In a proof-of-concept study, researchers demonstrated the effectiveness of a potential new therapy for Timothy syndrome, an often life-threatening and rare genetic disorder that affects a wide range of bodily systems, leading ...
Apr 24, 2024
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Hypertriglyceridemia, or high levels of lipids (fats) in the blood, increases the risk of heart attack, stroke and acute pancreatitis. Currently available medications, including statins, ezetimibe, fibrates and prescription ...
Apr 8, 2024
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Non-alcoholic fatty liver disease (NAFLD) is a major health problem affecting approximately 30% of people in Western countries and which has been in sharp increase over the last three decades.
Dec 5, 2023
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Scientists have developed a new approach to produce life-saving oligonucleotide therapeutics on a large scale, in high purity, and with minimal environmental impacts. The finding, by The University of Manchester, will facilitate ...
Jun 28, 2023
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The development of hepatocellular carcinoma, liver cancer, is known to involve the abnormal activation of a signaling pathway called Wnt; however, the underlying mechanisms have not been fully understood, until now.
Jun 28, 2023
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There is a growing body of evidence that iron in the brain may play a role in Alzheimer's disease. Lending weight to that idea, a new imaging probe has for the first time shown that in the same regions of the brain where ...
Apr 19, 2023
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To get therapies into the brain after a stroke, researchers are increasingly making use of the blood–brain barrier, which allows only certain molecules to pass from the blood into the brain. In a study published earlier ...
Apr 17, 2023
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A new report, coauthored by several major pharmaceutical companies, reviews the current state of sterile oligonucleotide drug product processing. The article, which provides recommendations to aid in the evaluation and development ...
Feb 22, 2023
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Muscular dystrophy is a debilitating disease that causes progressive weakening and loss of muscles. Fukuyama congenital muscular dystrophy (FCMD), the second most common form of childhood muscular dystrophy in Japan, is a ...
Dec 12, 2022
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Antisense oligonucleotides, or ASOs, are molecules that can be used to control protein levels in cells. Cold Spring Harbor Laboratory Professor Adrian Krainer leveraged ASO technology to develop the first FDA-approved treatment ...
Jul 14, 2022
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