Respiratory morbidity in patients with spinal muscular atrophy-a changing world in the light of disease-modifying therapies.
disease-modifying therapies
nusinersen
onasemnogene abeparvovec
respiratory morbidity
risdiplam
spinal muscular atrophy
Journal
Frontiers in pediatrics
ISSN: 2296-2360
Titre abrégé: Front Pediatr
Pays: Switzerland
ID NLM: 101615492
Informations de publication
Date de publication:
2024
2024
Historique:
received:
07
01
2024
accepted:
29
02
2024
medline:
29
3
2024
pubmed:
29
3
2024
entrez:
29
3
2024
Statut:
epublish
Résumé
Respiratory complications are common in spinal muscular atrophy (SMA) and significantly contribute to morbidity and mortality in these patients. Generalized respiratory and bulbar muscle weakness translates into diverse and complex clinical consequences necessitating strict follow-up and specialized care. The natural history of SMA has evolved drastically in recent years as a result of the introduction of novel, disease-modifying therapies. While the impact of these therapies on motor function is well described in literature, its consequence for respiratory management has not been extensively studied. In this review we aim to provide a comprehensive overview of the respiratory morbidities, their follow-up, management, and the impact of novel therapies in SMA.
Identifiants
pubmed: 38550632
doi: 10.3389/fped.2024.1366943
pmc: PMC10973112
doi:
Types de publication
Journal Article
Review
Langues
eng
Pagination
1366943Informations de copyright
© 2024 Lagae, Proesmans, Van den Hauwe, Vermeulen, De Waele and Boon.
Déclaration de conflit d'intérêts
The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest.