Year in review 2019: Neuromuscular diseases.

Pediatric Pulmonology
D J Birnkrant, Jane B Black

Abstract

Neuromuscular cardiopulmonary medicine is entering a new and exciting phase, with studies that assess the respiratory effect of emerging genetic and molecular therapies. In this year's neuromuscular Year in Review, we focus on Duchenne muscular dystrophy (DMD), reviewing studies that evaluate the respiratory effect of eteplirsen, the cardiopulmonary effects of ataluren, and a study comparing the use of spironolactone with eplerenone for the treatment of DMD-related cardiomyopathy.

References

Dec 26, 2001·American Journal of Respiratory and Critical Care Medicine·M F PhillipsP M Calverley
May 27, 2010·Journal of Child Neurology·David J BirnkrantRobert C Bahler
Jun 1, 2014·The American Journal of Cardiology·Mahi L AshwathRobert C Bahler
Jun 23, 2015·Pediatric Pulmonology·David J BirnkrantMaroun J Mhanna
Aug 11, 2016·Respiratory Care·Jennifer G AndrewsUNKNOWN Muscular Dystrophy Surveillance, Tracking, and Research Network (MD STARnet)
Feb 6, 2018·Lancet Neurology·David J BirnkrantUNKNOWN DMD Care Considerations Working Group
Dec 15, 2018·Pediatric Pulmonology·Justin B JinDavid J Birnkrant
Mar 12, 2019·Journal of Neuromuscular Diseases·Navid KhanUNKNOWN Eteplirsen Investigators and the CINRG DNHS Investigators
Sep 25, 2019·Journal of the American Heart Association·Subha V RamanLinda H Cripe
Jan 31, 2020·Journal of Comparative Effectiveness Research·Eugenio MercuriUNKNOWN CINRG Duchenne Natural History Investigators
Feb 29, 2020·European Journal of Epidemiology·Erik LandfeldtHanns Lochmüller

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Citations

Dec 5, 2020·Expert Opinion on Drug Discovery·Barbara Miziak, Stanisław Czuczwar

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