Efficacy and safety of nusinersen in children with later-onset spinal muscular atrophy (SMA): end of study results from the phase 3 CHERISH study

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The efficacy and safety of nusinersen, an antisense oligonucleotide, in children with later-onset SMA was assessed in CHERISH, a phase 3, multicenter, randomized, double-blind, sham-procedure controlled study (NCT02292537). Children aged 2–12 years with onset of SMA clinical symptoms at age >6 months and confirmed 5q SMA were enrolled and randomized (2:1 intrathecal nusinersen 12 mg vs sham-control, stratified based on screening age <6 versus ≥6 years; 4 dosing events over 15 months). Primary endpoint: change from baseline to month 15 in the Hammersmith Functional Motor Scale–Expanded (HFMSE) total score.

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