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.
Study Type
OBSERVATIONAL
Enrollment
700
Cochin Reference Center for Neuromuscular Diseases
Paris, France
RECRUITINGAll-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
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