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. ...

Medical research

CRISPR halts Duchenne muscular dystrophy progression in dogs

Scientists for the first time have used CRISPR gene editing to halt the progression of Duchenne muscular dystrophy (DMD) in a large mammal, according to a study by UT Southwestern that provides a strong indication that a ...

Medical research

A golden ticket to faster muscle recovery

Anyone who has ever torn or injured a muscle knows that swelling, redness, and pain soon follow the injury: classic signs of inflammation. Inflammation is the body's natural response to promote healing, but prolonged, excess ...

Genetics

CRISPR treats genetic disorder in adult mammal

Researchers have used CRISPR to treat an adult mouse model of Duchenne muscular dystrophy. This marks the first time that CRISPR has successfully treated a genetic disease inside a fully developed living mammal with a strategy ...

Medications

New hope for antibody to treat muscular dystrophy

Northwestern Medicine scientists have developed an antibody that they believe can be used to treat muscular dystrophy, findings that were published in Science Translational Medicine.

Genetics

Inactive genes surprisingly common in humans

(Medical Xpress) -- Every person carries on average 100 variants that disable genes - yet very few suffer ill effects, an international team of researchers led by Yale University and Wellcome Trust Sanger Institute report ...

Genetics

Toxic protein linked to muscular dystrophy and arhinia

Researchers at the National Institutes of Health and their colleagues have found that a toxic protein made by the body called DUX4 may be the cause of two very different rare genetic disorders. For patients who have facioscapulohumeral ...

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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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