Since the publication of the Duchenne muscular dystrophy (DMD) care considerations in
2010, multidisciplinary care of this severe, progressive neuromuscular disease has evolved …

A coordinated, multidisciplinary approach to care is essential for optimum management of
the primary manifestations and secondary complications of Duchenne muscular dystrophy …

Improvements in the function, quality of life, and longevity of patients with Duchenne
muscular dystrophy (DMD) have been achieved through a multidisciplinary approach to …

Background Duchenne muscular dystrophy (DMD) is the most common muscular dystrophy
of childhood. Untreated, this incurable disease, which has an X‐linked recessive …

Objective To continue evaluation of the long‐term efficacy and safety of eteplirsen, a
phosphorodiamidate morpholino oligomer designed to skip DMD exon 51 in patients with …

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

Duchenne muscular dystrophy (DMD) is a severe, progressive disease that affects 1 in 3600–
6000 live male births. Although guidelines are available for various aspects of DMD …

Objective To describe the quantification of novel dystrophin production in patients with
Duchenne muscular dystrophy (DMD) after long-term treatment with eteplirsen. Methods …

Importance Corticosteroids improve strength and function in boys with Duchenne muscular
dystrophy. However, there is uncertainty regarding the optimum regimen and dosage …

Muscular dystrophies are a heterogeneous group of inherited disorders that share similar
clinical features and dystrophic changes on muscle biopsy. An improved understanding of …