Duchenne muscular dystrophy is a severe, progressive, muscle-wasting disease that leads
to difficulties with movement and, eventually, to the need for assisted ventilation and …

Duchenne muscular dystrophy (DMD) cardiomyopathy (DCM) is characterized by a
hypokinetic, dilated phenotype progressively increasing with age. Regular cardiac care is …

Duchenne muscular dystrophy (DMD) is characterized by rapid wasting of skeletal muscle.
Mitochondrial dysfunction is a well-known pathological feature of DMD. However, whether …

Adeno-associated virus–mediated (AAV-mediated) CRISPR editing is a revolutionary
approach for treating inherited diseases. Sustained, often life-long mutation correction is …

Large-animal models for Duchenne muscular dystrophy (DMD) are crucial for the evaluation
of diagnostic procedures and treatment strategies. Pigs cloned from male cells lacking DMD …

Advancements in therapies for Duchenne muscular dystrophy (DMD) have made diagnosis
within the newborn period a high priority. We undertook a consortia approach to advance …

Spinal muscular atrophy (SMA) is one of the major genetic disorders associated with infant
mortality. More than 90% of cases of SMA result from deletions of or mutations in the …

Duchenne muscular dystrophy (DMD) is a fatal X-linked recessive condition caused
primarily by out-of-frame mutations in the dystrophin gene. In males, DMD presents with …

In this article, we outline a comprehensive plan for the psychosocial management of patients
with Duchenne muscular dystrophy (DMD) across the life span. In 2010, the Centers for …

Dystrophinopaties, eg, Duchenne muscular dystrophy (DMD), Becker muscular dystrophy
and X-linked dilated cardiomyopathy are inherited neuromuscular diseases, characterized …