The Importance of Early Treatment of Inherited Neuromuscular Conditions.
J Neuromuscul Dis
; 11(2): 253-274, 2024.
Article
em En
| MEDLINE
| ID: mdl-38306060
ABSTRACT
There has been tremendous progress in treatment of neuromuscular diseases over the last 20 years, which has transformed the natural history of these severely debilitating conditions. Although the factors that determine the response to therapy are many and in some instance remain to be fully elucidated, early treatment clearly has a major impact on patient outcomes across a number of inherited neuromuscular conditions. To improve patient care and outcomes, clinicians should be aware of neuromuscular conditions that require prompt treatment initiation. This review describes data that underscore the importance of early treatment of children with inherited neuromuscular conditions with an emphasis on data resulting from newborn screening efforts.
Palavras-chave
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Doenças Neuromusculares
Limite:
Child
/
Humans
/
Newborn
Idioma:
En
Revista:
J Neuromuscul Dis
Ano de publicação:
2024
Tipo de documento:
Article
País de afiliação:
Reino Unido