Leigh Ramos-Platt, MD, on Allowing Access and Ensuring Preparation for Gene Therapies

“It is hard to understand the gravity of these medicines, because we always focus on such positive outcomes. But there's a dark side to it. And it's scary. There are side effects and it's hard to sometimes convey that. So, one of the challenges is that we have is to make sure that the patients understand what they're signing up for, that there is a risk of death, that there is a risk of other serious injuries and that they can be admitted to the hospital for some very serious potential side effects."

As gene therapies enter the clinical space and become more common, it has become paramount to examine and standardize the logistics of delivering gene therapies, including interdepartmental collaboration in centers delivering these therapies as well as informed consent and preparing patients and families for what gene therapy entails.

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 the talk and the greater session on post-gene therapy monitoring. She stressed the importance of making sure that patients and families of all backgrounds be able to access these lifesaving therapies, but are also adequately prepared for them. She also shared that across talks in the session, communication was most often identified as the most important factor in the post-gene therapy time period.

REFERENCE

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.