Now showing items 1-2 of 2

    • 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 ...
    • Patterns of disease progression in type 2 and 3 SMA : Implications for clinical trials 

      Mercuri, Eugenio; Finkel, Richard; Montes, Jacqueline; Mazzone, Elena S; Sormani, Maria Pia; Main, Marion; Ramsey, Danielle; Mayhew, Anna; Glanzman, Allan M; Dunaway, Sally; Salazar, Rachel; Pasternak, Amy; Quigley, Janet; Pane, Marika; Pera, Maria Carmela; Scoto, Mariacristina; Messina, Sonia; Sframeli, Maria; Vita, Gian Luca; D'Amico, Adele; van den Hauwe, Marleen; Sivo, Serena; Goemans, Nathalie; Kaufmann, Petra; Darras, Basil T; Bertini, Enrico; Muntoni, Francesco; De Vivo, Darryl C (2015-12-03)
      The aim of the study was to establish 12-month changes in the Hammersmith Functional motor scale in a large cohort of SMA patients, to identify patterns of disease progression and the effect of different variables. 268 ...