Immunadsorption therapy for end stage heart failure due to Duchenne muscular dystrophy

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Immunadsorption therapy for end stage heart failure due to Duchenne muscular dystrophy

We report about a successful immunadsorption therapy of a boy with end stage heart failure due to Duchenne muscular dystrophy who has little chance to get cardiac transplantation. Prior to this therapy a medical therapy with an angiotensin converting enzyme inhibitor, a low dose betablocker, an aldosterone antagonist, and diuretics failed. In consent with the patient and his parents immunoadsor...

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Duchenne Muscular Dystrophy: From Diagnosis to Therapy.

Duchenne muscular dystrophy (DMD) is an X-linked inherited neuromuscular disorder due to mutations in the dystrophin gene. It is characterized by progressive muscle weakness and wasting due to the absence of dystrophin protein that causes degeneration of skeletal and cardiac muscle. The molecular diagnostic of DMD involves a deletions/duplications analysis performed by quantitative technique su...

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Exon skipping therapy for Duchenne muscular dystrophy.

Duchenne muscular dystrophy (DMD) is caused mostly by internal deletions in the gene for dystrophin, a protein essential for maintaining muscle cell membrane integrity. These deletions abrogate the reading frame and the lack of dystrophin results in progressive muscle deterioration. DMD patients experience progressive loss of ambulation, followed by a need for assisted ventilation, and eventual...

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Duchenne muscular dystrophy An overview of Duchenne muscular dystrophy

Duchenne muscular dystrophy (DMD) affects approximately 1 in 3,500 live male births [1]. It is caused by a large variety of mutations in the dystrophin gene. Because of these mutations, the body can no longer make dystrophin which is a protein important for stabilisation of the muscle cell during a contraction. Without dystrophin, muscle cells are damaged and slowly replaced by fat and scar tis...

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DMD certainly has a remarkable position amongst all hereditary muscle disorders. Noteworthy is its relatively high incidence as a " rare disease " , its X-chromosomal mode of inheritance, the high frequency of new mutations , as well as its relatively rapid and fatal course. For a century the disorder was considered to be untreatable. Only 40 years ago the patients' " natural " age at death was...

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ژورنال

عنوان ژورنال: Open Journal of Pediatrics

سال: 2013

ISSN: 2160-8741,2160-8776

DOI: 10.4236/ojped.2013.31003