Latest Hurler Syndrome Stories
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,...
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...
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...
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...
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...
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...
NOVATO, Calif., Nov. 5 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today that the United States Patent and Trademark Office has issued a patent covering the approved administration of Kuvan® (sapropterin dihydrochloride) with food for the treatment of phenylketonuria (PKU). The patent expires in 2024. "We believe that the food effect patent, based on the discovery that food increases the bioavailability of Kuvan, along with our patents for once daily...
NOVATO, Calif., Nov. 4 /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 Credit Suisse Healthcare Conference in Phoenix on Wednesday, November 11, 2009 at 12:00 p.m. MST. 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...
NOVATO, Calif., Oct. 28 /PRNewswire-FirstCall/ -- Financial Highlights ($ in millions, except per share data) Item Q3 2009 Q3 2008 Comparison Total BioMarin Revenue $80.8 11% increase Total Net Product Revenue $78.4 16% increase Naglazyme Net Product Revenue $42.1 26% increase Aldurazyme BioMarin Net Product Revenue $14.6...
NOVATO, Calif., Oct. 27 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that Hank Fuchs, M.D., Chief Medical Officer of BioMarin, will present a company update at the Oppenheimer Healthcare Conference in New York City on Tuesday, November 3, 2009 at 1:35 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 on the site...
