RECRUITINGOBSERVATIONAL
Long Term Trajectories of SMA Patients Receiving or Not Disease-modifying Treatments
SMA Natural History Study Evaluating Long Term Trajectories of SMA Patients Receiving or Not Disease-modifying Treatments
About This Trial
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.
Who May Be Eligible (Plain English)
Who May Qualify:
all patients with mutationsin the SMAN1 gene
Who Should NOT Join This Trial:
unable to proviude consent
Always talk to your doctor about whether this trial is right for you.
Original Eligibility Criteria
View original clinical language
Inclusion Criteria:
all patients with mutationsin the SMAN1 gene
Exclusion Criteria:
unable to proviude consent
Locations (5)
Istituto Gaslini
Genova, Italy
Nemo Sud
Messina, Italy
Centro Clinico nemo
Milan, Italy
Ospedale Bambino gesu
Rome, Italy
Policlinico gemelli
Rome, Italy