller states don't have. And because of that, our patients will have access to some of these treatments that, again, smaller states and more rural communities don't.

What we are hoping is that there can be policies which make it easier, either for small states to have their own ability to give these medications or make it easier for them to get it from centers like ours that can give these medications. I think it would also be of benefit if some of that cost was covered so that patients could get to us, because patient access is not just the medication itself. Patient access is also being able to get where you need to go.

I'm from Hawaii. I grew up there and just recently we gave our first dose of delandistrogene moxeparvovec, or Elevydys, for a [patient with DMD] in Hawaii. And the reason why they had to come here was because they don't have a center back home where they can give the medication. And even if they did, it's such a small number of subspecialists there who could handle the potential side effects.

A lot of these companies that are making these novel medications that also have the potential side effects have to select centers where they have the ability to not only administer it, but to take care of side effects. And so, policy to make it easier for patients to come to us to do their follow-ups would be helpful for these patients, again, that are not in urban or metropolitan high-density cities like ours.