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11.发明申请Therapy Regimens, Dosing Regimens And Stable Medicaments For The Treatment Of Pompe Disease 审中-公开治疗方案，剂量治疗和稳定药物治疗庞培病公开(公告)号：US20110268721A1
公开(公告)日：2011-11-03
申请号：US13105720
申请日：2011-05-11
Applicant: Hung V. Do , Ken Valenzano , David J. Lockhart
Inventor： Hung V. Do , Ken Valenzano , David J. Lockhart
CPC classification number: A61K38/47 , A61K2300/00
Abstract: The present application provides a method for treating Pompe disease in a subject in need thereof, that includes a method of administering to the subject a GAA enzyme in combination with an ASSC for the GAA enzyme. The present application also provides methods for increasing the in vitro and in vivo stability of a GAA enzyme formulation.
Abstract translation: 本申请提供了在有需要的受试者中治疗庞培病的方法，其包括将GAA酶与GAA酶的ASSC组合给受试者的方法。 本申请还提供了增加GAA酶制剂的体外和体内稳定性的方法。
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12.发明申请公开(公告)号：US20110143419A1
公开(公告)日：2011-06-16
申请号：US12952036
申请日：2010-11-22
Applicant: Hung V. Do
Inventor： Hung V. Do
CPC classification number: C12P21/02 , C07D201/00 , C07K1/14 , C07K1/22 , C12Y302/01054
Abstract: The present invention provides methods for improving the production of recombinant proteins through the use of pharmacological chaperones for the recombinant proteins. As exemplified by the present invention, the binding of a pharmacological chaperone to a recombinant protein expressed by a cell can stabilize the protein and increase export of the protein out of the cell's endoplasmic reticulum, and increase secretion of the protein by the cell.
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