Latest Hurler Syndrome Stories

NOVATO, Calif., Aug. 17 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today that the first subject has initiated treatment in a Phase 3b study (PKU-016) to evaluate the effects of Kuvan (sapropterin dihydrochloride) on neuropsychiatric symptoms in subjects with phenylketonuria (PKU). BioMarin expects to report results in the first half of 2012. "We hope that this trial will provide quantitative data regarding the neuropsychiatric effects of Kuvan for PKU...

NOVATO, Calif., Aug. 2 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today an update in the Phase 2 clinical study of PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase) for the treatment of phenylketonuria (PKU). Data reported in this announcement reflect preliminary results as of July 23, 2010. Complete top-line results are expected in the fourth quarter of 2010. Key highlights of the study: 23 adult patients have been enrolled in the study, and...

NOVATO, Calif., Aug. 2 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today that it has completed the Phase 1 clinical study of BMN 195, a small molecule utrophin up-regulator, for the treatment of Duchenne muscular dystrophy (DMD). The Phase 1 clinical trial was a single-center, double-blind, placebo-controlled, single-dose escalation study followed by a multiple-dose escalation study in healthy volunteers. The administration of up to 400 mg/kg did not...

NOVATO, Calif., July 6 /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 Monday, August 2, at 5:00 p.m. ET to discuss second quarter 2010 financial results. U.S. / Canada Dial-in Number: 800.573.4840 International Dial-in Number: 617.224.4326 Participant Code: 78258415 Replay Dial-in Number: 888.286.8010 Replay International...

NOVATO, Calif., June 17 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that Jeffrey Cooper, Chief Financial Officer of BioMarin, will present a company update at the Wells Fargo Healthcare Conference in Boston on Thursday, June 24, 2010 at 2:30 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 for one...

NOVATO, Calif., June 10 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that Jeffrey Cooper, Chief Financial Officer of BioMarin, will present a company update at the Goldman Sachs 31st Annual Healthcare Conference in Los Angeles on Thursday, June 17, 2010 at 8:00 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 archived on...

NOVATO, Calif., May 12 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that Jeffrey Cooper, Chief Financial Officer of BioMarin, will present a company update at the Baird Growth Stock Conference in Chicago on Wednesday, May 19, 2010 at 11:50 a.m. CT. 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 for one week...

NOVATO, Calif., May 4 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that Dan Maher, Senior Vice President of Product Development of BioMarin, will present a company update at the JMP Securities Research Conference in San Francisco on Tuesday, May 11, 2010 at 3:00 p.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...

NOVATO, Calif., April 29 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced positive results for 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. The company expects to initiate a pivotal Phase III trial in the fourth quarter of 2010. Highlights from the Phase I/II study: Endurance improvements with...

NOVATO, Calif., April 29 /PRNewswire-FirstCall/ -- Financial Highlights ($ in millions, except per share data, unaudited) Item Q1 2010 Q1 2009 Comparison Total BioMarin Revenue $85.0 14.8% increase Total Net Product Revenue $84.1 16.9% increase Naglazyme Net Product Revenue $48.6 23.4% increase...