Therapeutic Role of Nusinersen on Respiratory Progression in Pediatric Patients With Spinal Muscular Atrophy Type 2 and Nonambulant Type 3.
Federica TruccoDeborah RidoutHarriet WeststrateMariacristina ScotoAnnemarie RohwerGiorgia CorattiMarion L MainAnna G MayhewJacqueline MontesRoberto De SanctisMarika PaneMaria Carmela PeraValeria A SansoneEmilio AlbamonteAdele D'AmicoClaudio BrunoSonia S MessinaAnne-Marie ChildsTracey WillisMin T OngLaurent ServaisAnirban MajumdarImelda HughesChiara Marini-BettoloDeepak ParasuramanVasantha Lakshmi GowdaGiovanni BaranelloEnrico Silvio BertiniDarryl C De VivoBasil T DarrasJohn W DayOscar H MayerZarazuela Zolkipli-CunninghamRichard S FinkelEugenio MercuriFrancesco Muntoninull nullPublished in: Neurology. Clinical practice (2024)
This study provided Class III evidence that nusinersen slows progression for patients with SMA types 2 and 3 compared with a natural history cohort.
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