M. Pera

P.375 Clinical studies of RG7916 in patients with spinal muscular atrophy: SUNFISH part 1 study update

E. Mercuri, L. Servais, N. Goemans, 2017, Neuromuscular Disorders.

Predominant distal muscle involvement in spinal muscular atrophy

M. Pane, E. Mercuri, A. Pichiecchio, 2019, Neuromuscular Disorders.

Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy type 1 (STR1VE-EU): an open-label, single-arm, multicentre, phase 3 trial

S. Corti, M. Pane, E. Mercuri, 2021, The Lancet Neurology.

1st Italian SMA Family Association Consensus Meeting:: Management and recommendations for respiratory involvement in spinal muscular atrophy (SMA) types I–III, Rome, Italy, 30–31 January 2015

M. Pane, I. Salvo, C. Gregoretti, 2015, Neuromuscular Disorders.