BioMarin Pharmaceutical Inc. is a biotechnology company based in San Rafael, California. It has offices and facilities in the United States, South America, Asia, and Europe. BioMarin develops and commercializes innovative biopharmaceuticals for serious, life-threatening rare diseases and medical conditions. BioMarin's core business and research is in enzyme replacement therapies . BioMarin is the first company to provide therapeutics for mucopolysaccharidosis type I , by manufacturing laronidase . BioMarin is also the first company to provide therapeutics for phenylketonuria . Wikipedia.
BioMarin Pharmaceutical | Date: 2015-02-05
The present invention relates to the intrathecal (IT) administration of recombinant enzyme to treat lysosomal storage disorders. In an exemplary embodiment, intrathecal administration of human -L-iduronidase (rhIDU) injections in MPS I affected animals resulted in significant enzyme uptake, significant rh-iduronidase activity in brain and meninges and a decrease of glycosaminoglycan (GAG) storage in cells of MPS I subjects to that of normal subjects. Intrathecal administration proved more effective than intravenous treatment at alleviating MPS I symptoms, indicating it is a useful method of treating lysosomal storage disorders.
BioMarin Pharmaceutical | Date: 2015-05-07
Provided herein are processes for detecting oligosaccharides in a biological sample. In specific instances, the biological sample is provided from an individual suffering from a disorder associated with abnormal glycosaminoglycan accumulation.
BioMarin Pharmaceutical | Date: 2015-01-16
This invention relates to generally inhibiting histone deacetylase (HDAC) enzymes (e.g., HDAC1, HDAC2, and HDAC3).
BioMarin Pharmaceutical | Date: 2016-01-12
BioMarin Pharmaceutical | Date: 2015-03-27
A compound having the structure set forth in Formula (I) and Formula (II): wherein the substituents Y, Z, A, B, R