Actively Recruiting
Long Term Trajectories of SMA Patients Receiving or Not Disease-modifying Treatments
Led by Fondazione Policlinico Universitario Agostino Gemelli IRCCS · Updated on 2023-03-14
500
Participants Needed
5
Research Sites
260 weeks
Total Duration
On this page
Sponsors
F
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Lead Sponsor
U
University of Milan
Collaborating Sponsor
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Long Term Trajectories of SMA Patients Receiving or Not Disease-modifying Treatments
Who Can Participate
Eligibility Criteria
You may qualify if you...
- All patients with mutations in the SMN1 gene
You will not qualify if you...
- Unable to provide consent
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 5 locations
1
Istituto Gaslini
Genova, Italy
Actively Recruiting
2
Nemo Sud
Messina, Italy
Actively Recruiting
3
Centro Clinico nemo
Milan, Italy
Actively Recruiting
4
Ospedale Bambino gesu
Rome, Italy
Actively Recruiting
5
Policlinico gemelli
Rome, Italy
Actively Recruiting
Research Team
E
Eugenio Mercuri, MD
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
0
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