MetroHealth Advances Cardiorespiratory Management of Duchenne Muscular Dystrophy
Recently retired MetroHealth pediatric pulmonologist David Birnkrant, MD, and his colleague John Carter, MD, MetroHealth's pediatric sleep medicine physician, who also has a background in child neurology, co-authored a cover story in the April 2022 issue of the journal The Lancet Respiratory Medicine, among the top respiratory journals worldwide.
The article, which was co-authored by an international group of collaborators, offers insight into new and emerging therapies for Duchenne muscular dystrophy, a neuromuscular disease causing weakness of the skeletal and cardiac muscles. Over time, patients lose the ability to breathe normally and to cough effectively. Respiratory and cardiac impairment turn out to be life-limiting complications.
The article, titled "Cardiorespiratory management of Duchenne muscular dystrophy: emerging therapies, neuromuscular genetics and new clinical challenges," examines the new genetic therapies that are revolutionizing DMD care. The article explains best ways to evaluate the effect of the new therapies on heart and lung function, the need to understand the role of genetic modifiers, which can make DMD milder or more severe in patients with the same genetic mutation, and the ethical and clinical challenges that the new genetic therapies have caused.
Drs. Birnkrant and Carter summarize the importance of the article as follows: "Duchenne muscular dystrophy is closer to being a curable disease, with new advances in genetic therapy. This article includes ways to improve our understanding of how those new therapies affect heart and lung function, and discusses clinical and ethical challenges that have arisen, with the goal of improving quality of life for people with DMD, in addition to prolonging their survival."
Dr. Birnkrant has spent over 25 years working with patients and families in MetroHealth's MDA-sponsored pediatric neuromuscular clinic. He was the lead author and project co-leader of the CDC-sponsored Care Considerations for Duchenne muscular dystrophy, which was published in 2018 and is the premiere standard of care for the disease.
Founded in 1837, MetroHealth is leading the way to a healthier you and a healthier community through service, teaching, discovery, and teamwork. Cuyahoga County’s public, safety-net hospital system, MetroHealth meets people where they are, providing care through four hospitals, four emergency departments, and more than 20 health centers and 40 additional sites. Each day, our 8,000 employees focus on providing our community with equitable health care–through patient-focused research, access to care, and support services–that seeks to eradicate health disparities rooted in systematic barriers. For more information, visit metrohealth.org.