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Biomarin Pharmaceutical Inc
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Biomarin Pharmaceutical Inc_20100121

Biomarin Pharmaceutical Inc patents


Recent patent applications related to Biomarin Pharmaceutical Inc. Biomarin Pharmaceutical Inc is listed as an Agent/Assignee. Note: Biomarin Pharmaceutical Inc may have other listings under different names/spellings. We're not affiliated with Biomarin Pharmaceutical Inc, we're just tracking patents.

ARCHIVE: New 2018 2017 2016 2015 2014 2013 2012 2011 2010 2009 | Company Directory "B" | Biomarin Pharmaceutical Inc-related inventors


Methods for treating lysosomal storage disorders

Described herein are methods using compounds of Formula (I), (Ia), and (Ib) to treat or prevent a lysosomal storage disorder, methods of making such compounds, and methods of using pharmaceutical compositions and medicaments containing such compounds.... Biomarin Pharmaceutical Inc

Detection of oligosaccharides

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 Inc

Targeted therapeutic proteins

Targeted therapeutics that localize to a specific subcellular compartment such as the lysosome are provided. The targeted therapeutics include a therapeutic agent and a targeting moiety that binds a receptor on an exterior surface of the cell, permitting proper subcellular localization of the targeted therapeutic upon internalization of the receptor.... Biomarin Pharmaceutical Inc

Glucosylceramide synthase inhibitors for the treatment of diseases

Described herein are compounds of Formula I, methods of making such compounds, pharmaceutical compositions and medicaments containing such compounds, and methods of using such compounds to treat or prevent diseases or conditions associated with the enzyme glucosylceramide synthase (GCS).... Biomarin Pharmaceutical Inc

Targeted therapeutic lysosomal enzyme fusion proteins and uses thereof

The present invention relates in general to therapeutic fusion proteins useful to treat lysosomal storage diseases and methods for treating such diseases. Exemplary therapeutic fusion proteins comprise a lysosomal enzyme, a lysosomal targeting moiety, e.g., an IGF-II peptide, and a spacer peptide. Also provided are compositions and methods for treating... Biomarin Pharmaceutical Inc

Quantification of non-reducing end glycan residual compounds for determining the presence, identity, or severity of a disease or condition

Provided herein are methods of diagnosing or monitoring the treatment of abnormal glycan accumulation or a disorder associated with abnormal glycan accumulation.... Biomarin Pharmaceutical Inc

Delivery of therapeutic compounds to the brain and other tissues

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 α-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... Biomarin Pharmaceutical Inc

Adeno-associated virus factor viii vectors

The invention provides improved adeno-associated virus (AAV) Factor VIII (FVIII) vectors, including AAV FVIII vectors that produce a functional Factor VIII polypeptide and AAV FVIII vectors with high expression activity.... Biomarin Pharmaceutical Inc

Adeno-associated virus factor viii vectors, associated viral particles and therapeutic formulations comprising the same

The invention provides adeno-associated virus (AAV) Factor VIII (FVIII)-encoding/expressing vectors and virus, including AAV FVIII vectors with high expression activity and AAV FVIII vectors that express full-length or truncated functional FVIII protein. The invention also relates to methods of making the herein described AAV FVIII vectors, recombinant AAV FVIII virus... Biomarin Pharmaceutical Inc

Histone deacetylase inhibitors

This invention relates to generally inhibiting histone deacetylase (“HDAC”) enzymes (e.g., HDAC1, HDAC2, and HDAC3).... Biomarin Pharmaceutical Inc

Variants of c-type natriuretic peptide

The present disclosure provides variants of C-type natriuretic peptide (CNP), pharmaceutical compositions comprising CNP variants, and methods of making CNP variants. The CNP variants are useful as therapeutic agents for the treatment of diseases responsive to CNP, including but not limited to bone-related disorders, such as skeletal dysplasias (e.g., achondroplasia),... Biomarin Pharmaceutical Inc

Use of tetrahydrobiopterine derivatives in the treatment and nutrition of patients with amino acid metabolic disorders

The invention relates to the use of tetrahydrobiopterine and the derivatives thereof in the production of a medicament to improve protein tolerance for the treatment of diseases arising from an amino acid metabolic disorder, e.g. hyperphenylalaninemia. The invention also relates to a composition which contains tetrahydrobiopterine or derivatives thereof in... Biomarin Pharmaceutical Inc

Histone deacetylase inhibitors

This invention relates to generally inhibiting histone deacetylase (“HDAC”) enzymes (e.g., HDAC1, HDAC2, and HDAC3).... Biomarin Pharmaceutical Inc

Use of c-type natriuretic peptide variants to treat skeletal dysplasia

The present disclosure provides for use of variants of C-type natriuretic peptide (CNP), and novel pharmaceutical compositions and formulations comprising CNP variant peptides for the treatment of skeletal dysplasias, one or more symptoms of skeletal dysplasias, such as long bone growth or growth velocity, and other disorders having a skeletal... Biomarin Pharmaceutical Inc

Lysosomal targeting peptides and uses thereof

The present invention provides further improved compositions and methods for efficient lysosomal targeting based on the GILT technology. Among other things, the present invention provides methods and compositions for targeting lysosomal enzymes to lysosomes using furin-resistant lysosomal targeting peptides. The present invention also provides methods and compositions for targeting lysosomal... Biomarin Pharmaceutical Inc

Methods of administering tetrahydrobiopterin, associated compositions, and methods of measuring

The present invention is directed to treatment methods of administering tetrahydrobiopterin, including in oral dosage forms, in intravenous formulations, and with food. Also disclosed herein are biopterin assays for measuring the amount of biopterin and metabolites of biopterin in a sample.... Biomarin Pharmaceutical Inc








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