公开(公告)号：US20230084580A1

公开(公告)日：2023-03-16

申请号：US18046363

申请日：2022-10-13

Applicant: Genzyme Corporation

Inventor： Marco A. PASSINI ,  Lamya Shihabuddin ,  Seng H. Cheng

IPC: A61K38/17 ,  A61K31/7088 ,  A61K48/00 ,  C12N15/86 ,  C12N7/00 ,  C07K14/47

Abstract: Compositions and methods for treating disorders affecting motor function, such as motor function affected by disease or injury to the brain and/or spinal cord, are disclosed.

公开(公告)号：US20150284472A1

公开(公告)日：2015-10-08

申请号：US14440818

申请日：2013-11-04

Applicant: GENZYME CORPORATION

Inventor： Sergio Pablo Sardi ,  Lamya Shihabuddin ,  Seng Cheng

IPC: C07K16/40 ,  A61K38/47 ,  A61K35/76 ,  A61K9/00

CPC classification number: C07K16/40 ,  A61K9/0019 ,  A61K35/76 ,  A61K38/47 ,  C07K16/18 ,  C12N15/86 ,  C12N2750/14141 ,  C12Y302/01045

Abstract: This disclosure relates to methods for improving neural function in a mammal with a proteinopathy comprising administering a therapeutically effective amount of an agent that increases glucocerebrosidase activity in the mammal. Also disclosed are methods for reducing toxic lipids, reducing α-synuclein, and/or inhibiting the accumulation of protein aggregates in a mammal with a proteinopathy comprising administering a therapeutically effective amount of an agent that increases glucocerebrosidase activity.

Abstract translation: 本公开涉及用于改善具有蛋白质病变的哺乳动物中的神经功能的方法，包括施用治疗有效量的增加哺乳动物中葡糖脑苷脂酶活性的药剂。 还公开了减少有毒性脂质，还原α-突触核蛋白和/或抑制蛋白质聚集体在具有蛋白质病的哺乳动物中的积累的方法，包括施用治疗有效量的增加葡糖脑苷脂酶活性的试剂。