Integrating Genetics and Transitional Support in Muscular Dystrophy Care: Amanda Peltier, MD

WATCH TIME: 5 minutes

"We need to do a better job on transitional care because for a lot of young adults that make that leap to adulthood, one of the biggest challenges is the drop-off in insurance. This is a big challenge because a lot of states like [Tennessee] have better coverage for kids with disabilities, and when they become adults, that coverage drops off as far as care in the home.”

Advancements in care, including genetic testing for diagnosis, and new therapies, have allowed patients with neuromuscular diseases to live longer into their adulthood, increasing their life expectancy. Despite these improvements in the field, research shows there is a lack of guidelines to facilitate transitioning patients with neuromuscular diseases from pediatric to adult care.¹ This transition process is particularly challenging for patients as they get older, when health declines more and independence is lost, because of barriers such as lack of access to adult clinicians, incompatible health insurance, loss of resources in patients' medical structures, among others.²

At the 2024 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, held March 3-6, in Orlando, Florida, chair leader Amanda Peltier, MD, presented a talk on the multidisciplinary care approach for adult patients, and the possibilities versus realities of treatment in a session. Overall, the session centered on clinical approaches to streamlining care using a multidisciplinary approach to meet patients' needs in neuromuscular disorders, which also included a presentation on the pediatric aspects given by Urvi Desai, MD, FAAN.

Peltier, a professor of neurology at Vanderbilt University Medical Center, sat down with NeurologyLive® at the conference to discuss how clinics can better integrate personalized resources for patients with muscular dystrophy. She also talked about the role that genetic testing plays in advancing treatment options for neuromuscular disorders. Additionally, Peltier explained why transitional care is essential for young adult patients with muscular dystrophy, and the key challenges these patients face.

REFERENCES
1. Accogli G, Ferrante C, Fanizza I, et al. Neuromuscular disorders and transition from pediatric to adult care in a multidisciplinary perspective: a narrative review of the scientific evidence and current debate. Acta Myol. 2022;41(4):188-200. Published 2022 Dec 31. doi:10.36185/2532-1900-083
2. Cheng PC, Panitch HB, Hansen-Flaschen J. Transition of patients with neuromuscular disease and chronic ventilator-dependent respiratory failure from pediatric to adult pulmonary care. Paediatr Respir Rev. 2020;33:3-8. doi:10.1016/j.prrv.2019.03.005