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Latest Hurler Syndrome Stories

2010-02-04 15:03:00

NOVATO, Calif., Feb. 4 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today that it has entered into a stock purchase agreement to acquire LEAD Therapeutics, Inc. (LEAD), a small private drug discovery and early stage development company with key compound LT-673, an orally available poly (ADP-ribose) polymerase (PARP) inhibitor for the treatment of patients with rare, genetically defined cancers. "The acquisition of LEAD Therapeutics will augment our...

2010-02-04 15:00:00

NOVATO, Calif., Feb. 4 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced an update on the Phase I/II trial for BMN 110 or N-acetylgalactosamine 6-sulfatase (GALNS), intended for the treatment of the lysosomal storage disorder Mucopolysaccharidosis Type IVA (MPS IVA), or Morquio A Syndrome. Preliminary clinical data from the first 24 weeks of the study (12 weeks at 0.1mg/kg and 12 weeks at 1.0 mg/kg) have been evaluated, and BioMarin plans to announce...

2010-02-02 08:00:00

NOVATO, Calif., Feb. 2 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that Jean-Jacques Bienaime, Chief Executive Officer of BioMarin, will present a company update at the 12th Annual BIO CEO and Investor Conference in New York City on Tuesday, February 9, 2010 at 1:00 p.m. ET. 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...

2010-02-02 08:00:00

NOVATO, Calif., Feb. 2 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that Jean-Jacques Bienaime, Chief Executive Officer of BioMarin, will host a conference call and webcast on Tuesday, February 23, at 5:00 p.m. ET to discuss fourth quarter and full year 2009 financial results. U.S. / Canada Dial-in Number: 800.299.0433 International Dial-in Number: 617.801.9712 Participant Code: 98862458 Replay Dial-in Number: 888.286.8010...

2010-01-05 15:00:00

NOVATO, Calif., Jan. 5 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today that the European Commission has granted marketing approval for 3,4-diaminopyridine (amifampridine phosphate), for the rare autoimmune disease Lambert Eaton Myasthenic Syndrome (LEMS). Amifampridine phosphate, developed by the pharmaceutical unit (AGEPS) of the Paris Public Hospital Authority (AP-HP) and licensed from EUSA Pharma SAS, is the first approved treatment for LEMS,...

2010-01-05 08:00:00

NOVATO, Calif., Jan. 5 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that Jean-Jacques Bienaime, Chief Executive Officer of BioMarin, will present a company update at the 28th Annual J.P. Morgan Healthcare Conference in San Francisco on Monday, January 11, 2010 at 11:00 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...

2009-11-30 08:00:00

NOVATO, Calif., Nov. 30 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today that the Food and Drug Administration (FDA) has granted orphan drug designation for 3,4-diaminopyridine (3,4-DAP), amifampridine phosphate, for the rare autoimmune disease Lambert Eaton Myasthenic Syndrome (LEMS). 3,4-DAP has previously received orphan drug designation in the E.U. Also, in October 2009, the Committee for Medicinal Products for Human Use of the European Medicines...

2009-11-24 15:00:00

NOVATO, Calif., Nov. 24 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that Jean-Jacques Bienaime, Chief Executive Officer of BioMarin, will present a company update at the NASDAQ OMX 23rd Investor Program in London on Tuesday, December 1, 2009 at 9:00 a.m. GMT. 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 site...

2009-11-13 10:58:00

NOVATO, Calif., Nov. 13 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today that Genzyme notified the company that, in rare circumstances, it has detected foreign particles in some products filled at the Allston Landing facility. To ensure that patients are not exposed to foreign particles during product administration, Genzyme has issued a "Dear Doctor" letter to all physicians treating patients with enzyme replacement products that it markets, including...

2009-11-09 15:54:19

Researchers have transplanted genetically modified hematopoietic stem cells into mice so that their developing red blood cells produce a critical lysosomal enzyme "“preventing or reducing organ and central nervous system damage from the often-fatal genetic disorder Hurler's syndrome. The research team from Cincinnati Children's Hospital Medical Center reports its preclinical laboratory results this week in the early edition of Proceedings of the National Academy of Sciences. The study...


Word of the Day
penuche
  • A fudgelike confection of brown sugar, cream or milk, and chopped nuts.
'Penuche' is a variant of 'panocha,' a coarse grade of sugar made in Mexico. 'Panocha' probably comes from the Spanish 'panoja, panocha,' ear of grain.
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