A possible first-line treatment for a rare bone loss disease has been identified by a research team led by Tohoku University in Japan. The research findings, published in the journal Molecular Cell, could also provide insight ...
Feb 7, 2018
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A new study led by vision researchers at the Jacobs School of Medicine and Biomedical Sciences at the University at Buffalo and VA Western New York Healthcare System has demonstrated that the addition of widely available ...
Feb 5, 2018
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A team of researchers from the National Institutes of Health and University of Manchester have uncovered new insights into a rare genetic disease, with less than 500 cases of the disease on record, which devastates the lives ...
Dec 12, 2017
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An international team of scientists and doctors has identified a new disease that results in low levels of a common protein found inside our cells.
Dec 8, 2017
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Serious liver and heart problems can affect children with Alagille Syndrome early in life. While there is as yet no cure, researchers at Karolinska Institutet in Sweden have discovered that the liver disease part of the syndrome ...
Nov 21, 2017
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Researchers at Sanford Burnham Prebys Medical Discovery Institute (SBP) have led a preclinical study demonstrating that the drug palovarotene suppresses the formation of bony tumors (osteochondromas) in models of multiple ...
Nov 20, 2017
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A new therapy involving stem cells may offer a better quality of life for patients with Hurler syndrome, a rare genetic disease that can cause crippling physical conditions and lead to early death.
Nov 16, 2017
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A new class of drugs for blood cancers such as leukemia and multiple myeloma is showing promise. But it is hobbled by a problem that also plagues other cancer drugs: targeted cells can develop resistance. Now scientists, ...
Oct 25, 2017
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Pharmaceutical companies developing Orphan Drugs are increasingly partnering with direct-to-consumer (DTC) genetic testing firms to identify individuals with rare diseases, in a trend that is raising concerns related to privacy, ...
Sep 27, 2017
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Physicians are increasingly using genetic testing to unravel mysterious or rare diseases in patients with baffling symptoms. But untangling mystifying symptoms is only half the battle. The next frontier in genomic analysis ...
Sep 12, 2017
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