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Last updated on April 23, 2014 at 1:22 EDT

Latest Hurler Syndrome Stories

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...

2013-07-18 12:28:16

SANTA MONICA, Calif., July 18, 2013 /PRNewswire/ -- ARMAGEN announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to its lead product AGT-182 for the treatment of mucopolysaccharidosis type II (also known as Hunter syndrome or MPS II.) Hunter syndrome is a rare, genetic lysosomal storage disease caused by a deficient or absent enzyme, iduronate-2-sulfatase. It is a life-threatening disease affecting children as young as 2 years of age....

2013-02-04 16:25:54

CINCINNATI, Feb. 4, 2013 /PRNewswire-USNewswire/ -- Researchers have overcome a major challenge to treating brain diseases by engineering an experimental molecular therapy that crosses the blood-brain barrier to reverse neurological lysosomal storage disease in mice. (Logo: http://photos.prnewswire.com/prnh/20110406/MM79025LOGO) Posted online in PNAS Early Edition on Feb. 4, the study was led by scientists at Cincinnati Children's Hospital Medical Center. "This study provides a...

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...

2012-01-03 08:00:00

NOVATO, Calif., Jan. 3, 2012 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that Jean-Jacques Bienaime, Chief Executive Officer of BioMarin, will present a company update at the J.P. Morgan Healthcare Conference in San Francisco on Monday, January 9, 2012 at 11:30 a.m. PT. Interested parties may access a live audio webcast of the conference call via the investor section of the BioMarin website, www.BMRN.com. A replay of the call will be archived on the...

2012-01-03 08:00:00

NOVATO, Calif., Jan. 3, 2012 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today that the Investigational New Drug (IND) application for BMN-111, an analog of C-type Natriuretic Peptide (CNP), for achondroplasia is active. The company expects to initiate the Phase 1 trial in the first quarter of 2012. "BMN-111 has demonstrated benefits in moderately and severely affected animal models and has been shown to promote bone growth even in normal animals," stated Hank...

2011-12-19 08:00:00

NOVATO, Calif., Dec. 19, 2011 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended approval for the company's manufacturing facility expansion in Novato, CA. Final approval by the EMA is expected in the first quarter of 2012, which would allow for products manufactured at the facility to be distributed in the European Union. The two Novato...

2011-11-30 15:00:00

NOVATO, Calif., Nov. 30, 2011 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that it has completed the buy back of certain intellectual property from SA Pathology, a unit of the Central Adelaide Local Health Network located in Adelaide, Australia for an upfront payment of $81 million. The intellectual property includes patents related to the purified form of Naglazyme and the method of using the enzyme in the treatment of MPS VI, which expire between...

2011-11-29 15:00:00

NOVATO, Calif., Nov. 29, 2011 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced the initiation of a Phase 2 study for GALNS (N-acetylgalactosamine 6-sulfatase) in patients under five years of age with mucopolysaccharidosis IVA (MPS IVA). "We expect this study to build our knowledge base in enzyme replacement therapies wherein clinicians believe earlier therapy is more impactful," said Hank Fuchs, M.D., Chief Medical Officer of BioMarin. "Comparatively speaking,...

2011-11-28 08:00:00

NOVATO, Calif., Nov. 28, 2011 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today that it has received approval from the U.S. Food and Drug Administration (FDA) for the manufacturing facility expansion in Novato, CA. The facility will support up to $1.0 billion in revenue for BioMarin's growing enzyme replacement therapy portfolio, which is comprised of complex glycoprotein products produced by mammalian cells. "We are pleased with this first cycle approval of...