Long Term Trajectories of SMA Patients Receiving or Not Disease-modifying Treatments

Recruiting now

Conditions studied: Spinal Muscular Atrophy

In brief

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.

Key facts

Study ID

NCT05768048

Run by

Fondazione Policlinico Universitario Agostino Gemelli IRCCS

People needed

500

Starts

2022-11-28

Expected to finish

2027-11-27

Last updated by the study team

2023-03-14

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• all patients with mutationsin the SMAN1 gene

You may not qualify if…

• unable to proviude consent

Where it is running

• Istituto Gaslini — Genova, Italy (enrolling)
• Nemo Sud — Messina, Italy (enrolling)
• Centro Clinico nemo — Milan, Italy (enrolling)
• Ospedale Bambino gesu — Rome, Italy (enrolling)
• Policlinico gemelli — Rome, Italy (enrolling)

Full record on ClinicalTrials.gov

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