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

ConditionSpinal Muscular Atrophy
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
AgeNot listed
SponsorFondazione Policlinico Universitario Agostino Gemelli IRCCS

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

None

Disqualifiers

None

Trial population

All patients affected by Spinal Muscular Atrophy 5q, irrespective of genotype, phenotype, age, treatment status or gender

Trial design

Design model

Cohort

Time perspective

Prospective

Treatments tested in this trial

Not listed

Trial groups

No trial groups listed

Trial outcomes

Primary outcomes

1

motor function using the HFMSE (min score 0, max 74 indicating best performance)

motor scale

Time frame
5 years

Secondary outcomes

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Fondazione Policlinico Universitario Agostino Gemelli IRCCS

Lead sponsor

University of Milan

Collaborator

Bambino Gesù Hospital and Research Institute

Collaborator

University of Messina

Collaborator

Gaslini Children's Hospital

Collaborator

Source ClinicalTrials.gov
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