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RecruitingNCT05768048

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

Sponsor

Fondazione Policlinico Universitario Agostino Gemelli IRCCS

Enrollment

500 participants

Start Date

Nov 28, 2022

Study Type

OBSERVATIONAL

Conditions

Spinal Muscular Atrophy

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

Inclusion Criteria1

  • all patients with mutationsin the SMAN1 gene

Exclusion Criteria1

  • unable to proviude consent

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Locations(5)

Istituto Gaslini

Genova, Italy

Nemo Sud

Messina, Italy

Centro Clinico nemo

Milan, Italy

Ospedale Bambino gesu

Rome, Italy

Policlinico gemelli

Rome, Italy

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NCT05768048

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