Long Term Trajectories of SMA Patients Receiving or Not Disease-modifying Treatments

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.

NCT Number	NCT05768048
Lead Sponsor	Fondazione Policlinico Universitario Agostino Gemelli IRCCS Collaborators: University of Milan, Bambino Gesù Hospital and Research Institute, University of Messina, Gaslini Children's Hospital
Conditions	Spinal Muscular Atrophy
Enrollment	500 participants
Start Date	2022-11-28
Primary Completion	2027-11-27 (estimated)
Study Completion	2027-11-27 (estimated)
Updated on ClinicalTrials.gov	2023-03-14