The U.S. Food and Drug Administration has approved Duvyzat (givinostat) as an oral medication for the treatment of Duchenne muscular dystrophy (DMD) in patients six years of age and older.
Mar 26, 2024
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A specific gene may play a key role in new treatments that prevent muscle in the body from breaking down in serious muscle diseases, or muscular dystrophies. This is shown in a new study carried out at Umeå University, Sweden, ...
Mar 6, 2024
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The drug vamorolone (Agamree) has been hailed as a promising new drug to treat Duchenne muscular dystrophy (DMD). It has recently been approved for clinical use in the United States by the Food and Drug Administration (FDA) ...
Jan 25, 2024
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Muscular dystrophies are a group of diseases in which proteins in the muscles do not function properly, either through inherited or spontaneous mutations. This, in turn, leads to muscle tissue breaking down over time and ...
Jan 22, 2024
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About 100 cancer predisposition genes (CPGs) are known. The probability of developing cancer is greater in individuals who inherit certain altered variants of one of these genes.
Jan 16, 2024
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Northwestern Medicine scientists have developed a deep learning algorithm capable of identifying the location where a genetic process called polyadenylation occurs on the genome, according to findings published in Nature ...
Dec 15, 2023
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New research from Intermountain Health in Salt Lake City finds that genetic screening is effective in identifying patients who have a heart condition known as cardiomyopathy to improve their quality of life and reduce deaths.
Nov 13, 2023
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Skeletal muscle plays an extremely important role in supporting movement and energy metabolism. However, its function can be impaired by aging and muscle-related diseases, resulting in decreased endurance or strength of skeletal ...
Nov 1, 2023
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Duchenne muscular dystrophy (DMD) is the most common muscular dystrophy diagnosed in childhood, with approximately 20,000 new cases reported each year. It is a progressive muscle disorder that results in the loss of muscle ...
Sep 29, 2023
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Researchers have gained insight into how a toxic protein, known to trigger muscular dystrophy, could be deactivated—a pre-clinical discovery that could spearhead a treatment for the debilitating disease.
Sep 25, 2023
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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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