The pediatric neurologist at Nemours Children’s Health offered advice and discussed his experiences delivering pediatric gene therapy.
“It's really exciting to see the sheer number of new therapies that are available to our patients now. It's kind of a double-edged sword in a way, because now we're having to learn how to administer all these new medications. But that's a good problem to have.”
As gene therapies enter the clinical space and become more common, centers and hospitals seeking to administer these therapies must be educated and equipped for safely treating and following up patients that would receive these therapies. There is also an opportunity for centers experienced with gene therapy logistics to share their learnings and strategies to others looking to expand into the space.
Omer A. Abdul Hamid, MD, pediatric neurologist, Nemours Children’s Health, gave a talk at the 2024 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, held March 3-6, in Orlando, Florida, on gene therapy logistics, specifically on logistics during the day of dosing.
CGTLive® spoke with Hamid to learn more about his talk. He stressed the importance of learning from each other and using validated models of care. The logistics of gene therapy go beyond neuromusculardisease, so there is much to learn and share with other fields of medicine as well. He also shared the importance of making sure the dosing day goes smoothly, as it's a very meaningful day for patients, through the lens of his experience with pediatric gene therapy. This includes scheduling and making sure patients and their families have the resources they need to make these treatments accessible to all, including transportation and lodging on the day of dosing, as well as limiting outside exposure in the acute phase after receiving gene therapy. He also shared advice for centers looking to offer gene therapy treatments.