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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: December 04, 2019
start date: June 27, 2017
estimated completion: February 16, 2018
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]
• Genetically-proven Duchenne Muscular Dystrophy (DMD mutation)
• Covering by social security
• Patient refusal to participate to the study
Dystrophin-Deficient CardiomyopathyDystrophinopathies are a group of distin...
Stabilization of the Cardiac Sarcolemma by Sarcospan Rescues DMD-Associated CardiomyopathyIn the current preclinical study, we dem...
Antisense Oligonucleotides for the Treatment of Cardiomyopathy in Duchenne Muscular DystrophyDuchenne muscular dystrophy (DMD) is an ...
Eplerenone for early cardiomyopathy in Duchenne muscular dystrophy: a randomised, double-blind, placebo-controlled t...Background: Cardiomyopathy is a leading...
MLPA Analyses Reveal a Spectrum of Dystrophin Gene Deletions/Duplications in Pakistani Patients Suspected of Having ...Introduction: Duchenne muscular dystr...
Duchenne and Becker Muscular Dystrophies: Mutations and Potential Therapieshttps://pdfs.semanticscholar.org/4f9d/71...
Small Mutation Screening in the DMD Gene by Whole Exome Sequencing of an Argentine Duchenne/Becker Muscular Dystroph...Dystrophinopathies are neuromuscular X-l...