Content validity and clinical meaningfulness of the HFMSE in spinal muscular atrophy.
Maria C PeraGiorgia CorattiNicola ForcinaElena S MazzoneMariacristina ScotoJacqueline MontesAmy PasternakAnna MayhewSonia MessinaMaria SframeliMarion MainRobert Muni LofraTina DuongDanielle RamseySally DunawayRachel SalazarLavinia FanelliMatthew CivitelloRoberto de SanctisLaura AntonaciLeonardo LapentaSimona LucibelloMarika PaneJohn DayBasil T DarrasDarryl C De VivoFrancesco MuntoniRichard FinkelEugenio MercuriPublished in: BMC neurology (2017)
Our findings support the use of the HFMSE as a key outcome measure in SMA clinical trials because the individual items and the detected changes have clear content validity and clinical meaningfulness for patients and their caregivers.