phenotypeS in Non Ambulant Duchenne Muscular Dystrophy

Recruiting Observational Study
Duchenne Muscular Dystrophy Natural History Motor Function; Retardation
No Study Drug Researchers observe your health over time — no experimental treatment is given.
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At a Glance
Age
8 – 35
Sex
Male
Study type
Observational
Participants needed
250 (estimated)
Sponsor
Fondazione Policlinico Universitario Agostino Gemelli IRCCS · Other
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About This Trial

The aims of the study are to prospectively collect information on several aspects of function in non-ambulant DMD patients by using a structured battery of tests including motor, respiratory and cardiac function

Trial Locations
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Eligibility Criteria
Inclusion Criteria: * Children with genetically confirmed diagnosis of Duchenne Muscular Dystrophy will be included in the study. We will include all Duchenne Muscular Dystrophy boys who have lost the ability to walk independently. * All patients in whom consent can be obtained will be enrolled wit…
Contacts

Marika Pane, Prof

00390630156742

marika.pane@policlinicogemelli.it

CONTACT

Daniela Leone, MD

00390630158821

daniela.leone@policlinicogemelli.it

CONTACT