Using combinations of well-known approved drugs has for the first time been shown to be potentially safe in treating a rare disease, according to the results of a clinical trial published in the open access Orphanet Journal ...
Dec 17, 2014
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(Medical Xpress)—Scientists at the Medical Research Council (MRC) Laboratory of Molecular Biology have found that stem cells in the body's 'blood cell factory'—the bone marrow—are extremely sensitive to the main breakdown ...
Aug 27, 2012
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Each year, about 20,000 children are diagnosed with Duchenne muscular dystrophy, a rare genetic condition that causes progressive muscle weakness and other systemic damage.
Feb 18, 2022
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A first-in-man clinical trial, testing a newly developed drug for a rare genetic condition called mucopolysaccharidosis type II (MPS II) or Hunter syndrome, is underway at Emory University.
Jun 3, 2015
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A rare genetic condition which causes inflammatory bowel disease can be successfully treated by bone marrow transplant, according to University of Manchester and Manchester University NHS Foundation Trust researchers.
Oct 13, 2020
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Scientists reported today the first human recipients of laboratory-grown vaginal organs. A research team led by Anthony Atala, M.D., director of Wake Forest Baptist Medical Center's Institute for Regenerative Medicine, describes ...
Apr 10, 2014
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Massachusetts General Hospital investigators appear to have found the mechanism behind a previously reported link between the rare genetic condition Gaucher disease and the common neurodegenerative disorder Parkinson's disease. ...
Jun 23, 2011
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A Bangladeshi father dubbed the "tree man" because of the bark-like warts that once covered his body will soon be able to leave hospital after groundbreaking treatment for one of the world's rarest diseases.
Jan 6, 2017
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Deleting a single gene in the cerebellum of mice can cause key autistic-like symptoms, researchers have found. They also discovered that rapamycin, a commonly used immunosuppressant drug, prevented these symptoms.
Jul 2, 2012
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Individuals with the neurodevelopmental disorder Williams syndrome have a gregarious "cocktail party" personality, while those with the opposite genetic alteration, in contrast, tend to have autistic traits and are prone ...
Feb 28, 2024
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