Spinal Muscular Atrophy: A Potential Target for In Utero Therapy.
Clin Obstet Gynecol
; 64(4): 917-925, 2021 12 01.
Article
em En
| MEDLINE
| ID: mdl-34560767
ABSTRACT
Spinal muscular atrophy (SMA) is a life-threatening autosomal recessive disease that leads to progressive muscle weakness and atrophy, respiratory insufficiency and scoliosis. SMA is currently the most common monogenic cause of infant mortality. Amazing advancements have been made in the therapeutic options available for these children since 2016. What has also become clear is that the earlier the treatment is administered, the better the clinical outcome. For several reasons, which we will review in this chapter, SMA may be an excellent disease candidate for in utero therapy.
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Atrofia Muscular Espinal
Limite:
Child
/
Humans
/
Infant
Idioma:
En
Ano de publicação:
2021
Tipo de documento:
Article