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Examples
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Lysosomal enzyme storage disease such as the mucolipidoses are usually diagnosed and confirmed in white blood cells.
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Trey is a beautiful 3 year old boy who suffers from an illness called MPS II or Hunter's Syndrome, a Lysosomal Storage Disease that inhibits the body's ability to break down and recycle specific mucopolysaccharides.
September 21st, 2007 2007
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"There is a significant amount of interest in the medical community for a more effective treatment option for late-onset Pompe disease, and we believe, based on in vitro and in vivo nonclinical studies, that using our proprietary Glycosylation Independent Lysosomal Targeting, or GILT technology, BMN 701 has the potential to deliver more enzyme to lysosomes compared to traditional mannose-6-phosphate targeted approaches."
BioMarin Initiates Phase 1/2 Trial for BMN 701 for the Treatment of Pompe Disease - Yahoo! Finance 2011
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Gaucher disease was presented at the Annual Meeting of the Lysosomal Disease Network: WORLD Symposium 2010 in
THE MEDICAL NEWS Editors 2010
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Gaucher disease was presented at the Annual Meeting of the Lysosomal Disease Network: WORLD Symposium 2010 in
THE MEDICAL NEWS Editors 2010
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On 02/11/10, Protalix BioTherapeutics, Inc. (NYSE: PLX), announced additional data from the Company's pivotal Phase III clinical trial of taliglucerase alfa in patients with Gaucher disease was presented at the Annual Meeting of the Lysosomal Disease Network: WORLD Symposium
American Chronicle American Chronicle 2010
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On 02/11/10, Protalix BioTherapeutics, Inc. (NYSE: PLX), announced additional data from the Company's pivotal Phase III clinical trial of taliglucerase alfa in patients with Gaucher disease was presented at the Annual Meeting of the Lysosomal Disease Network: WORLD Symposium
American Chronicle American Chronicle 2010
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Amicus Therapeutics (Nasdaq: FOLD) announced today additional positive preliminary data from its ongoing Phase 2 extension study of its investigational drug Amigal ™ (migalastat HCl) for Fabry disease at the Lysosomal Disease Network WORLD Symposium in
THE MEDICAL NEWS Editors 2010
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On 02/11/10, Protalix BioTherapeutics, Inc. (NYSE: PLX), announced additional data from the Company's pivotal Phase III clinical trial of taliglucerase alfa in patients with Gaucher disease was presented at the Annual Meeting of the Lysosomal Disease Network: WORLD Symposium
American Chronicle American Chronicle 2010
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The data were presented in an oral presentation at the Lysosomal Disease Network (LDN) World Symposium in Miami, Florida.
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