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Latest Morquio syndrome Stories

2014-07-07 16:24:20

- MPS IV A patients and families urge swift, publicly funded access to life-altering drug VIMIZIM(TM) - VANCOUVER, July 7, 2014 /CNW/ - The Canadian Society for Mucopolysaccharide & Related Diseases Inc. (Canadian MPS Society) is thrilled by Health Canada's recent approval of VIMIZIM(TM) (elosulfase alfa), the first and only pharmaceutical treatment option available for long-term enzyme replacement therapy in patients with a confirmed diagnosis of Mucopolysaccharidosis IVA (MPS...

2014-07-07 08:27:54

Approval delivers first pharmaceutical therapy for children and adults with ultra-rare, life-limiting disorder TORONTO, July 7, 2014 /CNW/ - BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) announced today that Health Canada has approved VIMIZIM(TM)(elosulfase alfa) for long-term enzyme replacement therapy in patients with a confirmed diagnosis of mucopolysaccharidosis IVA (MPS IVA), also known as Morquio A syndrome. The approval makes VIMIZIM the first and only pharmaceutical...

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...

2011-02-01 08:00:00

NOVATO, Calif., Feb. 1, 2011 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today that it has initiated a pivotal Phase 3 trial for N-acetylgalactosamine 6-sulfatase (GALNS or BMN 110), intended for the treatment of the lysosomal storage disorder Mucopolysaccharidosis Type IVA (MPS IVA), also called Morquio A Syndrome. "In under two years, we have progressed the GALNS program from Clinical Trial Application to initiation of the Phase 3 trial. We have received...

2010-04-29 15:00:00

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...

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...

2009-08-27 07:55:00

ALEXANDRIA, Va., Aug. 27 /PRNewswire/ -- A special "Mad Hatter" Tea Party is being held on October 4th in Alexandria, Va. to raise funds for a little-known disease that affects thousands of children worldwide. A full range of activities for children and adults is planned, including a "Mad Hat" contest and silent auction. The special family-fundraising event was conceived by Stephanie Bozarth, whose daughter, Annabelle, was diagnosed with the disease called Morquio Syndrome when she was...

2009-07-13 08:00:00

NOVATO, Calif., July 13 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today that patient enrollment has been completed for the Phase I/II clinical 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 report initial results in the first half of 2010. "The efficient enrollment of twenty patients...

2009-04-21 15:00:00

NOVATO, Calif., April 21 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today the initiation of a Phase 1/2 clinical 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 report initial results in the first half of 2010. "We plan to leverage our clinical, manufacturing and regulatory expertise to...

2009-03-18 08:00:00

NOVATO, Calif., March 18 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today that its application for clinical trial authorization (CTA) 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, has been accepted by the United Kingdom Medicines and Healthcare Products Regulatory Agency (MHRA). BioMarin expects to initiate a Phase 1/2...


Word of the Day
vermicular
  • Like a worm in form or movement; vermiform; tortuous or sinuous; also, writhing or wriggling.
  • Like the track or trace of a worm; appearing as if worm-eaten; vermiculate.
  • Marked with fine, close-set, wavy or tortuous lines of color; vermiculated.
  • A form of rusticated masonry which is so wrought as to appear thickly indented with worm-tracks.
This word ultimately comes from the Latin 'vermis,' worm.
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