Simon Sander AlbrechtsenAlfred Peter BornMagnus Spangsberg Boesen2026-03-222026-03-222020https://pubmed.ncbi.nlm.nih.gov/32800069https://andeanlibrary.org/handle/123456789/44789Citaciones: 44Nusinersen increased survival without permanent ventilatory support in children with SMA type 1. Improvements in SMA type 2 and 3 were less evident. Better outcomes were seen in young children with a short disease duration, particularly in children receiving nusinersen before symptom onset. Newborn SMA screening may facilitate presymptomatic treatment with splice modification (nusinersen, risdiplam) or gene implantation therapy (AVXS-101, zolgensma).enSMA*MedicineSpinal muscular atrophyCohortNeuromuscular diseasePediatricsCohort studyRandomized controlled trialSurgeryPhysical therapyarticle