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Long Term Trajectories of SMA Patients Receiving or Not Disease-modifying Treatments
Sponsor: Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Summary
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.
Official title: SMA Natural History Study Evaluating Long Term Trajectories of SMA Patients Receiving or Not Disease-modifying Treatments
Key Details
Gender
All
Age Range
Any - Any
Study Type
OBSERVATIONAL
Enrollment
500
Start Date
2022-11-28
Completion Date
2027-11-27
Last Updated
2023-03-14
Healthy Volunteers
No
Conditions
Locations (5)
Istituto Gaslini
Genova, Italy
Nemo Sud
Messina, Italy
Centro Clinico nemo
Milan, Italy
Ospedale Bambino gesu
Rome, Italy
Policlinico gemelli
Rome, Italy