Assessment of respiratory muscles and motor function in children with SMA treated by nusinersen.

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Abstract

Nusinersen is associated with an improvement in motor function in children with spinal muscular atrophy (SMA) but data on respiratory muscles strength are scarce. Respiratory muscles performance and lung function were evaluated in children with SMA 1c and 2 after six injections of nusinersen (M14). Results from patients with SMA2 were compared with data of age-matched historical controls. Motor function tests (MFM and HINE-2) were assessed at baseline and M14 in the treated patients.

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Journal

Journal ID (iso-abbrev): Pediatr Pulmonol

Title: Pediatric pulmonology

Publisher: Wiley

ISSN (Electronic): 1099-0496

ISSN (Print): 1099-0496

Publication date (Electronic): January 2021

Volume: 56

Issue: 1

Affiliations

[1 ] Pediatric Neurology and ICU Department, AP-HP Université Paris Saclay, DMU Santé de l'Enfant et de l'Adolescent, Hôpital Raymond Poincaré, Garches, France.

[2 ] Centre de Référence des Maladies Neuromusculaires Garches-Necker-Mondor-Hendaye (GNMH), Centre Nord- Est- Ile de France, Réseau National des Maladies Neuromusculaires, FILNEMUS, France.

[3 ] European Reference Center Network (Euro-NMD ERN), Paris, France.

[4 ] Pediatric Noninvasive Ventilation and Sleep Unit, AP-HP Hôpital Necker Enfants-Malades, Paris, France.

[5 ] Université de Paris, VIFASOM, Paris, France.

[6 ] ASV Santé, Gennevilliers, France.

[7 ] Pediatric Neurology Department, AP-HP Hôpital Necker Enfants Malades, Paris, France.

[8 ] Service de Néonatologie, Réanimation Pédiatrique, Neuropédiatrie et éDucation Fonctionnelle de l'enfant, CHU de Rouen, INSERM U 1245, ED497, Rouen, France.

[9 ] Pediatric Neurology Department, Centre de Référence des Maladies Neuromusculaires Atlantique Occitanie Caraïbes (AOC), Centre Hospitalier Universitaire, Angers, France.

[10 ] Radiology Department, AP-HP Université Paris Saclay, DMU Smart Imaging, Hôpital Raymond Poincaré, Garches, France.

[11 ] INSERM U1179, Université de Versailles Saint-Quentin (UVSQ), UFR des Sciences de la Santé Simone Vieil, Montigny, France.

Article

DOI: 10.1002/ppul.25142

PubMed ID: 33118682

SO-VID: 478bc990-4cce-4934-9094-a49a23a570b0

History

Keywords: spinal muscular atrophy,lung function,motor function,nusinersen,respiratory muscles

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Keywords: spinal muscular atrophy, lung function, motor function, nusinersen, respiratory muscles

Assessment of respiratory muscles and motor function in children with SMA treated by nusinersen.

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