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

Gómez-García de la Banda M, Amaddeo A, Khirani S, Pruvost S, Barnerias C, Dabaj I, Bénézit A, Durigneux J, Carlier RY, Desguerre I, Quijano-Roy S, Fauroux B.
Pediatr Pulmonol. 2021. 56(1):299-306.
Respiratory muscles performance and lung function were evaluated in children with SMA 1 and 2 after six injections of nusinersen (M14). Treated subjects were assessed with MFM and HINE-2 at baseline and M14.
A significant improvement in MFM and HINE-2 was observed at M14 as compared with baseline.

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Keywords: lung function; motor function; nusinersen; respiratory muscles; spinal muscular atrophy.

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