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    • 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 (2016-02)
      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 ...