An observational cohort study on pulmonary function in adult patients with 5q-spinal muscular atrophy under nusinersen therapy.
Bogdan BjelicaCamilla WohnradeAlma OsmanovicOlivia Schreiber-KatzSusanne PetriPublished in: Journal of neurology (2023)
Mean pulmonary function remained stable during nusinersen treatment over a period of up to 30 months. Improvement in pulmonary function was associated with improvement in motor function, fatigue and QoL, early after nusinersen initiation.