发明名称 DEUTERATED TREHALOSE FORMULATIONS AND USES THEREOF
摘要 The presently invention relates to methods, formulations and kits comprising deuterated trehalose for treating myopathies, neurodegenerative disorders, or tauopathies associated abnormal protein aggregation.
申请公布号 US2016303150(A1) 申请公布日期 2016.10.20
申请号 US201615099168 申请日期 2016.04.14
申请人 BioBlast Pharma Ltd. 发明人 MEGIDDO Dalia
分类号 A61K31/7016;A61K9/08;A61K9/00 主分类号 A61K31/7016
代理机构 代理人
主权项 1. A method for treating or alleviating a disease associated with abnormal protein aggregation and/or inclusion bodies formation in myocytes, neurons and other cells or extracellular compartments or at least one symptom associated therewith, in a human subject in need thereof comprising administering to said subject a therapeutically effective amount of deuterated trehalose or a mixture of several deuterated trehaloses, optionally together with non-deuterated trehalose or a pharmaceutical formulation comprising a therapeutically effective amount of deuterated trehalose or a mixture of several deuterated trehaloses, and optionally comprising non-deuterated trehalose, wherein in said deuterated trehalose at least one hydrogen atom attached to a carbon atom is replaced by a deuterium atom.
地址 Tel Aviv IL