Browsing by Autor "Alfred Peter Born"

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    Nusinersen treatment of spinal muscular atrophy - a systematic review.
    (National Institutes of Health, 2020) Simon Sander Albrechtsen; Alfred Peter Born; Magnus Spangsberg Boesen
    Nusinersen 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).