If Alzheimer's disease is to be treated in the future it requires an early diagnosis, which is not yet possible. Now researchers at higher education institutions such as Linköping University have identified six proteins ...
Oct 23, 2013
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(Medical Xpress)—Michael Gelb and František Tureček have worked more than a decade to devise and implement newborn screening for some debilitating, often-fatal conditions that show up in the first year to several years ...
Apr 2, 2013
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REGENXBIO Inc. today announced that gene transfer mediated by REGENXBIO's NAV AAV8 vectors resulted in sustained serum α-L-iduronidase (IDUA) expression, as well as correction of systemic features of MPS I, or Hurler syndrome, ...
Sep 30, 2014
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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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Sanfilippo A syndrome or Mucopolysaccharidosis IIIA (MPS-IIIA) is a rare genetic lysosomal storage disease inherited from the parents of the patient. Lysosomes are the body's vehicle for breaking down many of its by-products ...
May 22, 2014
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