Medical research

Using CRISPR to find muscular dystrophy treatments

CRISPR-Cas9 gene editing technology is best known for its potential role in correcting genetic diseases. But it can also be used as a tool to find genes that act as supporting players, making the disease better or worse. ...

Medical research

Cancer cachexia: Extracellular ligand helps to prevent muscle loss

Cancer cachexia is a complex metabolic disease accounting for approximately one third of all cancer-related deaths worldwide. So far, there is no effective therapy for this muscle wasting disease. Researchers from the Leibniz ...

Genetics

New gene correction therapy for Duchenne muscular dystrophy

Duchenne type muscular dystrophy (DMD) is the most common hereditary muscular disease among children, leaving them wheelchair-bound before the age of 12 and reducing life expectancy. Researchers at Technical University of ...

Medical research

New treatment for muscular dystrophy wins US regulatory approval

Research led by Professor Steve Wilton and Professor Sue Fletcher and licensed to Sarepta Therapeutics has delivered a second treatment for Duchenne muscular dystrophy, with the drug gaining accelerated approval by the U.S. ...

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Muscular dystrophy

Muscular dystrophy (abbreviated MD) refers to a group of genetic, hereditary muscle diseases that weaken the muscles that move the human body. Muscular dystrophies are characterized by progressive skeletal muscle weakness, defects in muscle proteins, and the death of muscle cells and tissue. Nine diseases including Duchenne, Becker, limb girdle, congenital, facioscapulohumeral, myotonic, oculopharyngeal, distal, and Emery-Dreifuss are always classified as muscular dystrophy but there are more than 100 diseases in total with similarities to muscular dystrophy. Most types of MD are multi-system disorders with manifestations in body systems including the heart, gastrointestinal and nervous systems, endocrine glands, skin, eyes and other organs.

In the 1860s, descriptions of boys who grew progressively weaker, lost the ability to walk, and died at an early age became more prominent in medical journals. In the following decade, French neurologist Guillaume Duchenne gave a comprehensive account of 13 boys with the most common and severe form of the disease (which now carries his name — Duchenne muscular dystrophy). It soon became evident that the disease had more than one form, and that these diseases affected males of all ages.[citation needed]

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