Arginine Depletion Therapy For Treatment of GAMT Deficiency

    公开(公告)号:US20210379167A1

    公开(公告)日:2021-12-09

    申请号:US17286041

    申请日:2019-10-18

    申请人: Aerase, Inc.

    IPC分类号: A61K38/50

    摘要: Methods and compositions therefor of treating GAMT deficiency or guanidino acetate (GAA) toxicity in a subject comprising administration of an arginine depleting enzyme. An therapeutic formulation can include an arginase, an arginine deiminase or a combination thereof and optionally other compounds, and can be adapted for intravenous or subcutaneous administration to a subject.