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Duchenne Muscular Dystrophy Heart Study
study id #: NCT03443115
condition: Duchenne Muscular Dystrophy, Duchenne Muscular Dystrophy-Associated Dilated Cardiomyopathy
Retrospective cohort study including patients with genetically proven Duchenne muscular dystrophy, diagnosed from January 1993 to March 2020. Inclusion of the data relative to genetic diagnosis, clinical characteristics at baseline, cardiac and respiratory workup, medical treatments (ACE inhibitors, steroids), surgical procedures, and occurrence during follow-up of cardiac, respiratory and fatal events. Objectives are to describe long-term natural history of the disease, vital prognosis, genotype-phenotype correlations, effect of treatments.
mechanism of action: No pharmaceutical intervention
last updated: October 19, 2020
start date: June 27, 2017
estimated completion: February 16, 2018
phase of development: N/A
size / enrollment: 700
- All-cause mortality [ Time Frame: Follow-up completed in March 2020 ]
- Hospitalization for heart failure [Time Frame: Follow-up completed in March 2020]
- Dilated cardiomyopathy [Time Frame: Follow-up completed in March 2020]
- Hospitalization for acute respiratory failure [Time Frame: Follow-up completed in March 2020]
• Eligible Sexes: male
• Genetically-proven Duchenne Muscular Dystrophy (DMD mutation)
• Covering by social security
• Patient refusal to participate to the study
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