V. Tahon

Natural history of Type 1 spinal muscular atrophy: a retrospective, global, multicenter study

G. Comi, E. Zanoteli, N. Goemans, 2022, Orphanet Journal of Rare Diseases.

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.

Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled trial

G. Comi, S. Corti, E. Mazzone, 2022, The Lancet Neurology.

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.

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.

Nusinersen versus Sham Control in Infantile‐Onset Spinal Muscular Atrophy

R. J. Ramamurthi, S. Y. Kim, H. Y. Wang, 2017, The New England journal of medicine.

Patients’ Perceptions of Nusinersen Effects According to Their Responder Status

V. Tahon, Laurent Servais, Charlotte Lilien, 2024, Journal of clinical medicine.