phenotypeS in Non Ambulant Duchenne Muscular Dystrophy

Launched by FONDAZIONE POLICLINICO UNIVERSITARIO AGOSTINO GEMELLI IRCCS · Apr 10, 2024

Keywords

ClinConnect Summary

This clinical trial is looking at boys with Duchenne Muscular Dystrophy (DMD) who can no longer walk independently. The main goal is to gather information about how this condition affects their motor skills, breathing, and heart function by using a series of structured tests. By understanding these aspects better, researchers hope to learn more about the natural progression of DMD in non-ambulant patients.

To participate in this study, boys aged 2 to 12 years who have been genetically diagnosed with DMD and can no longer walk on their own are eligible. There are no additional exclusion criteria, meaning that if they meet the age and diagnosis requirements, they can join the trial. Participants can expect to undergo various tests that will help researchers assess their physical abilities and health over time. This study is currently recruiting, and it aims to provide valuable insights that could help improve the care and support for those living with DMD.

Gender

MALE

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 with no exclusion criteria.
• Exclusion Criteria:
• • Patients lacking genetic confirmation of Duchenne Muscular Dystrophy
• • Patients still able to walk for more than 10 meters.