This is an observational multicenter retrospective and prospective study on natural history of SMA also considering the 'new natural history' secondary to the availability of commercially available therapies.
All the patients enrolled to date in the Italian registry, if not part of clinical trials, will be included in the present study.
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Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Lead sponsor
University of Milan
Collaborator
Bambino Gesù Hospital and Research Institute
Collaborator
University of Messina
Collaborator
Gaslini Children's Hospital
Collaborator