The X-linked Duchenne and Becker muscular dystrophies (DMD and BMD) are allelic disorders
occurring due to mutations in the DMD gene, which consists of 79 exons encoding the …

Duchenne muscular dystrophy (DMD) is an X-linked recessive disorder in which the loss of
dystrophin causes progressive degeneration of skeletal and cardiac muscle. Potential …

Duchenne and Becker muscular dystrophy (DMD and BMD) are related disorders that occur
because of mutations in the DMD gene, encoding the dystrophin protein. DMD is more …

Objective In prior open‐label studies, eteplirsen, a phosphorodiamidate morpholino oligomer,
enabled dystrophin production in Duchenne muscular dystrophy (DMD) with genetic …

Objective: Creatine kinase (CK) levels are increased on dried blood spots in newborns
related to the birthing process. As a marker for newborn screening, CK in Duchenne muscular …

Analyzing the type and frequency of patient‐specific mutations that give rise to Duchenne
muscular dystrophy (DMD) is an invaluable tool for diagnostics, basic scientific research, trial …

Objective Myostatin is an endogenous negative regulator of muscle growth and a novel target
for muscle diseases. We conducted a safety trial of a neutralizing antibody to myostatin, …

The primary muscle disorders are a diverse group of diseases caused by various defective
structural proteins, abnormal signaling molecules, enzymes and proteins involved in …

Introduction: Dystrophinopathy is a rare, severe muscle disorder, and nonsense mutations
are found in 13% of cases. Ataluren was developed to enable ribosomal readthrough of …

Background Duchenne muscular dystrophy (DMD) is a severe, progressive, and rare
neuromuscular, X-linked recessive disease. Dystrophin deficiency is the underlying cause of …