Nusinersen treatment of spinal muscular atrophy - a systematic review.

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dc.contributor.authorSimon Sander Albrechtsen
dc.contributor.authorAlfred Peter Born
dc.contributor.authorMagnus Spangsberg Boesen
dc.coverage.spatialBolivia
dc.date.accessioned2026-03-22T14:08:34Z
dc.date.available2026-03-22T14:08:34Z
dc.date.issued2020
dc.descriptionCitaciones: 44
dc.description.abstractNusinersen 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).
dc.identifier.urihttps://pubmed.ncbi.nlm.nih.gov/32800069
dc.identifier.urihttps://andeanlibrary.org/handle/123456789/44789
dc.language.isoen
dc.publisherNational Institutes of Health
dc.relation.ispartofPubMed
dc.sourceUniversity of San Simón
dc.subjectSMA*
dc.subjectMedicine
dc.subjectSpinal muscular atrophy
dc.subjectCohort
dc.subjectNeuromuscular disease
dc.subjectPediatrics
dc.subjectCohort study
dc.subjectRandomized controlled trial
dc.subjectSurgery
dc.subjectPhysical therapy
dc.titleNusinersen treatment of spinal muscular atrophy - a systematic review.
dc.typearticle

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