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Amicus Therapeutics Inc patents


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

ARCHIVE: New 2018 2017 2016 2015 2014 2013 2012 2011 2010 2009 | Company Directory "A" | Amicus Therapeutics Inc-related inventors


Alpha-galactosidase a and 1-deoxygalactonojirimycin co-formulation

The present application provides for compositions comprising α-galactosidase A in combination with an active site-specific chaperone for the α-galactosidase A, and methods for treating Fabry disease in a subject in need thereof, that includes a method of administering to the subject such compositions. The present application also provides methods for... Amicus Therapeutics Inc

Novel compounds for preventing and/or treating lysosomal storage disorders and/or degenerative disorders of the central nervous system

Described are novel salts of the compound (3R,4R,5S)-5-(difluoromethyl) piperidine-3,4-diol, as well as methods of using the same for preventing and/or treating lysosomal storage disorders and/or degenerative disorders of the central nervous system. In particular, the present invention provides methods for preventing and/or treating Gaucher's disease and/or Parkinson's disease.... Amicus Therapeutics Inc

Formulations comprising recombinant acid alpha-glucosidase

Provided are pharmaceutical formulations comprising a recombinant acid α-glucosidase, wherein the recombinant acid α-glucosidase is expressed in Chinese hamster ovary (CHO) cells and comprises an increased content of N-glycan units bearing one or two mannose-6-phosphate residues when compared to a content of N-glycan units bearing one or two mannose-6-phosphate residues... Amicus Therapeutics Inc

Method for selection of high m6p recombinant proteins

Methods for the production, capturing and purification of recombinant human lysosomal proteins are described. Such recombinant human lysosomal proteins can have high content of mannose-6-phosphate residues. Also described are pharmaceutical compositions comprising such recombinant human lysosomal proteins, as well as methods of treatment and uses of such recombinant human lysosomal... Amicus Therapeutics Inc

Methods for coupling targeting peptides onto recombinant lysosomal enzymes for improved treatments of lysosomal storage diseases

Described herein are methods of making targeting peptides conjugated to a recombinant lysosomal enzyme by modifying the amino (N)-terminus and one or more lysine residues on a recombinant human lysosomal enzyme using a first crosslinking agent to give rise to a first crosslinking agent modified recombinant human lysosomal enzyme, modifying... Amicus Therapeutics Inc

Highly potent acid alpha-glucosidase with enhanced carbohydrates

Recombinant human alpha glucosidase (rhGAA) composition derived from CHO cells that contains a more optimized glycan composition consisting of a higher amount of rhGAA containing N-glycans carrying mannose-6-phosphate (M6P) or bis-M6P than conventional rhGAAs, along with low amount of non-phosphorylated high mannose glycans, and low amount of terminal galactose on... Amicus Therapeutics Inc

Methods of treating fabry disease in patients having the g9331a mutation in the gla gene

Provided are methods of treating a patient diagnosed with Fabry disease and methods of enhancing α-galactosidase A in a patient diagnosed with or suspected of having Fabry disease. Certain methods comprise administering to a patient a therapeutically effective dose of a pharmacological chaperone for α-galactosidase A, wherein the patient has... Amicus Therapeutics Inc

Dosing regimens for treating and/or preventing cerebral amyloidoses

Described herein are dosing regimens and kits for the treatment and/or prevention of cerebral amyloidoses such as Alzheimer's disease (AD) and/or cerebral amyloid angiopathy (CAA).... Amicus Therapeutics Inc

Alpha-galactosidase a and 1-deoxygalactonojirimycin co-formulation for the treatment of fabry disease

The present application provides compositions comprising α-galactosidase A in combination with an active site-specific chaperone for the α-galactosidase A, and methods for treating Fabry disease in a subject in need thereof, that includes a method of administering to the subject such compositions. The present application also provides methods for increasing... Amicus Therapeutics Inc

Augmented acid alpha-glucosidase for the treatment of pompe disease

A method for treating Pompe disease including administration of recombinant human acid α-glucosidase having optimal glycosylation with mannose-6-phosphate residues in combination with an amount of miglustat effective to maximize tissue uptake of recombinant human acid α-glucosidase while minimizing inhibition of the enzymatic activity of the recombinant human acid α-glucosidase is... Amicus Therapeutics Inc

Sugar derivatives comprising sulfur-containing moieties and methods of making same and methods of using the same for the treatment of mps iiic

Described herein are modified sugar, iminosugar and azasugar compounds and methods of making same. One or more of these modified compounds contain sulfates, sulfites, sulfamates and/or sulfonamides. Also described are pharmaceutical compositions/formulations comprising these compounds, as well as methods using these modified sugar compounds for the treatment of MPS IIIC... Amicus Therapeutics Inc

Compounds and methods for the treatment of alzheimer's disease and/or cerebral amyloid angiopathy

Described herein are novel compounds and methods for the treatment and/or prevention of cerebral amyloidoses such as Alzheimer's disease (AD) and/or cerebral amyloid angiopathy (CAA).... Amicus Therapeutics Inc

Variant, recombinant beta-glucocerebrosidase proteins with increased stability and increased retained catalytic activity

Described herein are variant, recombinant β-glucocerebrosidase proteins characterized as having increased stability relative to recombinant wild-type β-glucocerebrosidase. Also provided herein are variant, recombinant β-glucocerebrosidase proteins characterized as retaining more catalytic activity relative to recombinant wild-type β-glucocerebrosidase. Further described herein are variant, recombinant β-glucocerebrosidase proteins that can have amino acid variations... Amicus Therapeutics Inc

Regimens for treating and preventing lysosomal disorders and degenerative disorders of the central nervous system

Described herein are dosing regimens and kits for the treatment and/or prevention of lysosomal storage disorders such as Gaucher disease. Also described are dosing regimens and kits for the treatment and/or prevention of degenerative disorders of the central nervous system, such as the synucleinopathies Parkinson's disease or Lewy Body Dementia.... Amicus Therapeutics Inc

High concentration alpha-glucosidase compositions for the treatment of pompe disease

The present application provides for compositions comprising high concentrations of acid α-glucosidase in combination with an active site-specific chaperone for the acid α-glucosidase, and methods for treating Pompe disease in a subject in need thereof, that includes a method of administering to the subject such compositions. The present application also... Amicus Therapeutics Inc

Methods for treatment of fabry disease

Provided are in vitro and in vivo methods for determining whether a patient with Fabry disease will respond to treatment with a specific pharmacological chaperone.... Amicus Therapeutics Inc

New preparing isofagomine and its derivatives

A method for preparing isofagomine, its derivatives, intermediates and salts thereof using novel processes to make isofagomine from D-(−)-arabinose and L-(−)-xylose.... Amicus Therapeutics Inc

Method for the treatment of neurological disorders by enhancing the activity of beta-glucocerebrosidase

Provided is a method of increasing the stability of wild-type β-glucocerebrosidase. Also provided are methods of treating and/or preventing an individual having a neurological disease in which increased expression or activity of β-glucocerebrosidase in the central nervous system would be beneficial. This method comprises administering an effective amount of a... Amicus Therapeutics Inc

Novel compounds for preventing and/or treating lysosomal storage disorders and/or degenerative disorders of the central nervous system

Described are novel salts of the compound (3R,4R,5S)-5-(difluoromethyl) piperidine-3,4-diol, as well as methods of using the same for preventing and/or treating lysosomal storage disorders and/or degenerative disorders of the central nervous system. In particular, the present invention provides methods for preventing and/or treating Gaucher's disease and/or Parkinson's disease.... Amicus Therapeutics Inc








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