Conversations on Holistic Approaches to Neuromuscular Diseases at MDA 2023

Anne Connolly, MD

The 2023 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference is set to kick off March 19-22 in Dallas, Texas. The first day of the event includes meetings for care center directors, trainees, and neuromuscular disease advocates, concluding with the Welcome Reception at the end of the day. Comprised of hundreds of in-person and virtual attendees, the conference will explore all aspects of pre-clinical, translational, and clinical research and care across neuromuscular diseases to support to development of better care and treatments for the community.

On Tuesday, March 21, Anne Connolly, MD, will chair a session geared towards holistic approaches to transitioning into adulthood. The session will feature other speakers, including Jamie Twanow, MD, section chief of neurology at Nationwide Children’s, and Richard Shell, MD, professor of pediatrics and section chief of the Department of Pulmonary Medicine at Nationwide Children’s Hospital and The Ohio State University College of Medicine. Ahead of the meeting, Connolly, the chief of the Division of Neurology at Nationwide Children’s Hospital, provided insight on the state of translational care in neuromuscular diseases, the main challenges with this process, and the exciting aspects of her chaired session.

NeurologyLive®: How would you describe the state of transitional care in NMD?

Anne Connolly, MD: The state of transitional care in neuromuscular diseases is best summarized in a single word, “variable.” Some centers do transition readiness and transitional care better than others. Some adolescents and young adults are easier to transition than others. Some parents are ready but some really are afraid to let their child begin to make his or her own decisions.

What are the main challenges or lingering difficulties in this process?

There are at least three major challenges I can think of recognizing that different sites face different challenges. First, Some centers have different rules about the exact age at which transition must happen. Second, while most sites have well trained adult neuromuscular specialists who are willing to take care of young adults, those sites may not have cardiologists or pulmonologists who are prepared to do the same. Third, multidisciplinary clinics in pediatric neuromuscular centers are becoming the norm but that may not be possible for adult clinics and then families, used to having all services available at every visit, the addition of a full time cardiologist or pulmonologist is not always possible at adult centers.

What should participants expect from the Holistic Approach to Transitioning to Adulthood session at MDA?

The three talks discuss the approach that has been successful for young people with other complex disorders, the science of the young person’s own readiness and then share some direct experiences of becoming an adult with neuromuscular disease.

What are you most excited about regarding the upcoming meeting?

My favorite part of the Muscular Dystrophy Association meeting every year is catching up on both basic and translational science for many disorders. It is a terrific way to catch up on best practice for all children and adults with neuromuscular disorders. I also deeply enjoy the camaraderie of catching up with friends and colleagues with whom I have lived “parallel lives” for many years.