Dystrophinopathies are a group of distinct neuromuscular diseases that result from
mutations in the structural cytoskeletal Dystrophin gene. Dystrophinopathies include …

For many neuromuscular diseases (NMDs), cardiac disease represents a major cause of
morbidity and mortality. The management of cardiac disease in NMDs is made challenging …

Importance Micro-dystrophin gene transfer shows promise for treating patients with
Duchenne muscular dystrophy (DMD) using recombinant adeno-associated virus serotype …

The classes of recommendations and levels of evidence in these guidelines were
determined and described according to the “Guidelines for the treatment of acute and …

Pediatric heart failure (HF) is an important cause of morbidity and mortality in childhood. This
article presents guidelines for the recognition, diagnosis, and early medical management of …

Skeletal muscle or cardiac symptoms are known to appear in a certain proportion of female
patients carrying the dystrophin gene mutation. There is limited high-quality evidence to …

Muscular dystrophy (MD) connotes a heterogeneous group of inherited disorders
characterized by progressive wasting and weakness of the skeletal muscles. In several …

Aims Only few data are available regarding a direct comparison of both non-invasive CMR
and invasive EMB with respect to conformity of procedure-derived diagnoses in the same …

Background Patients with Duchenne muscular dystrophy exhibit progressive cardiac and
skeletal muscle dysfunction. Based on prior data, cardiac dysfunction in Duchenne muscular …

OBJECTIVE: The aim of this study was to determine in pediatric Duchenne (DMD) and
Becker muscular dystrophy (BMD) or other dilated cardiomyopathies (ODCM) whether …