E. Maczek

Safety and efficacy of risdiplam in patients with type 1 spinal muscular atrophy (FIREFISH part 2): secondary analyses from an open-label trial

G. Comi, C. Palermo, M. Pane, 2022, The Lancet Neurology.

Assessing Bulbar Function in Spinal Muscular Atrophy Using Patient-Reported Outcomes

M. Mcdermott, J. Day, W. Martens, 2023, Journal of neuromuscular diseases.

Risdiplam in Patients Previously Treated with Other Therapies for Spinal Muscular Atrophy: An Interim Analysis from the JEWELFISH Study

R. Finkel, M. Pane, A. Mayhew, 2023, Neurology and Therapy.