The effect of emerging molecular and genetic therapies on cardiopulmonary disease in Duchenne muscular dystrophy.
Pediatr Pulmonol
; 56(4): 729-737, 2021 04.
Article
em En
| MEDLINE
| ID: mdl-33142052
ABSTRACT
Gene therapy is an attractive approach being intensively studied to prevent muscle deterioration in patients with Duchenne muscular dystrophy. While clinical trials are only in early stages, initial reports are promising for its effects on ambulation. Cardiopulmonary failure, however, is the most common cause of mortality in Duchenne muscular dystrophy (DMD) patients, and little is known regarding the prospects for gene therapy on alleviating DMD-associated cardiomyopathy and respiratory failure. Here we review current knowledge regarding effects of gene therapy on DMD cardiomyopathy and discuss respiratory endpoints that should be considered as outcome measures in future clinical trials.
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Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Terapia Genética
/
Distrofia Muscular de Duchenne
Limite:
Humans
Idioma:
En
Ano de publicação:
2021
Tipo de documento:
Article