Leigh Ramos-Platt, MD, on Sharing Experience With Gene Therapy Administration


The clinical professor of neurology and pediatrics at Keck School of Medicine of USC discussed her learning experience administering gene therapies for the first time.

“We were not a trial side for either for either onasemnogene or delandistrogene. I had to rely on people that were. I was lucky that in our state, I can reach out to Craig McDonald at University of California Davis, or Perry Shieh at the University of California, Los Angeles, with questions. I was also very lucky that for the first patient that we infused, I shared that patient with Gerry Mendel... so I could look to them for guidance. And for those centers that were not trial sites, reaching out to experience centers, especially those that were involved in the trial, have a perspective that is very helpful.”

As gene therapies grow in prevalence in the clinical space and become more common, it has become paramount to examine current strategies for logistics of delivering gene therapies and strategize how they are administered in the clinic. Another important aspect with gene logistics is coordinating interdepartmental collaboration in centers delivering these therapies as well as ensuring 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), spoke with CGTLive® about her experience with administering gene therapies such as onasemnogene abeparvovec and delandistrogene moxaparvovec at USC. USC was not a trial site for either therapy and Ramos-Platt shared that it was a learning experience adapting to gene therapy. She emphasized how she leaned on colleagues in the space experienced with gene therapy.

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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