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Clinical Trial Summary

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


Clinical Trial Description

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, to retrospectively review similar information on the data collected in the last decade and to establish the effect of steroids after loss of ambulation on different aspects of function. We also aim to use this integrated approach to identify patterns of severity and progression, the most appropriate outcome measures and endpoints in each group and possible genotype/phenotype correlations. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT06366815
Study type Observational [Patient Registry]
Source Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Contact Marika Pane, Prof
Phone 00390630156742
Email marika.pane@policlinicogemelli.it
Status Recruiting
Phase
Start date August 30, 2022
Completion date December 30, 2025

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