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Latest Hurler Syndrome Stories

2014-07-23 08:30:41

Strengthens Leadership Position in Treatments for Hunter syndrome and Commitment to MPS II Patient Community LEXINGTON, Massachusetts, July 23, 2014 /PRNewswire/ -- Shire plc (LSE: SHP, NASDAQ: SHPG), the global specialty biopharmaceutical company, and ArmaGen, a US privately held biotechnology company, today announced a worldwide licensing and collaboration agreement for AGT-182, an investigational enzyme replacement therapy (ERT) for the potential treatment of both the...

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...

2014-07-07 16:24:20

- MPS IV A patients and families urge swift, publicly funded access to life-altering drug VIMIZIM(TM) - VANCOUVER, July 7, 2014 /CNW/ - The Canadian Society for Mucopolysaccharide & Related Diseases Inc. (Canadian MPS Society) is thrilled by Health Canada's recent approval of VIMIZIM(TM) (elosulfase alfa), the first and only pharmaceutical treatment option available for long-term enzyme replacement therapy in patients with a confirmed diagnosis of Mucopolysaccharidosis IVA (MPS...

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...

BioMarin Studies Treatment Of MPS IVA
2012-11-08 07:05:03

Connie K. Ho for redOrbit.com — Your Universe Online Advances in medical technology have made it possible for new treatments to be developed. One such example of the development of treatments is work done by BioMarin Pharmaceutical Inc., a company focused on developing and commercializing biopharmaceuticals for serious diseases and other medical conditions. Having met the primary endpoint of change in a six minute distance walk as compared to a placebo at 24 weeks for participants...


Word of the Day
grass-comber
  • A landsman who is making his first voyage at sea; a novice who enters naval service from rural life.
According to the OED, a grass-comber is also 'a sailor's term for one who has been a farm-labourer.'