Advancing Clinical Trials and Treatment Strategies for Charcot-Marie-Tooth Disease: Joshua Burns, PhD

WATCH TIME: 7 minutes

“I'm really excited about chairing this track. It's in the Lab to Life Track of the Muscular Dystrophy Association Conference. I've been to many of these sessions before, and I'm really looking forward to this one because we really are on the cusp of rational, disease-modifying therapies for Charcot-Marie-Tooth disease and other inherited peripheral neuropathies.”

Starting March 16th, 2025, the Muscular Dystrophy Association (MDA) will host its yearly Clinical and Scientific Conference in Dallas, Texas. The annual 4-day event brings together scientific and medical professionals, patients, and advocacy groups to explore the latest advances in the field of neuromuscular disorders.¹ The conference will feature presentations on all different types of neuromuscular diseases such as amyotrophic lateral sclerosis, Duchenne muscular dystrophy, spinal muscular atrophy, limb-girdle muscular dystrophy, myasthenia gravis, Charcot-Marie-Tooth (CMT) disease, and much more.

In this upcoming meeting, session tracks will feature topics including strategies for regenerating muscle and nerve tissue, new insights into disease mechanisms, clinical experience in gene therapy, access and reimbursement, and clinical trial readouts. At the conference held last year, 1492 in-person and 567 virtual attended from over 30 countries, which made it the biggest global gathering of the neuromuscular disease community. For this year, those who are registered with MDA have the opportunity to partake in the virtual meeting at no-cost or may register in-person at the patient/caregiver rate until allotted spots are filled.¹

Prior to the event, one of the conference track chairs, Joshua Burns, PhD, a clinical researcher at St. Jude Children's Research Hospital, sat down with NeurologyLive^® in an interview to discuss the key advancements in disease-modifying therapies for CMT disease, and how close we are to seeing their widespread clinical application. He also talked about how he envisions the future of treatment for CMT and related neuropathies evolving over the next few years. Moreover, Burns spoke about the biggest challenges currently preventing the development of effective disease-modifying therapies for these conditions and what he is looking forward to at this year’s MDA conference.

Registration for the 2025 MDA Conference is now open! The meeting is set to be held at the Hilton Anatole, Dallas, Texas, from March 16 to 19, 2025. To register and for more information, head to www.mdaconference.org

REFERENCES
1. Registration Now Open for the 2025 MDA Clinical & Scientific Conference Featuring the Latest Innovations in Neuromuscular Disease Research and Care with Global Leaders, March 16-19 in Dallas, TX. News Release. MDA. July 30, 2024. Accessed September 25, 2024. https://www.mda.org/press-releases/2024/registration-now-open-for-the-2025-mda-clinical-and-scientific-conference-featuring-the-latest-innovations-in-neuromuscular-disease-research-and-care-with-global-leaders-march-16-19-in-dallas-tx