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
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

1366943

Informations 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.

Auteurs

Leen Lagae (L)

Department of Pediatrics, University Hospital Leuven, Leuven, Belgium.

Marijke Proesmans (M)

Department of Pediatrics, University Hospital Leuven, Leuven, Belgium.
Woman and Child Unit, Department of Development and Regeneration, Catholic University of Leuven, Leuven, Belgium.

Marleen Van den Hauwe (M)

Department of Pediatrics, University Hospital Leuven, Leuven, Belgium.
Department of Rehabilitation Sciences, Research Group for Neurorehabilitation, Catholic University of Leuven, Leuven, Belgium.

François Vermeulen (F)

Department of Pediatrics, University Hospital Leuven, Leuven, Belgium.
Woman and Child Unit, Department of Development and Regeneration, Catholic University of Leuven, Leuven, Belgium.

Liesbeth De Waele (L)

Department of Pediatrics, University Hospital Leuven, Leuven, Belgium.
Department of Development and Regeneration, Locomotor and Neurological Disorders, Catholic University of Leuven, Leuven, Belgium.

Mieke Boon (M)

Department of Pediatrics, University Hospital Leuven, Leuven, Belgium.
Woman and Child Unit, Department of Development and Regeneration, Catholic University of Leuven, Leuven, Belgium.

Classifications MeSH