Amy Wolfe, Georgia Stimpson, Danielle Ramsey, Giorgia Coratti, Sally Dunaway Young, Anna Mayhew, Marika Pane, Annemarie Rohwer, Robert Muni Lofra, Tina Duong, Emer O'Reilly, Evelin Milev, Matthew Civitello, Valeria A Sansone, Adele D'Amico, Enrico Bertini, Sonia Messina, Claudio Bruno, Emilio Albamonte, Elena Mazzone, Marion Main, Jacqueline Montes, Allan M Glanzman, Zarazuela Zolkipli-Cunningham, Amy Pasternak, Chiara Marini-Bettolo, John W Day, Basil T Darras, Darryl C De Vivo, Giovanni Baranello, Mariacristina Scoto, Richard S Finkel, Eugenio Mercuri, Francesco Muntoni
BACKGROUND: Spinal muscular atrophy (SMA) is a neuromuscular disorder characterised by progressive motor function decline. Motor function is assessed using several functional outcome measures including the Revised Hammersmith Scale (RHS). OBJECTIVE: In this study, we present longitudinal trajectories for the RHS in an international cohort of 149 untreated paediatric SMA 2 and 3 patients (across 531 assessments collected between March 2015 and July 2019). METHODS: We contextualise these trajectories using both the Hammersmith Functional Motor Scale Expanded (HFMSE) and Revised Upper Limb Module (RULM)...
February 27, 2024: Journal of Neuromuscular Diseases