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    BIOMARKERS OF POLYCYSTIC KIDNEY DISEASE AND USES THEREOF
    41.
    发明公开
    BIOMARKERS OF POLYCYSTIC KIDNEY DISEASE AND USES THEREOF 审中-公开
    多发性肾病的生物标志物及其应用

    公开(公告)号：EP3177932A2

    公开(公告)日：2017-06-14

    申请号：EP15757363.5

    申请日：2015-08-03

    申请人： Genzyme Corporation

    发明人： BESKROVNAYA, Oxana ,  BUKANOV, Nikolai ,  MORENO, Sarah

    IPC分类号： G01N33/68 ,  C07K16/00

    CPC分类号： G01N33/6893 ,  G01N2800/347 ,  G01N2800/52

    摘要： Provided herein are methods for determining the efficacy of treatment for polycystic kidney disease (PKD) in a patient, diagnosing PKD in a patient, staging PKD in a patient, and monitoring PKD in a patient. These methods include determining a single or multiple levels of one or more markers selected from the group of Proliferating Cell Nuclear Antigen (PCNA), cyclin D1, cyclin D3, MAPKERK kinase 1 (MEK), ribosomal protein S6 (S6), phosphorylated ribosomal protein S6 (pS6), extracellular signal-regulated kinase (ERK), phosphorylated extracellular signal-regulated kinase (pERK), protein kinase B (Akt), phosphorylated protein kinase B (pAkt), caspase-2, total S6, and retinoblastoma binding protein (RBBP). Also provided are kits that include at least three antibodies that specifically bind to one or more of these markers.

    摘要翻译： 本文提供了用于确定治疗患者中的多囊肾病（PKD），诊断患者中的PKD，分级患者中的PKD和监测患者中的PKD的功效的方法。 这些方法包括确定选自增殖细胞核抗原（PCNA），细胞周期蛋白D1，细胞周期蛋白D3，MAPKERK激酶1（MEK），核糖体蛋白S6（S6），磷酸化核糖体蛋白 S6（pS6），细胞外信号调节激酶（ERK），磷酸化细胞外信号调节激酶（pERK），蛋白激酶B（Akt），磷酸化蛋白激酶B（pAkt），caspase-2，总S6和成视网膜细胞瘤结合蛋白 （RBBP）。 还提供了包含至少三种特异性结合这些标志物中的一种或多种的抗体的试剂盒。

    INHIBITING OR DOWNREGULATING GLYCOGEN SYNTHASE BY CREATING PREMATURE STOP CODONS USING ANTISENSE OLIGONUCLEOTIDES
    43.
    发明公开
    INHIBITING OR DOWNREGULATING GLYCOGEN SYNTHASE BY CREATING PREMATURE STOP CODONS USING ANTISENSE OLIGONUCLEOTIDES 审中-公开
    糖原合成酶由产生抑制或下调提前终止反义寡核苷酸的密码子

    公开(公告)号：EP3146050A1

    公开(公告)日：2017-03-29

    申请号：EP15727794.8

    申请日：2015-05-22

    申请人： Genzyme Corporation

    发明人： NELSON, Carol A. ,  WENTWORTH, Bruce M. ,  SCHEULE, Ronald K. ,  WEEDEN, Timothy E. ,  CLAYTON, Nicholas P.

    IPC分类号： C12N15/113 ,  A61K31/712 ,  A61K31/7105

    摘要： The present disclosure relates to antisense oligonucleotides (AONs) for modulating the expression of glycogen synthase. AONs of the present disclosure may be useful in treating diseases associated with the modulation of the expression of the enzyme glycogen synthase, such as Pompe disease. Also provided by the present disclosure are compositions comprising AONs, as well as methods of down regulating mRNA coding for glycogen synthase, methods for reducing glycogen synthase in skeletal and cardiac muscle, and methods for treating Pompe disease.

    摘要翻译： 本发明涉及反义寡核苷酸（AON），用于调制糖原合成酶的表达。 本发明的AON可用于治疗与酶糖原合酶,:如蓬珀病的表达的调节相关的疾病是有用的。 因此，通过本发明提供了包含AON，以及向下的方法调节mRNA编码的糖原合酶，减少在骨骼肌和心肌糖原合成酶的方法，以及治疗庞贝氏症的方法。

    TREATMENT OF MULTIPLE SCLEROSIS (MS) WITH CAMPATH-1H
    50.
    发明公开
    TREATMENT OF MULTIPLE SCLEROSIS (MS) WITH CAMPATH-1H 审中-公开
    百慕达馄饨（SK）MIT CAMPATH-1H

    公开(公告)号：EP3028718A1

    公开(公告)日：2016-06-08

    申请号：EP15191491.8

    申请日：2007-09-11

    申请人： Alcafleu Management GmbH & Co. KG ,  Genzyme Corporation

    发明人： SACHSE, Andreas ,  MARGOLIN, David Harris

    IPC分类号： A61K39/395 ,  C07K16/28

    CPC分类号： C07K16/2893 ,  A61K2039/505 ,  A61K2039/545 ,  C07K2317/24

    摘要： A method for treatment of multiple sclerosis (MS) with Campath-1H with significant efficacy and a favourable safety profile is described, which offers an acceptable benefit/risk ratio. Especially described is the use of Campath-1H (alemtuzumab) for the production of a medicament for the treatment of multiple sclerosis (MS), comprising a first treatment cycle followed by at least one further treatment cycle of Campath-1H (alemtuzumab), in which each treatment cycle comprises 1-5 daily doses which are applied on consecutive days, wherein the daily dose is >0 and ≤ 12 mg, and wherein each treatment cycle is separated from the next cycle by at least 1 - 24 months. Also described are treatment regimens comprising the administration of less than 12 mg/day of Campath-1H for a period of 1-5 consecutive days.

    摘要翻译： 描述了用Campath-1H治疗多发性硬化（MS）的方法，其具有显着的功效和良好的安全性，其提供了可接受的益处/风险比。 特别描述了Campath-1H（alemtuzumab）用于生产治疗多发性硬化症（MS）的药物的用途，其包括第一个治疗周期，随后是至少一个Campath-1H（alemtuzumab）的治疗周期， 每个治疗周期包括连续施用的1-5个日剂量，其中日剂量> 0和‰¤12mg，并且其中每个治疗周期与下一个周期分开至少1-2个月。 还描述了包括施用少于12mg /天的Campath-1H连续1-5天的治疗方案。