Leigh Ramos-Platt, MD, on Looking Forward to Gene Therapy’s Growth


The clinical professor of neurology and pediatrics at Keck School of Medicine of USC also discussed current strategies with gene therapy administration.

“I think gene therapies in general are going to grow. If we look at the approvals that have happened, at first it was just Luxturna and then maybe onasemnogene... and then they just started taking off. I mean, last year, there were several that were approved. I'm listening to not just in neuromuscular, but also in epilepsy, neurodevelopmental disorders, all of these therapies that are coming out in the hematology world, in the leukodystrophy world. And I think it's going to be a very different experience as a pediatric medicine provider in the next 10 years. It's exciting. It's scary. I don't know how our systems are going to be able to handle all of these kinds of treatments. But it's always a learning experience."

As gene therapies become more common and grow in prevalence in the clinical space, it is important to strategize their administered in the clinic and examine current strategies for logistics of delivering gene therapies.Coordinating interdepartmental collaboration in centers delivering these therapies is also paramount, as well as ensuring informed consent.

Leigh Ramos-Platt, MD, Medical Director, Muscular Dystrophy Association Neuromuscular Clinic and attending physician, Division of Neurology, Children’s Hospital Los Angeles, and Clinical Professor of Neurology and Pediatrics, Keck School of Medicine, University of Southern California (USC), gave a talk around this topic at the 2024 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, held March 3-6, in Orlando, Florida.

CGTLive® spoke with Ramos-Platt to learn more about some takeaways from the talk, including communication between parties and strategizing care as the most salient points. She also shared her excitement for the gene therapy space in general, which she has seen grow since her time in the field and expects to continue to grow in the near future.

Ramos-Platt L. DMD Gene Therapy Day Of: Institutional and Patient/Family Readiness- When We Were Not a Trial Site. Presented at: 2024 MDA Clinical and Scientific Conference; March 3-6; Orlando, FL.
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