Advancements and Challenges in Genetic Treatments for Neuromuscular Diseases: John W. Day, MD, PhD

WATCH TIME: 5 minutes

“I think one of the important things the MDA has always emphasized is the importance of bringing together clinical care, clinical research, and translational and basic research. I think that's really going to be at the forefront of this meeting, as we're trying to understand how to deliver these genetic treatments effectively and safely to the population.”

In the coming weeks, the Muscular Dystrophy Association (MDA) will reconvene for its annual Clinical and Scientific Conference in Orlando, Florida, held March 3-6. This gathering unites scientific and medical experts, patients, and advocacy groups, creating a forum to exchange the latest data on potential therapies, collaborate on challenges, and address the unique needs of diverse patient populations living with neuromuscular disorders.

At the meeting, cochair John W. Day, MD, PhD, medical advisor and care center director at the MDA, professor of neurology and pediatrics, and director of the Neuromuscular Division at Stanford Medicine, will present in a clinical considerations track session on rapid-fire clinical case reviews. The first of the 2-part session will be focused on motor neuron disease and neuromuscular junction disorders, while the second will be centered on muscle diseases.

Day sat down with NeurologyLive® in an interview prior to the meeting to discuss his track sessions that will be presented. He talked about the practical and ethical concerns arising from the use of genetic treatments for neuromuscular diseases. Day also spoke about how the success in identifying genetic causes of neuromuscular diseases posed challenges in treating rare conditions effectively. Additionally, he shared his perspective on how the FDA prioritizes the importance of rare diseases, and what implications effective treatment of rare diseases could have for the broader field of medicine.

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