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公开(公告)号：US12109205B2
公开(公告)日：2024-10-08
申请号：US17078840
申请日：2020-10-23
发明人： Jeff Castelli , Elfrida Benjamin
IPC分类号： A61K31/445 , A61P13/12 , A61K9/48
CPC分类号： A61K31/445 , A61P13/12 , A61K9/48
摘要： Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in α-galactosidase A. Certain methods comprise administering migalastat or a salt thereof every other day, such as administering about 150 mg of migalastat hydrochloride every other day.
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公开(公告)号：US20240197706A1
公开(公告)日：2024-06-20
申请号：US18315928
申请日：2023-05-11
发明人： Franklin Johnson
IPC分类号： A61K31/445
CPC分类号： A61K31/445
摘要： Provided are methods of improving the pharmacokinetics of migalastat by limiting caffeine intake during migalastat therapy.
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公开(公告)号：US20230321065A1
公开(公告)日：2023-10-12
申请号：US18199120
申请日：2023-05-18
发明人： Jeff Castelli
IPC分类号： A61K31/45 , A61K31/445
CPC分类号： A61K31/445 , A61K31/45 , A61K31/7008
摘要： Provided are dosing regimens for the treatment of Fabry disease in a patient. Certain methods relate to the treatment of ERT-experienced or ERT-naïve Fabry patients. Certain methods comprise administering to the patient about 123 mg free base equivalent of migalastat for improving left ventricular mass and/or improving podocyte globotriaosylceramide.
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公开(公告)号：US20230203465A1
公开(公告)日：2023-06-29
申请号：US18111321
申请日：2023-02-17
发明人： Russell Gotschall , Hung V. Do
CPC分类号： C12N9/2465 , C12Y302/0102 , A61K48/005 , C12N15/09 , C12N2510/02
摘要： 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 complex oligosaccharides. Compositions containing the rhGAA, and methods of use are described.
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公开(公告)号：US20230158123A1
公开(公告)日：2023-05-25
申请号：US17838741
申请日：2022-06-13
发明人： Russell Gotschall
IPC分类号： A61K38/47 , A61K31/445 , C12N9/40 , C12P21/00
CPC分类号： A61K38/47 , A61K31/445 , C12N9/2465 , C12P21/005 , C12Y302/01022
摘要： Described are compositions comprising α-galactosidase A enzymes with unique carbohydrate profiles, as well as methods for manufacturing and purifying such enzymes. Also described methods of treating, preventing, and/or ameliorating Fabry Disease by administering such enzymes to a subject in need thereof. Also described are compositions comprising migalastat in combination with such α-galactosidase A enzymes.
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公开(公告)号：US11612594B2
公开(公告)日：2023-03-28
申请号：US17077389
申请日：2020-10-22
发明人： Jeff Castelli , Elfrida Benjamin
IPC分类号： A61K31/445 , A61P13/12 , A61K9/48
摘要： Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in α-galactosidase A. Certain methods comprise administering migalastat or a salt thereof every other day, such as administering about 150 mg of migalastat hydrochloride every other day.
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公开(公告)号：US20220387406A1
公开(公告)日：2022-12-08
申请号：US17675536
申请日：2022-02-18
发明人： Jeff Castelli , Elfrida Benjamin
IPC分类号： A61K31/445 , A61P13/12
摘要： Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in α-galactosidase A. Certain methods comprise administering migalastat or a salt thereof every other day, such as administering about 150 mg of migalastat hydrochloride every other day.
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公开(公告)号：US11491211B2
公开(公告)日：2022-11-08
申请号：US16654521
申请日：2019-10-16
发明人： Hing Char , Sergey Tesler , Wendy Sunderland , Enrique Diloné , Russell Gotschall , Hung Do
摘要： 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 of alglucosidase alfa; at least one buffer selected from the group consisting of a citrate, a phosphate and combinations thereof; and at least one excipient selected from the group consisting of mannitol, polysorbate 80, and combinations thereof, wherein the formulation has a pH of from about 5.0 to about 7.0. Also provided are methods of treating Pompe disease using these pharmaceutical formulations.
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公开(公告)号：US11357765B1
公开(公告)日：2022-06-14
申请号：US17670088
申请日：2022-02-11
发明人： Jeff Castelli , Elfrida Benjamin
IPC分类号： A61K31/445 , A61K38/16 , A61P13/12 , A61K9/48
摘要： Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in α-galactosidase A. Certain methods comprise administering migalastat or a salt thereof every other day, such as administering about 150 mg of migalastat hydrochloride every other day.
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公开(公告)号：US11357761B2
公开(公告)日：2022-06-14
申请号：US17400548
申请日：2021-08-12
发明人： Jeff Castelli , Elfrida Benjamin
IPC分类号： A61K31/445 , A61P13/12 , A61K9/48
摘要： Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in α-galactosidase A. Certain methods comprise administering migalastat or a salt thereof every other day, such as administering about 150 mg of migalastat hydrochloride every other day.
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