METHODS FOR TREATING SUBJECTS WITH PRADER-WILLI SYNDROME OR SMITH-MAGENIS SYNDROME
    1.
    发明申请
    METHODS FOR TREATING SUBJECTS WITH PRADER-WILLI SYNDROME OR SMITH-MAGENIS SYNDROME 审中-公开
    用PRADER-WILLI综合征或史密斯综合征治疗子宫的方法

    公开(公告)号:WO2016077629A1

    公开(公告)日:2016-05-19

    申请号:PCT/US2015/060455

    申请日:2015-11-12

    申请人: ESSENTIALIS, INC.

    发明人: COWEN, Neil M.

    摘要: Provided are immediate or prolonged administration of certain potassium ATP (K ATP ) channel openers, optionally in combination with growth hormone, to a subject to achieve novel pharmacodynamic, pharmacokinetic, therapeutic, physiological, metabolic and compositional outcomes in the treatment of diseases or conditions involving K ATP channels. Also provided are pharmaceutical formulations, methods of administration and dosing of K ATP channel openers that achieve these outcomes and reduce the incidence of adverse effects in treated individuals. Further provided are methods of co-administering K ATP channel openers with other drugs (e.g., in combination with growth hormone) to treat diseases of humans and animals (e.g., Prader-Willi Syndrome (PWS), Smith-Magenis syndrome (SMS), and the like.

    摘要翻译: 提供某些钾ATP(KATP)通道开放剂(任选与生长激素组合)立即或长期给予受试者,以在治疗涉及KATP的疾病或病症中获得新颖的药效学,药代动力学,治疗学,生理学,代谢和组合结果 通道。 还提供了实现这些结果并降低治疗个体的不良反应发生率的药物制剂,给药方法和KATP通道开放剂的给药方法。 还提供了将KATP通道开放剂与其他药物(例如与生长激素组合)共同治疗以治疗人和动物疾病(例如,Prader-Willi综合征(PWS),Smith-Magenis综合征(SMS))和 类似。

    RICIN INHIBITORS AND METHODS FOR USE THEREOF
    3.
    发明申请
    RICIN INHIBITORS AND METHODS FOR USE THEREOF 审中-公开
    RICIN抑制剂及其使用方法

    公开(公告)号:WO01073438A1

    公开(公告)日:2001-10-04

    申请号:PCT/US2001/009400

    申请日:2001-03-23

    摘要: Ricin A-chain is an N-glycosidase that attacks ribosomal RNA at a highly conserved adenine residue. Crystallographic studies show that not only adenine and formycin, but also pterin-based rings can bind in the ricin active site. For a better understanding of the recognition mode between ricin, and adenine-like rings, the interaction energies and geometries were calculated for a number of complexes. Shiga toxin, a compound essentially identical to the protein originally isolated from Shigella dysenteriae , has an active protein chain that is a homologue of the ricin active chain, and catalyzes the same depurination reaction. The present invention is drawn to identifying inhibitors of ricin and Shiga toxin, using methods molecular mechanics and ab initio methods and using the identified inhibitors as antidotes to ricin or Shiga toxin, or to facilitate immunotoxin treatment by controlling non-specific cytotoxicity.

    摘要翻译: 蓖麻毒素A链是一种在高度保守的腺嘌呤残基下攻击核糖体RNA的N-糖苷酶。 晶体学研究表明,不仅腺嘌呤和霉素,而且基于蝶呤的环可以在蓖麻毒素活性位点结合。 为了更好地了解蓖麻毒蛋白和腺嘌呤样环之间的识别模式,计算了多种复合物的相互作用能和几何形状。 志贺毒素,与最初从痢疾志贺氏菌分离的蛋白质基本相同的化合物具有活性蛋白质链,其是蓖麻毒素活性链的同系物,并催化相同的去血液反应。 本发明涉及使用分子力学和起始方法的方法鉴定蓖麻毒素和志贺毒素的抑制剂,并使用鉴定的抑制剂作为蓖麻毒蛋白或志贺毒素的解毒剂,或通过控制非免疫毒素来促进免疫毒素治疗 特异性细胞毒性。

    [1,2,4]THIADIAZINE 1,1-DIOXIDE COMPOUNDS FOR LOWERING SERUM URIC ACID
    4.
    发明申请
    [1,2,4]THIADIAZINE 1,1-DIOXIDE COMPOUNDS FOR LOWERING SERUM URIC ACID 审中-公开
    [1,2,4]噻二嗪1,1-二氧化物,用于降低血清尿酸

    公开(公告)号:WO2012170536A1

    公开(公告)日:2012-12-13

    申请号:PCT/US2012/041106

    申请日:2012-06-06

    发明人: FREDDO, James, L.

    IPC分类号: C07D285/22 A61K31/5415

    CPC分类号: A61K31/549

    摘要: The present invention describes [1,2,4]thiadiazine 1,1-dioxide compounds and pharmaceutically acceptable salts thereof, which are useful in lowering serum uric acid in a patient in need thereof comprising administering to the patient a therapeutically or prophylactically effective amount of a [1,2,4]thiadiazine 1,1- dioxide compound.

    摘要翻译: 本发明描述了可用于降低有需要的患者血清尿酸的[1,2,4]噻二嗪1,1-二氧化物化合物及其药学上可接受的盐,其包括向患者施用治疗或预防有效量的 [1,2,4]噻二嗪1,1-二氧化物。