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Latest Hurler Syndrome Stories

2010-09-09 08:00:00

NOVATO, Calif., Sept. 9 /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 Stifel Nicolaus Healthcare Conference in Boston on Friday, September 17, 2010 at 9:10 a.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...

2010-09-08 08:00:00

NOVATO, Calif., Sept. 8 /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 Robert W. Baird Healthcare Conference in New York City on Wednesday, September 15, 2010 at 10:30 a.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...

2010-09-07 08:00:00

NOVATO, Calif., Sept. 7 /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 Morgan Stanley Global Healthcare Conference in New York City on Tuesday, September 14, 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...

2010-09-01 08:00:00

NOVATO, Calif., Sept. 1 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today that results from investigator-sponsored studies on Kuvan (sapropterin dihydrochloride) are being presented at the 2010 Annual Symposium of the Society for The Study of Inborn Errors of Metabolism (SSIEM), August 31st-September 3rd in Istanbul, Turkey. Investigators reported that PKU patients taking Kuvan showed improvements in brain functioning, mood and behaviors. "While these...

2010-08-30 08:00:00

NOVATO, Calif., Aug. 30 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today that it has received orphan drug designation from the U.S. Food and Drug Administration (FDA) for BMN-701, a novel fusion of insulin-like growth factor 2 and alpha glucosidase (IGF2-GAA) in development for the treatment of Pompe disease. An investigational new drug application (IND) for BMN-701 has been submitted, investigational material has been manufactured and a Phase I/II...

2010-08-17 15:27:00

NOVATO, Calif., Aug. 17 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today that it has acquired ZyStor Therapeutics, Inc. (ZyStor), a privately-held biotechnology company developing enzyme replacement therapies (ERT) for the treatment of lysosomal storage disorders. ZyStor's lead product candidate is ZC-701, a novel fusion of insulin-like growth factor 2 and alpha glucosidase (IGF2-GAA) in development for Pompe disease. Under the terms of the...

2010-08-17 08:00:00

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...

2010-08-02 15:01:00

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,...

2010-08-02 15:00:00

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...

2010-07-06 08:00:00

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...


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.'