发明名称 COMPOSITIONS AND METHODS FOR TREATING SPINOCEREBELLAR ATAXIA
摘要 The present invention provides alleviating a sign or symptom of spinocerebellar ataxia by administering an injection of an aqueous formulation comprising trehalose.
申请公布号 US2016101122(A1) 申请公布日期 2016.04.14
申请号 US201514973465 申请日期 2015.12.17
申请人 BIOBLAST PHARMA LTD. 发明人 MEGIDDO Dalia
分类号 A61K31/7016 主分类号 A61K31/7016
代理机构 代理人
主权项 1. A method of alleviating a sign or symptom of spinocerebellar ataxia (SCA) by administering to a subject in need thereof an injection of an aqueous formulation comprising a single active ingredient consisting of trehalose, wherein the formulation (i) has a pH about 4.5 to 7.0, (ii) contains less than 0.75 endotoxin units per mL, and (iii) is administered at a per administration dose of between 5 to 50 grams trehalose.
地址 Tel Aviv IL