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Latest Lysosomal storage disease Stories

2014-08-18 12:26:52

DUBLIN, Aug. 18, 2014 /PRNewswire/ -- Research and Markets has announced the addition of the "Global Lysosomal Storage Diseases Market 2014-2018" report to their offering. http://photos.prnewswire.com/prnvar/20130307/600769 The term lysosomal storage diseases refers to a group of rare inherited disorders caused by the deficiency of lysosomal enzymes, activator proteins, proteins required for normal post-translational modification of lysosomal enzymes or proteins required for intracellular...

2014-08-11 20:24:21

LONDON, Aug. 11, 2014 /PRNewswire/ -- Reportbuyer.com has added a new market research report:Lysosomal Storage Disorder-Pipeline Insights, 2014https://www.reportbuyer.com/product/2278642/-Lysosomal-Storage-Disorder-Pipeline-Insights-2014.htmlSUMMARYDelveInsight's," Lysosomal Storage Disorder-Pipeline Insights, 2014", report provides comprehensive insights about pipeline drugs across this indication. A key objective of the report is to establish the understanding for all the pipeline...

2014-07-27 23:01:10

LifeScienceIndustryResearch.com adds “Metachromatic Leukodystrophy (MLD) Global Clinical Trials Review, H2, 2014” to its store. This report provides elemental information and data relating to the clinical trials on MLD. Dallas, TX (PRWEB) July 27, 2014 Metachromatic leukodystrophy (MLD, also called Arylsulfatase A deficiency) is a lysosomal storage disease which is commonly listed in the family of leukodystrophies as well as among the sphingolipidoses as it affects the metabolism of...

2014-07-23 08:30:28

ArmaGen to Receive up to Approximately $225 Million Including Initial Upfront Payment of $15 Million in Cash and Equity, an Additional Equity Investment, R&D Funding, Development and Sales Milestones, in Addition to Future Royalties CALABASAS, Calif., July 23, 2014 /PRNewswire/ -- ArmaGen, a privately held biotechnology company focused on developing revolutionary therapies to treat severe neurological disorders, announced today that it has entered into a worldwide licensing and...

2014-07-22 08:32:03

- Neurotrope enters into world-wide, exclusive license agreement with Mount Sinai; Files related provisional patent for Bryostatin-1 - PLANTATION, Fla., July 22, 2014 /PRNewswire/ -- Neurotrope Bioscience, Inc., a subsidiary of Neurotrope, Inc. (OTCQB: NTRP) has signed a world-wide, exclusive license agreement with the Icahn School of Medicine at Mount Sinai (Mount Sinai) to utilize its proprietary information and data package for the use of Bryostatin-1 in the treatment of...

2014-07-07 16:24:11

NEW YORK, July 7, 2014 /PRNewswire/ -- Lysosomal storage disorders are a group of approximately 50 rare inherited metabolic disorders resulting from defects in lysosomal function. These severe, chronic genetic diseases have unmet medical needs, and include Fabry disease, Gaucher disease, Parkinson's disease, Pompe disease, and Tay-Sachs disease. The market for current drug therapies to address these disorders is estimated to exceed $3 billion. Amicus Therapeutics, Inc. (NASDAQ:...

2014-07-07 08:27:54

Approval delivers first pharmaceutical therapy for children and adults with ultra-rare, life-limiting disorder TORONTO, July 7, 2014 /CNW/ - BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) announced today that Health Canada has approved VIMIZIM(TM)(elosulfase alfa) for long-term enzyme replacement therapy in patients with a confirmed diagnosis of mucopolysaccharidosis IVA (MPS IVA), also known as Morquio A syndrome. The approval makes VIMIZIM the first and only pharmaceutical...

2014-02-04 12:28:02

CINCINNATI, Feb. 4, 2014 /PRNewswire-USNewswire/ -- Researchers used blood platelets and bone marrow cells to deliver potentially curative gene therapy to mouse models of the human genetic disorder Hurler syndrome - an often fatal condition that causes organ damage and other medical complications. (Logo: http://photos.prnewswire.com/prnh/20110406/MM79025LOGO) Scientists from Cincinnati Children's Hospital Medical Center and the National Institute of Neurological Disorders and...

2013-12-03 23:02:00

St. Jude Children’s Research Hospital scientists identify enzyme that could lead to better diagnosis and treatment of Alzheimer’s disease and related disorders. Memphis, Tenn. (PRWEB) December 03, 2013 St. Jude Children’s Research Hospital scientists have identified an enzyme that can halt or possibly even reverse the build-up of toxic protein fragments known as plaques in the brains of mice with Alzheimer’s disease. The research appeared in a recent edition of the scientific...


Word of the Day
mallemaroking
  • Nautical, the visiting and carousing of sailors in the Greenland ships.
This word is apparently from a confusion of two similar Dutch words: 'mallemerok,' a foolish woman, and 'mallemok,' a name for some persons among the crew of a whaling vessel.