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Special Issue: Neuromuscular Cardiopulmonary Medicine in the Age of Emerging Therapies
  • David Birnkrant,
  • Linda Cripe
David Birnkrant
MetroHealth Medical Center
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Linda Cripe
Nationwide Children’s Hospital
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Abstract

Neuromuscular medicine is being revolutionized by new genetic and molecular therapies. The purpose of this Special Issue is to present an overview of these new therapies, to examine their cardiopulmonary effects, and to consider the future of neuromuscular cardiopulmonary care. The emphasis will be on Duchenne muscular dystrophy (DMD) and, to a lesser extent, spinal muscular atrophy (SMA), as these are the diseases with the most robust new drug development and related cardiopulmonary outcome data. This Special Issue contains articles on a number of relevant topics, including an overview of new genetic and molecular therapies for DMD, examining the currently available cardiopulmonary outcome data; and a critical examination of pulmonary outcome measures, assessing which outcomes should be used in treatment studies. We will provide an overview of cardiopulmonary phenotypic variability and discordance and their implications for assessing patient prognosis and response to therapies, and present a new perspective on neuromuscular-induced sleep-disordered breathing, viewed in the context of new and emerging therapies. Finally, we will consider which cardiac imaging modalities should be used as outcome measures in studies assessing DMD heart function, and take a look at novel therapeutic approaches to DMD heart disease, including management of rhythm disorders and heart failure, and the use of left ventricular assist devices.

Peer review status:ACCEPTED

06 Nov 2020Submitted to Pediatric Pulmonology
09 Nov 2020Assigned to Editor
09 Nov 2020Submission Checks Completed
09 Nov 2020Editorial Decision: Accept