Duchenne Muscular Dystrophy Clinical Trial
Official title:
Multicenter Cohort Study on Duchenne Muscular Dystrophy 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.
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