Muscular dystrophy classification and diagnosis has been transformed over the 13 or so
years since the cloning of the dystrophin gene (involved in Duchenne and Becker muscular …

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Clinical trials represent a critical avenue for new treatment development, where early
phases (I, I/II) are designed to test safety and effectiveness of new therapeutics or diagnostic …

Duchenne muscular dystrophy (DMD) is an X-linked recessive disease caused by the lack of
expression of the dystrophin protein in muscle tissues. We genetically engineered a mouse …

With the discovery of the X-linked gene product dystrophin, the mdx mouse came to be
regarded as the only suitable mouse model of human muscular dystrophy. However …

Despite intensive research efforts, the cause of the muscular dystrophies has remained
elusive for many decades. In the late 1980s, major advances in molecular genetics have led …

Identification of new genes involved in muscle disorders has dramatically changed the
traditional clinical classification of the large and heterogeneous group of the muscular …

The 14 kb muscle isoform of the Duchenne muscular dystrophy (DMD) gene is expressed
primarily in skeletal and cardiac muscle. Transcription of the muscle isoform is induced as …

Muscular dystrophy is a group of genetically determined muscular disorders marked by
progressive wasting and weakness of the skeletal muscle, but which often affect cardiac and …

The outstanding advances in the molecular characterization of muscle diseases, including
muscular dystrophies, inflammatory myopathies, and ion channel disorders, have resulted in …