Natural History of SMA

This is an investigator initiated observational study with the aim to record several aspects of function, care and adverse events in a large cohort of SMA patients followed longitudinally by using a structured academic disease registry.

NCT Number	NCT05755451
Lead Sponsor	Fondazione Policlinico Universitario Agostino Gemelli IRCCS Collaborators: University of Milan, Istituto Giannina Gaslini, Ospedale Pediatrico Bambin Gesù, University of Messina
Conditions	Muscular Atrophy, Spinal
Enrollment	1,200 participants
Start Date	2018-06-21
Primary Completion	2033-12-30 (estimated)
Study Completion	2033-12-30 (estimated)
Updated on ClinicalTrials.gov	2023-03-06