Conquering Diseases

A Study Of The Natural History Of Duchenne Muscular Dystrophy In Boys

Description

Seeking boys aged 5-9 years of age who have been diagnosed with Duchenne Muscular Dystrophy (DMD) to learn more about the natural course of the disease.

Overview

The aim of this study is to better understand Duchenne Muscular Dystrophy (DMD) in young boys by recording very accurately specific data related to Duchenne disease and, therefore, to better understand and anticipate the disease course evolution. Participating children will be followed regularly to assess in particular his muscular, cardiac and respiratory symptoms, but also their quality of life, with dedicated tests and scales.

What we're hoping for

To learn more about the course children's Duchenne Muscular Dystrophy (DMD). Such a study is particularly important in the perspective of future therapeutic clinical studies that might be proposed to people presenting Duchenne disease.

Additional Information

ClinicalTrials.gov Identifier: NCT03882827

 Principal Investigator

Brenda  Wong, MD

UMass Memorial Health Care

 Study Contact

Sarah  Figueira

508-856-1604

sarah.figueira@umassmed.edu

 Location

UMMS University Campus

55 Lake Ave North 

Worcester,MA 01655

508-856-8989