SMA Natural History Study Evaluating Long Term Trajectories of SMA Patients Receiving or Not Disease-modifying Treatments
Status: Recruiting
Location: See all (5) locations...
Study Type: Observational
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.
Eligibility
Participation Requirements
Sex: All
Healthy Volunteers: f
View:
• all patients with mutationsin the SMAN1 gene
Locations
Other Locations
Italy
Istituto Gaslini
RECRUITING
Genova
Nemo Sud
RECRUITING
Messina
Centro Clinico nemo
RECRUITING
Milan
Ospedale Bambino gesu
RECRUITING
Rome
Policlinico gemelli
RECRUITING
Rome
Contact Information
Primary
Eugenio Mercuri, MD
eugeniomaria.mercuri@policlinicogemelli.it
063015
Time Frame
Start Date: 2022-11-28
Estimated Completion Date: 2027-11-27
Participants
Target number of participants: 500
Related Therapeutic Areas
Sponsors
Collaborators: University of Milan, University of Messina, Gaslini Children's Hospital, Bambino Gesù Hospital and Research Institute
Leads: Fondazione Policlinico Universitario Agostino Gemelli IRCCS