Duchenne muscular dystrophy (DMD) is a severe, progressive, and incurable X-linked
disorder caused by mutations in the dystrophin gene. Patients with DMD have an absence of …

Importance Thoracic aortic aneurysms leading to acute aortic dissections are a major cause
of morbidity and mortality despite significant advances in surgical treatment, which remains …

Rationale: Duchenne muscular dystrophy is a severe inherited form of muscular dystrophy
caused by mutations in the reading frame of the dystrophin gene disrupting its protein …

Familial dilated cardiomyopathy (DCM) is mostly caused by mutations in genes encoding
cytoskeletal and sarcomeric proteins. In the pediatric population, DCM is the predominant …

Aims Heart disease is recognized as the leading cause of morbidity and mortality in patients
with muscular dystrophy (MD). Our study demonstrates the clinical utility of cardiac …

Duchenne muscular dystrophy (DMD) is a rare X‐linked recessive disorder characterized by
loss‐of‐function mutations in the gene encoding dystrophin. These mutations lead to …

Background The aim of this study was to pool multiple data sets to build a patient-centric,
data-informed, natural history model (NHM) for Duchenne muscular dystrophy (DMD) to …

In this retrospective cohort study, we characterize the health profile of preterm males with
Duchenne muscular dystrophy. Major clinical milestones (ambulation cessation, assisted …

Background Muscular dystrophy (MD) is a group of inherited neuromuscular disorders with
heart disease as a leading cause of morbidity and mortality. Dystrophinopathies such as …

Numerous factors make the initial diagnostic evaluation of children with suspected arterial
ischemic stroke (AIS) a relatively unsettling challenge, even for the experienced stroke …