Patient and parent oriented tools to assess health-related quality of life, activity of daily living and caregiver burden in SMA. Rome, 13 July 2019

Twenty-five participants from Italy, United States, United Kingdom, Germany, Spain and Switzerland met to discuss Patient Reported Outcome Measures (PROM) in Spinal Muscular atrophy (SMA). The group included physicians and physical therapists with a specific experience in developing or validating PROMS in neuromuscular disorders, advocacy groups and representatives from pharmaceutical companies.

Source: Neuromuscular Disorders - March 5, 2020 Category: Neurology Authors: Eugenio Mercuri, Sonia Messina, Jacqueline Montes, Amy Pasternak, Erik Henricson, Anna Lia Frongia, Mencia de Lemus, Nicole Gusset, Mary Schroth, Ksenija Gorni, Marcus Droege, Ivana Rubino, Francesco Muntoni, Valeria A Sansone, SMA PROM working group Tags: Workshop report Source Type: research