Browsing by Author "Pera, Maria C"

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    • Content validity and clinical meaningfulness of the HFMSE in spinal muscular atrophy 

      Pera, Maria C; Coratti, Giorgia; Forcina, Nicola; Mazzone, Elena S; Scoto, Mariacristina; Montes, Jacqueline; Pasternak, Amy; Mayhew, Anna; Messina, Sonia; Sframeli, Maria; Main, Marion; Lofra, Robert Muni; Duong, Tina; Ramsey, Danielle; Dunaway, Sally; Salazar, Rachel; Fanelli, Lavinia; Civitello, Matthew; de Sanctis, Roberto; Antonaci, Laura; Lapenta, Leonardo; Lucibello, Simona; Pane, Marika; Day, John; Darras, Basil T; De Vivo, Darryl C; Muntoni, Francesco; Finkel, Richard; Mercuri, Eugenio (2017-02-23)
      BACKGROUND: Reports on the clinical meaningfulness of outcome measures in spinal muscular atrophy (SMA) are rare. In this two-part study, our aim was to explore patients' and caregivers' views on the clinical relevance of ...