Long Term Trajectories of SMA Patients Receiving or Not Disease-modifying Treatments
Recruiting
Observational Study
Spinal Muscular Atrophy
No Study Drug
Researchers observe your health over time — no experimental treatment is given.
At a Glance
- Sex
- Any
- Study type
- Observational
- Participants needed
- 500 (estimated)
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS · Other
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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.
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
all patients with mutationsin the SMAN1 gene
Exclusion Criteria:
unable to proviude consent
Contacts