Determining minimal clinically important differences in the Hammersmith Functional Motor Scale Expanded for untreated spinal muscular atrophy patients: An international study.

Giorgia CorattiFrancesca BovisMaria Carmela PeraMariacristina ScotoJacqueline MontesAmy PasternakAnna MayhewRobert Muni-LofraTina DuongAnnemarie RohwerSally Dunaway YoungMatthew CivitelloFrancesca SalminIrene MizzoniSimone MorandoMarika PaneEmilio AlbamonteAdele D'AmicoNoemi BrolattiMaria SframeliChiara Marini-BettoloValeria Ada SansoneClaudio BrunoSonia MessinaEnrico BertiniGiovanni BaranelloJohn DayBasil T DarrasDarryl C De VivoMichio HiranoFrancesco MuntoniRichard FinkelEugenio Mercurinull null

Published in: European journal of neurology (2024)

These results emphasize that the interpretation of a single MCID or MDC value obtained in large cohorts with different functional status needs to be made with caution, especially when these may be used to assess possible responses to new therapies.

Keyphrases

end stage renal disease
ejection fraction
chronic kidney disease
newly diagnosed
prognostic factors
peritoneal dialysis
patient reported outcomes