Mucopolysaccharidosis News and Research

RSS

Mucopolysaccharidoses are a group of metabolic disorders caused by the absence or malfunctioning of lysosomal enzymes needed to break down molecules called glycosaminoglycans - long chains of sugar carbohydrates in each of our cells that help build bone, cartilage, tendons, corneas, skin and connective tissue. Glycosaminoglycans (formerly called mucopolysaccharides) are also found in the fluid that lubricates our joints.

BioMarin Pharmaceutical reports GAAP net income of $4.7M for fourth-quarter 2009

BioMarin Pharmaceutical Inc. today announced financial results for the fourth quarter and year ended December 31, 2009. GAAP net income was $4.7 million ($0.05 per diluted share) for the fourth quarter of 2009, compared to GAAP net income of $24.5 million ($0.21 per diluted share) for the fourth quarter of 2008, which included a $30.0 million payment from Merck Serono related to the approval of Kuvan in the EU.

24 Feb 2010

BioMarin announces update on BMN 110 Phase I/II trial for Morquio A Syndrome

BioMarin Pharmaceutical Inc. 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 top-line results for the full 36-week study after completion of dosing at 2.0 mg/kg in the second quarter of 2010.

5 Feb 2010

Naglazyme approved in Japan

BioMarin Pharmaceutical Inc. has announced that AnGes MG, Inc. (AnGes), BioMarin's marketing and distribution partner in Japan, has received approval for its Marketing Application for Naglazyme(R) (galsulfase) from the Japanese Ministry of Health, Labour and Welfare (MHLW) for the treatment of patients with Mucopolysaccharidosis VI ( MPS VI).

1 Apr 2008