L. Binetti

Stakeholder collaboration for spinal muscular atrophy therapy development

R. Finkel, E. Mercuri, F. Muntoni, 2017, The Lancet Neurology.

Expanded access program with Nusinersen in SMA type I in Italy: Strengths and pitfalls of a successful experience

E. Bertini, C. Palermo, M. Pane, 2017, Neuromuscular Disorders.