(Medical Xpress)—To remain healthy, the body's cells must properly manage their waste recycling centers. Problems with these compartments, known as lysosomes, lead to a number of debilitating and sometimes lethal conditions.
Feb 20, 2014
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Researchers from the National Institutes of Health have entered into an agreement with biotechnology company Vtesse, Inc., of Gaithersburg, Maryland, to develop treatments for Niemann-Pick disease type C (NPC) and other lysosomal ...
Jan 7, 2015
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University of Rochester Medical Center researchers believe they have identified a potential new means of treating some of the most severe genetic diseases of childhood, according to a study in PLOS Biology. The diseases, ...
Dec 15, 2016
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A study aiming to develop a new therapeutic technique could bring a revolution in our approach to treating rare, fatal Sanfilippo syndrome, a disorder that affects children as young as 2 years old and leads to childhood dementia ...
Feb 24, 2021
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VIB researchers from UGent and Vrije Universiteit Brussel , together with a team of the firm Oxyrane have developed a new technology that can lead to a more efficient and possibly also cheaper therapy for diseases such as ...
Nov 19, 2012
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When people think about Alzheimer's disease, they usually associate it with seniors who have had a long and fulfilling life.
Jun 21, 2023
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A European Journal of Neuroscience study suggests that it is critical to treat lysosomal storage disorders early, before symptoms arise. These genetic disorders, which are caused by the malfunction of enzymes that normally ...
Aug 4, 2014
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(HealthDay)—A set of screening tests designed to detect four rare metabolic disorders in newborns has been approved by the U.S. Food and Drug Administration.
Feb 6, 2017
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Researchers have developed a quality control method to evaluate the pharmacological activity and potential effectiveness of different preparations of the therapeutic agent methyl-β-cyclodextrin (MβCD). Distinct batches ...
Sep 20, 2017
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