Assessment of respiratory muscles and motor function in children with SMA treated by nusinersen.
Marta Gómez-García de la BandaAlessandro AmaddeoSonia KhiraniSandrine PruvostChristine BarneriasIvana DabajAudrey BénézitJulien DurigneuxRobert Y CarlierIsabelle DesguerreSusana Quijano-RoyBrigitte FaurouxPublished in: Pediatric pulmonology (2020)
In children with SMA Type 2, respiratory muscle performance was significantly better after six injections of nusinersen as compared with age-matched SMA Type 2 historical controls.