The respiratory impact of novel therapies for spinal muscular atrophy.
SMA
respiratory outcomes
treatment
Journal
Pediatric pulmonology
ISSN: 1099-0496
Titre abrégé: Pediatr Pulmonol
Pays: United States
ID NLM: 8510590
Informations de publication
Date de publication:
04 2021
04 2021
Historique:
revised:
19
10
2020
received:
12
08
2020
accepted:
20
10
2020
pubmed:
25
10
2020
medline:
13
10
2021
entrez:
24
10
2020
Statut:
ppublish
Résumé
The phenotype of spinal muscular atrophy (SMA) has been changing with the recent availability of three FDA-approved treatments: intrathecal nusinersen, intravenous onasemnogene abeparvovec-xioi, and enteral risdiplam. The degree of improvement in muscle strength and respiratory health varies with SMA genotype, severity of baseline neuromuscular and pulmonary impairment, medication used, and timing of the first dose. A spectrum of pulmonary outcomes has been reported with these novel medications when used early and in conjunction with proactive multidisciplinary management of comorbidities. In this review, we summarize the reported impact of these novel therapies on pulmonary well-being and the improving trajectory of pulmonary morbidity, compared to the natural history of SMA. The importance of ongoing clinical monitoring albeit the improved phenotype is reiterated. We also discuss the limitations of the current SMA-therapy trials and offer suggestions for future clinical-outcome studies and long-term monitoring.
Substances chimiques
Biological Products
0
Recombinant Fusion Proteins
0
Zolgensma
0
Types de publication
Journal Article
Review
Langues
eng
Sous-ensembles de citation
IM
Pagination
721-728Informations de copyright
© 2020 Wiley Periodicals LLC.
Références
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