{"id":49864,"date":"2018-09-22T01:00:00","date_gmt":"2018-09-26T02:02:38","guid":{"rendered":"http:\/\/www.05921.net\/index.php\/2018\/09\/26\/amicus%e5%85%ac%e5%8f%b83%e4%ba%bf%e7%be%8e%e5%85%83%e6%94%b6%e8%b4%ad10%e4%b8%aa%e5%9f%ba%e5%9b%a0%e7%96%97%e6%b3%95%e9%a1%b9%e7%9b%ae\/"},"modified":"2018-09-26T10:02:39","modified_gmt":"2018-09-26T02:02:39","slug":"amicus%e5%85%ac%e5%8f%b83%e4%ba%bf%e7%be%8e%e5%85%83%e6%94%b6%e8%b4%ad10%e4%b8%aa%e5%9f%ba%e5%9b%a0%e7%96%97%e6%b3%95%e9%a1%b9%e7%9b%ae","status":"publish","type":"post","link":"https:\/\/www.05921.net\/index.php\/49864\/","title":{"rendered":"Amicus\u516c\u53f83\u4ebf\u7f8e\u5143\u6536\u8d2d10\u4e2a\u57fa\u56e0\u7597\u6cd5\u9879\u76ee"},"content":{"rendered":"<div class=\"bpp-post-content\">\n<p style=\"text-align: center;\"><img class=\"\"       style=\"\" src=\"http:\/\/www.05921.net\/wp-content\/uploads\/2018\/09\/9-1537927359.jpeg\" alt=\"Amicus\u516c\u53f83\u4ebf\u7f8e\u5143\u6536\u8d2d10\u4e2a\u57fa\u56e0\u7597\u6cd5\u9879\u76ee\" title=\"Amicus\u516c\u53f83\u4ebf\u7f8e\u5143\u6536\u8d2d10\u4e2a\u57fa\u56e0\u7597\u6cd5\u9879\u76ee\"  \/><\/p>\n<p style=\"margin-top: 20px;font-size: 16px;color: rgb(102, 102, 102);line-height: 28px;word-break: break-all;font-family: &quot;Microsoft YaHei&quot;, \u5fae\u8f6f\u96c5\u9ed1, Simsun, \u5b8b\u4f53;white-space: normal;background-color: rgb(255, 255, 255);text-indent: 2em;\">\u8fd1\u65e5\uff0c<strong><span style=\"color: rgb(0, 51, 153);\">Amicus Therapeutics<\/span><\/strong>\u516c\u53f8\u901a\u8fc7\u6536\u8d2d<strong>Celenex<\/strong>\u516c\u53f8\u7b7e\u7f72\u4e86\u6700\u7ec8\u534f\u8bae\u6765\u83b7\u5f97\u5728\u5168\u56fd\u513f\u7ae5\u533b\u9662\uff08\u7f8e\u56fd\uff09\u8bb8\u53ef\u7684<strong>10\u4e2a\u57fa\u56e0\u7597\u6cd5\u9879\u76ee<\/strong>\u3002\u8fd9\u4e9b\u9879\u76ee\u8ba1\u5212\u7531\u5168\u56fd\u513f\u7ae5\u533b\u9662\u548c\u4fc4\u4ea5\u4fc4\u5dde\u7acb\u5927\u5b66\u7814\u7a76\u6240\u7684\u57fa\u56e0\u6cbb\u7597\u4e2d\u5fc3\u5f00\u53d1\u3002Celenex\u662f\u5168\u56fd\u513f\u7ae5\u533b\u9662\u7684\u5206\u652f\u673a\u6784\u3002<\/p>\n<p style=\"margin-top: 20px;font-size: 16px;color: rgb(102, 102, 102);line-height: 28px;word-break: break-all;font-family: &quot;Microsoft YaHei&quot;, \u5fae\u8f6f\u96c5\u9ed1, Simsun, \u5b8b\u4f53;white-space: normal;background-color: rgb(255, 255, 255);text-align: center;\"><img align=\"center\" class=\"\"     style=\"border-width: initial;border-style: none;border-color: initial;max-width: 604px;\" src=\"http:\/\/www.05921.net\/wp-content\/uploads\/2018\/09\/6-1537927359.jpg\" alt=\"Amicus\u516c\u53f83\u4ebf\u7f8e\u5143\u6536\u8d2d10\u4e2a\u57fa\u56e0\u7597\u6cd5\u9879\u76ee\" title=\"Amicus\u516c\u53f83\u4ebf\u7f8e\u5143\u6536\u8d2d10\u4e2a\u57fa\u56e0\u7597\u6cd5\u9879\u76ee\"  \/><\/p>\n<p style=\"margin-top: 20px;font-size: 16px;color: rgb(102, 102, 102);line-height: 28px;word-break: break-all;font-family: &quot;Microsoft YaHei&quot;, \u5fae\u8f6f\u96c5\u9ed1, Simsun, \u5b8b\u4f53;white-space: normal;background-color: rgb(255, 255, 255);text-indent: 2em;\">\u6839\u636e\u8be5\u4ea4\u6613\u6761\u6b3e\uff0cAmicus\u516c\u53f8\u5c06\u4ee5\u73b0\u91d1\u652f\u4ed8<strong>1\u4ebf\u7f8e\u5143<\/strong>\u9884\u4ed8\u6b3e\u3002Celenex\u80a1\u4e1c\u4eec\u6709\u8d44\u683c\u83b7\u5f97\u9ad8\u8fbe1500\u4e07\u7f8e\u5143\u7684\u53d1\u5c55\u91cc\u7a0b\u7891\u548c<strong>2.62\u4ebf\u7f8e\u5143<\/strong>\u7684\u6ce8\u518c\u7533\u62a5\u548c\u6279\u51c6\u91cc\u7a0b\u7891\u3002Amicus\u8868\u793a\uff0c\u5728\u672a\u6765\u56db\u5e74\u7684\u91cc\u7a0b\u7891\u4ed8\u6b3e\u4e2d\uff0c\u9884\u8ba1\u4e0d\u4f1a\u8d85\u8fc77500\u4e07\u7f8e\u5143\u3002<\/p>\n<p style=\"margin-top: 20px;font-size: 16px;color: rgb(102, 102, 102);line-height: 28px;word-break: break-all;font-family: &quot;Microsoft YaHei&quot;, \u5fae\u8f6f\u96c5\u9ed1, Simsun, \u5b8b\u4f53;white-space: normal;background-color: rgb(255, 255, 255);text-indent: 2em;\">\u6240\u6709\u7684\u9879\u76ee\u90fd\u662f\u5173\u4e8e<strong>\u795e\u7ecf\u7cfb\u7edf<span style=\"color: rgb(0, 51, 153);\">\u6eb6\u9176\u4f53\u8d2e\u79ef\u75c7<\/span><\/strong>\u3002\u5176\u4e2d3\u4e2a\u4e3b\u8981\u9879\u76ee\u9488\u5bf9<strong>\u8d1d\u6566\u6c0f\u75c7(Batten Disease)<\/strong>\uff0c\u5206\u522b\u662fCLN6\u3001CLN3\u548cCLN8\u3002<strong><span style=\"color: rgb(0, 51, 153);\">Batten\u75c5<\/span><\/strong>\u4e5f\u88ab\u79f0\u4e3a\u795e\u7ecf\u5143\u8721\u6837\u8102\u8910\u8d28\u6c89\u79ef\u75c7\uff08NCL\uff09\uff0c\u4e00\u79cd\u7f55\u89c1\u4e14\u5371\u53ca\u751f\u547d\u7684\u75be\u75c5\u3002<\/p>\n<p style=\"margin-top: 20px;font-size: 16px;color: rgb(102, 102, 102);line-height: 28px;word-break: break-all;font-family: &quot;Microsoft YaHei&quot;, \u5fae\u8f6f\u96c5\u9ed1, Simsun, \u5b8b\u4f53;white-space: normal;background-color: rgb(255, 255, 255);text-indent: 2em;\">Amicus\u8463\u4e8b\u957f\u517c\u9996\u5e2d\u6267\u884c\u5b98John F. Crowley\u5728\u4e00\u4efd\u58f0\u660e\u4e2d\u8bf4\uff0c\u201c\u8fd9\u4e9b\u57fa\u56e0\u7597\u6cd5\u9879\u76ee\u7684\u8bb8\u53ef\u548c\u6536\u8d2d\u63d0\u4f9b\u4e86\u4e00\u4e2a\u5f88\u597d\u7684\u673a\u4f1a\uff0c\u53ef\u4ee5\u6539\u53d8\u6210\u5343\u4e0a\u4e07\u60a3\u6709\u4e00\u4e9b\u6700\u5177\u7834\u574f\u6027\u7684\u6eb6\u9176\u4f53\u8d2e\u79ef\u75c7\u7684\u513f\u7ae5\u751f\u6d3b\uff0c\u76ee\u524d\u51e0\u4e4e\u6ca1\u6709\u6cbb\u7597\u9009\u62e9\u3002Drs Brian Kaspar\u548cKathrin Meyer\u5728\u5168\u56fd\u513f\u7ae5\u533b\u9662\u7684\u5f00\u521b\u6027\u5de5\u4f5c\uff0c\u4ee5\u53ca\u4fc4\u4ea5\u4fc4\u5dde\u7acb\u5927\u5b66\u7684\u6559\u6388Arthur Burghes\u535a\u58eb\uff0c\u4ed6\u4eec\u5728\u5728\u8fd9\u4e9b\u9879\u76ee\u4e0a\u7684\u4ed8\u51fa\u5df2\u7ecf\u83b7\u5f97\u4e86\u975e\u5e38\u5f3a\u800c\u4e14\u4e00\u81f4\u6027\u7684\u4e34\u5e8a\u524d\u7814\u7a76\u7ed3\u679c\uff0c\u76ee\u524d\u5728CLN6 Batten\u75be\u75c5\u4e2d\uff0c\u8fd9\u4e9b\u9f13\u52b1\u4e86\u5b69\u5b50\u4eec\u4e2d\u65e9\u671f\u7684\u7814\u7a76\u7ed3\u679c\u3002\u201d<\/p>\n<p style=\"margin-top: 20px;font-size: 16px;color: rgb(102, 102, 102);line-height: 28px;word-break: break-all;font-family: &quot;Microsoft YaHei&quot;, \u5fae\u8f6f\u96c5\u9ed1, Simsun, \u5b8b\u4f53;white-space: normal;background-color: rgb(255, 255, 255);text-indent: 2em;\">\u8be5\u4ea4\u6613\u4e2d\u6240\u6536\u8d2d\u7684\u6240\u670910\u4e2a\u9879\u76ee\u90fd\u91c7\u7528\u4e86<strong>\u9798\u5185\u7ed9\u836f<\/strong>\u3002\u8be5\u65b9\u6cd5\u5229\u7528AAV\u8f7d\u4f53\u6280\u672f\uff0c\u8be5\u6280\u672f\u5df2\u6210\u529f\u7528\u4e8e\u5176\u4ed6\u7f55\u89c1\u7684\u4e2d\u67a2\u795e\u7ecf\u7cfb\u7edf\uff08CNS\uff09\u9002\u5e94\u75c7\uff0c\u4f8b\u5982\u810a\u9ad3\u6027\u808c\u840e\u7f29\u75c7\uff08SMA\uff09\u300210\u4e2a\u9879\u76ee\u4e2d\u67092\u9879\u5df2\u8fdb\u5165\u4eba\u4f53\u4e34\u5e8a\u8bd5\u9a8c\u9636\u6bb5\u3002Amicus\u516c\u53f8\u5f53\u524d\u9500\u552eGalafold\u6765\u6cbb\u7597<strong>\u6cd5\u5e03\u745e\u6c0f\u75c7\uff08Fabry disease\uff09<\/strong>\uff0c\u76ee\u524d\u5176\u6b63\u5728\u5f00\u53d1\u9488\u5bf9<strong>\u5e9e\u8d1d\u6c0f\u75c7\uff08<\/strong><strong>Pompe disease<\/strong><strong>\uff09<\/strong>\u7684\u7b2c\u4e8c\u79cd\u7597\u6cd5\u3002\u6b64\u6b21\u6536\u8d2d\u663e\u8457\u5730\u6269\u5927\u4e86\u516c\u53f8\u7684\u7ba1\u9053\u836f\u7269\uff0c\u5c06\u91cd\u70b9\u4ece\u7a33\u5b9a\u4f46\u672a\u6cbb\u6108\u6eb6\u9176\u4f53\u8d2e\u79ef\u75c7\u7684\u836f\u7269\u8f6c\u79fb\u5230\u5177\u6709\u6f5c\u5728\u7684\u6cbb\u7597\u65b9\u6cd5\u4e0a\u3002<\/p>\n<p style=\"margin-top: 20px;font-size: 16px;color: rgb(102, 102, 102);line-height: 28px;word-break: break-all;font-family: &quot;Microsoft YaHei&quot;, \u5fae\u8f6f\u96c5\u9ed1, Simsun, \u5b8b\u4f53;white-space: normal;background-color: rgb(255, 255, 255);text-indent: 2em;\">STAT\u5199\u9053\uff0c\u201c\u5728\u8fd9\u4e9b\u65b0\u6536\u8d2d\u7684\u57fa\u56e0\u7597\u6cd5\u4e2d\uff0c\u6709\u4e00\u79cd\u5c31\u662f\u9488\u5bf9Batten\u75c5\u7684CLN6\u7a81\u53d8\u5f62\u5f0f\u3002\u597d\u83b1\u575e\u7535\u5f71\u5236\u7247\u4ebaGordon Gray\u548c\u4ed6\u7684\u59bb\u5b50Kristen\u7684\u4e24\u4e2a\u5973\u513f\u5929\u751f\u5c31\u60a3\u6709\u8fd9\u79cd\u5f62\u5f0f\u7684Batten\u75c7\u72b6\u3002\u4e3a\u6b64Gray\u592b\u5987\u7ec4\u5efa\u4e86\u4e00\u4e2a\u79d1\u5b66\u57fa\u91d1\u4f1a\uff0c\u540e\u6765\u5171\u540c\u521b\u7acb\u4e86Celenex\uff0c\u4f5c\u4e3a\u4e00\u79cd\u65b9\u5f0f\u6765\u4e3a\u5973\u513f\u548c\u60a3\u6709\u8fd9\u79cd\u75be\u75c5\u7684\u4eba\u627e\u5230\u6cbb\u7597\u65b9\u6cd5\u3002\u201dGordon Gray\u6700\u51fa\u540d\u7684\u662f\u4ed6\u5728\u56e0\u4ed6\u7684\u7535\u5f71\u4f5c\u54c1\u300a\u5fc3\u7075\u6295\u624b\u300b\u3001\u300a\u4e00\u4ee3\u9a84\u9a6c\u300b\u548c\u300a\u4e07\u592b\u83ab\u654c\u300b\u800c\u88ab\u5927\u4f17\u6240\u719f\u77e5\u3002<\/p>\n<p style=\"margin-top: 20px;font-size: 16px;color: rgb(102, 102, 102);line-height: 28px;word-break: break-all;font-family: &quot;Microsoft YaHei&quot;, \u5fae\u8f6f\u96c5\u9ed1, Simsun, \u5b8b\u4f53;white-space: normal;background-color: rgb(255, 255, 255);text-indent: 2em;\">Crowley\u8bf4\u5230\uff0c\u201cGray\u592b\u5987\u7aed\u5c3d\u5168\u529b\u4e0e\u8fd9\u4e9b\u7814\u7a76\u4eba\u5458\u5408\u4f5c\uff0c\u4e3a\u4ed6\u4eec\u7684\u5973\u513f\u4ee5\u53ca\u5f53\u524d\u6570\u5343\u4eba\u6765\u63a8\u8fdb\u8fd9\u4e9b\u53ef\u80fd\u633d\u6551\u751f\u547d\u7684\u836f\u7269\u3002\u6211\u5f88\u8363\u5e78\u4ed6\u4eec\u548c\u4ed6\u4eec\u7684\u7814\u7a76\u56e2\u961f\u9009\u62e9\u5c06\u8fd910\u4e2a\u9879\u76ee\u59d4\u6258\u7ed9Amicus\u516c\u53f8\u5145\u6ee1\u6fc0\u60c5\u7684\u79d1\u5b66\u5bb6\u548c\u4f01\u4e1a\u5bb6\u56e2\u961f\u3002\u5bf9Amicus\u516c\u53f8\u8fdb\u519b\u57fa\u56e0\u6cbb\u7597\uff0c\u6211\u60f3\u4e0d\u51fa\u66f4\u597d\u7684\u8fdb\u5165\u57fa\u7840\u6846\u67b6\u3002\u201d<\/p>\n<p style=\"margin-top: 20px;font-size: 16px;color: rgb(102, 102, 102);line-height: 28px;word-break: break-all;font-family: &quot;Microsoft YaHei&quot;, \u5fae\u8f6f\u96c5\u9ed1, Simsun, \u5b8b\u4f53;white-space: normal;background-color: rgb(255, 255, 255);text-indent: 2em;\">\u9488\u5bf9Batten\u75c5\uff0c<strong>BioMarin<\/strong>\u5236\u836f\u516c\u53f8\u4f9b\u5e94\u7684Brineura\u662f\u4e00\u79cd\u513f\u7ae5CLN2\u578bBatten\u75c5\u7684\u6cbb\u7597\u836f\u7269\uff1b<strong>Abeona Therapeutics<\/strong>\u516c\u53f8\u4e5f\u6b63\u5728\u5f00\u53d1CLN1\u578bBatten\u7684\u57fa\u56e0\u7597\u6cd5\u836f\u7269\u3002<\/p>\n<p style=\"margin-top: 20px;font-size: 16px;color: rgb(102, 102, 102);line-height: 28px;word-break: break-all;font-family: &quot;Microsoft YaHei&quot;, \u5fae\u8f6f\u96c5\u9ed1, Simsun, \u5b8b\u4f53;white-space: normal;background-color: rgb(255, 255, 255);text-indent: 2em;\">Kathrin Meyer\u5728\u4e00\u4efd\u58f0\u660e\u4e2d\u8bf4\uff0c\u201c\u8fc4\u4eca\u4e3a\u6b62\u6211\u4eec\u5728CLN6\u3001CLN3\u548cCLN8\u4e2d\u6240\u770b\u5230\u7684\u4e34\u5e8a\u524d\u6982\u5ff5\u9a8c\u8bc1\u8fdb\u4e00\u6b65\u652f\u6301\u4e86\u6211\u4eec\u5728\u5168\u56fd\u513f\u7ae5\u533b\u9662CNS\u9057\u4f20\u75c5\u4e2d\u5f00\u53d1\u7684AAV\u8f7d\u4f53\u7684\u9002\u7528\u6027\u3002\u6211\u575a\u4fe1Amicus\u662f\u63a8\u8fdb\u8fd9\u4e9b\u9879\u76ee\u7684\u6700\u4f73\u7684\u79d1\u5b66\u53ca\u4e34\u5e8a\u5408\u4f5c\u4f19\u4f34\uff0c\u5e76\u671f\u5f85\u4e0eAmicus\u56e2\u961f\u79ef\u6781\u5408\u4f5c\u5f00\u53d1\u8fd9\u4e9b\u91cd\u8981\u7684\u6f5c\u5728\u7597\u6cd5\uff0c\u5e76\u5c3d\u5feb\u8ba9\u5c3d\u53ef\u80fd\u591a\u7684\u5b69\u5b50\u4eec\u63a5\u89e6\u5230\u4ed6\u4eec\u7684\u7597\u6cd5\u3002\u8fd9\u4e9b\u7597\u6cd5\u771f\u7684\u6709\u53ef\u80fd\u6539\u53d8\u4ed6\u4eec\u7684\u751f\u6d3b\u3002\u201d<\/p>\n<p style=\"text-align: left;\">\n<\/p><\/div>\n","protected":false},"excerpt":{"rendered":"<p>\u8fd1\u65e5\uff0cAmicus Therapeutics\u516c\u53f8\u901a\u8fc7\u6536\u8d2dCelenex\u516c\u53f8\u7b7e\u7f72\u4e86\u6700\u7ec8\u534f\u8bae\u6765\u83b7\u5f97\u5728\u5168\u56fd\u513f\u7ae5\u533b\u9662\uff08 [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":49865,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":[],"categories":[2],"tags":[],"_links":{"self":[{"href":"https:\/\/www.05921.net\/index.php\/wp-json\/wp\/v2\/posts\/49864"}],"collection":[{"href":"https:\/\/www.05921.net\/index.php\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.05921.net\/index.php\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.05921.net\/index.php\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/www.05921.net\/index.php\/wp-json\/wp\/v2\/comments?post=49864"}],"version-history":[{"count":1,"href":"https:\/\/www.05921.net\/index.php\/wp-json\/wp\/v2\/posts\/49864\/revisions"}],"predecessor-version":[{"id":49868,"href":"https:\/\/www.05921.net\/index.php\/wp-json\/wp\/v2\/posts\/49864\/revisions\/49868"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.05921.net\/index.php\/wp-json\/wp\/v2\/media\/49865"}],"wp:attachment":[{"href":"https:\/\/www.05921.net\/index.php\/wp-json\/wp\/v2\/media?parent=49864"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.05921.net\/index.php\/wp-json\/wp\/v2\/categories?post=49864"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.05921.net\/index.php\/wp-json\/wp\/v2\/tags?post=49864"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}