The neuromuscular physician at Virginia Commonwealth University talked about the promising future of gene therapy and access to resources in Duchenne muscular dystrophy. [WATCH TIME: 5 minutes]
WATCH TIME: 5 minutes
“One of the things that we always hear from parents is that it's difficult to access resources, whether that be educational modification and/or behavioral health for their children, especially when they are very young. It doesn't seem to be that much of a problem when they grow up to be teenagers. But I think the lack of access to mental health professionals and also out-of-pocket costs is a great burden for young families.”
In the field of Duchenne muscular dystrophy (DMD), the focus has been on muscle health, but recent conversations have called attention to the importance of cognitive symptoms as well. Some feel there is a lack of research and understanding of the standardized measures in DMD symptoms, as well as support for the community that is affected by the disease.
At the recent 2023 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, March 19-22, in Dallas, Texas, Mathula Thangarajh, MD, PhD, a child neurologist and neuromuscular physician at Virginia Commonwealth University, presented in a talk session focused on cognitive and behavioral health in muscular dystrophy. The rest of the session highlighted recent advances in the understanding of brain health in muscular dystrophy, including topics on pragmatic approaches and available resources to help patients navigate challenges in the field.1
In an interview with CGTLive®, a sister publication to NeurologyLive®, Thangarajh provided an overview of some of the main highlights presented during the session. She elaborated on the invisible cognitive and behavioral symptoms in muscular dystrophy and detailed why they are important. Additionally, she discussed the importance of easy access to behavioral health resources for patients with these conditions, especially those who are younger. Furthermore, Thangarajh spoke about the potential of gene therapy to address cognitive symptoms and the different approaches to neuromuscular disease with gene therapies.