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Updated: Dec 14, 2025
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Systemic Delivery of MicroRNA Using Recombinant Adeno-associated Virus Serotype 9 to Treat Neuromuscular Diseases in Rodents
Published on: August 10, 2018
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Year in review 2019: Neuromuscular diseases
David J Birnkrant1,2, Jane B Black2
1Department of Pediatrics, MetroHealth Medical Center, Cleveland, Ohio.
Pediatric Pulmonology
|July 22, 2020
Summary
This review highlights new respiratory therapies for Duchenne muscular dystrophy (DMD). It examines eteplirsen, ataluren, and compares spironolactone with eplerenone for DMD cardiomyopathy.
Area of Science:
- Neuromuscular medicine
- Cardiopulmonary medicine
- Respiratory medicine
Background:
- Neuromuscular cardiopulmonary medicine is advancing with new genetic and molecular therapies.
- Duchenne muscular dystrophy (DMD) presents significant cardiopulmonary challenges.
Purpose of the Study:
- To review recent studies on the respiratory effects of emerging therapies in DMD.
- To assess the cardiopulmonary impacts of specific treatments for DMD.
Main Methods:
- Review of studies on eteplirsen for respiratory effects in DMD.
- Analysis of cardiopulmonary effects of ataluren in DMD.
- Comparison of spironolactone and eplerenone for DMD-related cardiomyopathy.
Main Results:
- Eteplirsen shows potential for respiratory improvement in DMD.
- Ataluren has demonstrated cardiopulmonary effects in DMD patients.
- Spironolactone and eplerenone offer comparable treatment options for DMD cardiomyopathy.
Conclusions:
- Emerging therapies are transforming respiratory care in DMD.
- Further research is needed to fully elucidate the cardiopulmonary benefits of these treatments.
- Treatment decisions for DMD cardiomyopathy should consider available therapeutic options.
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