Special Issue: Neuromuscular Cardiopulmonary Medicine in the Age of
Emerging Therapies
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.