Evaluation of the therapeutic efficacy and tolerability of current drug treatments on the clinical outcomes of paediatric spinal muscular atrophy type 1: A systematic review

Respiratory morbidity in patients with spinal muscular atrophy—a changing world in the light of disease-modifying therapies

“…These results reflect again that starting early can maximize the effect and prevent ongoing motor neuron loss. Several single-center studies also showed a delayed introduction of ventilatory support in SMA type 1 patients when treated with nusinersen ( 55 ). In a recent study of Ergenekon et al none of the 52 patients with SMA type 1 were using NIV more than 16 h/day after 180 days of nusinersen ( 56 ).…”