Challenges for the Neuromuscular Community Entering Treatment Era: Barry Byrne, MD, PhD

WATCH TIME: 4 minutes

"We’re trying to educate everyone about how those [AAV antibodies] are measured, how they’re meaningful in terms of access to therapy, and also how to mitigate the presence of those antibodies with strategies. This is a topic that we’ll be discussing in the gene therapy readiness program that MDA has supported."

Several in neurology would agree that over time, the field has changed from a diagnostic specialty, to a therapeutic one. Now, there are emerging, novel treatments for all types of conditions, including rare, neuromuscular diseases, which have only benefitted from symptomatic approaches for years. Because of advances in genetic testing and research, these disorders are able to be identified at the earliest stages possible, allowing for instant intervention and better long-term outcomes.

In recent years, the FDA has approved a number of new treatments for those with Duchenne muscular dystrophy (DMD), spinal muscular atrophy (SMA), amyotrophic lateral sclerosis, and others. In addition, the field has broken through with a handful of genetic-based approaches that seek to modify or manipulate the expression of a gene or alter the biological properties of living cells. While these are certainly promising times for the neuromuscular community, they come with challenges, says Barry Byrne, MD, PhD.

Byrne, associate chair of pediatrics, and director, Powell Gene Therapy Center, University of Florida, will be heading a panel discussion at the upcoming Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, held March 3-6, in Orlando, Florida, on neuromuscular disease in the age of therapy. Prior to the meeting, he sat down to give an overview of the session, and the reasons behind it. Byrne, who also serves as a chief medical advisor to the MDA, discussed some of the specific challenges clinicians will face with new treatments, including gene therapies that use adeno-associated virus (AAV) vector mechanisms of action and the antibodies associated with them.

Registration for the 2024 MDA Conference is now open! The meeting is set to be held at the Hilton Orlando, in Orlando, Florida, from March 3 to 6, 2024. To register and for more information, head to www.mdaconference.org.