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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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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Gaucher (pronounced "go-shay") disease affects one in 450 Jewish people of Ashkenazi (eastern European) descent (one in 10 is a carrier), yet only 1 in 40,000 people in the general population. Of course mutations can happen ...
Dec 9, 2016
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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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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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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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Several young children suffering from a severe degenerative genetic disease received injections of therapeutic genes packaged within a noninfectious viral delivery vector. Safety, tolerability, and efficacy results from this ...
Mar 11, 2014
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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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After years of building hope for a treatment, Rochester researchers and clinicians will begin the first controlled clinical trial for Juvenile Batten disease this summer, thanks to $1 million in grants from the Food and Drug ...
May 31, 2011
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Researchers at Rush University Medical Center have identified a key immunological defense reaction to the metals in joint replacement devices, leading to loosening of the components and early failure.
Mar 4, 2009
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