{"id":49067,"date":"2018-09-21T01:00:00","date_gmt":"2018-09-26T01:54:47","guid":{"rendered":"http:\/\/www.05921.net\/index.php\/2018\/09\/26\/%e9%80%9f%e9%80%92-%e5%9b%8a%e8%8e%b710%e9%a1%b9%e5%9c%a8%e7%a0%94%e5%9f%ba%e5%9b%a0%e7%96%97%e6%b3%95%ef%bc%8camicus%e6%8b%93%e5%b1%95%e7%bd%95%e8%a7%81%e7%97%85%e7%ae%a1%e7%ba%bf\/"},"modified":"2018-09-26T09:54:49","modified_gmt":"2018-09-26T01:54:49","slug":"%e9%80%9f%e9%80%92-%e5%9b%8a%e8%8e%b710%e9%a1%b9%e5%9c%a8%e7%a0%94%e5%9f%ba%e5%9b%a0%e7%96%97%e6%b3%95%ef%bc%8camicus%e6%8b%93%e5%b1%95%e7%bd%95%e8%a7%81%e7%97%85%e7%ae%a1%e7%ba%bf","status":"publish","type":"post","link":"https:\/\/www.05921.net\/index.php\/49067\/","title":{"rendered":"\u901f\u9012 | \u56ca\u83b710\u9879\u5728\u7814\u57fa\u56e0\u7597\u6cd5\uff0cAmicus\u62d3\u5c55\u7f55\u89c1\u75c5\u7ba1\u7ebf"},"content":{"rendered":"
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\u258e\u836f\u660e<\/span><\/strong>\u5eb7\u5fb7\/\u62a5\u9053<\/span><\/strong><\/strong><\/span><\/strong><\/strong><\/span><\/strong><\/strong><\/strong><\/span><\/strong><\/strong><\/span><\/strong><\/strong><\/h1>\n

\u4eca\u65e5\uff0cAmicus Therapeutics\u516c\u53f8<\/strong>\u5ba3\u5e03\uff0c\u901a\u8fc7\u6536\u8d2dCelenex\u516c\u53f8\uff0c\u5b83\u4ece\u5168\u56fd\u513f\u7ae5\u533b\u9662\uff08Nationwide Children\u2019s Hospital\uff09\u548c\u4fc4\u4ea5\u4fc4\u5dde\u7acb\u5927\u5b66\uff08Ohio State University\uff09\u83b7\u5f9710\u4e2a\u57fa\u56e0\u7597\u6cd5\u9879\u76ee\u7684\u5168\u7403\u7814\u53d1\u8bb8\u53ef\u3002\u5176\u4e2d\u6cbb\u7597CLN6\uff0cCLN3\uff0c\u548cCLN8 Batten\u75c5\uff08Batten disease\uff09\u7684\u4e3b\u6253\u7814\u7a76\u9879\u76ee\u53ef\u80fd\u6210\u4e3a\u6cbb\u7597\u8fd9\u4e9b\u4e25\u91cd\u7f55\u89c1\u75c5\u7684\u6cbb\u6108\u6027\u7597\u6cd5\u3002<\/strong><\/span><\/p>\n


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\"\u901f\u9012<\/p>\n


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Batten\u75c5\u53c8\u79f0\u4e3a\u795e\u7ecf\u5143\u8721\u6837\u8d28\u8102\u8910\u8d28\u6c89\u79ef\u75c5\uff08neuronal ceroid lipofuscinosis, NCL\uff09\uff0c\u662f\u4e00\u7c7b\u5371\u53ca\u751f\u547d\u7684\u7f55\u89c1\u9057\u4f20\u6027\u795e\u7ecf\u7cfb\u7edf\u75be\u75c5\u3002\u8fd9\u7c7b\u75be\u75c5\u5c5e\u4e8e\u6eb6\u9176\u4f53\u8d2e\u79ef\u75c7\uff08LSD\uff09\uff0c\u7531\u4e8e\u5728\u4e0d\u540cCLN\u57fa\u56e0\u4e2d\u51fa\u73b0\u7684\u7a81\u53d8\u800c\u5bfc\u81f4\u6eb6\u9176\u4f53\u964d\u89e3\u7ec6\u80de\u5185\u5927\u5206\u5b50\u7684\u529f\u80fd\u5931\u5e38\u3002\u60a3\u8005\u901a\u5e38\u5728\u7ae5\u5e74\u65f6\u5c31\u4f1a\u8868\u73b0\u51fa\u75c7\u72b6\uff0c\u5927\u591a\u6570\u60a3\u8005\u65e0\u6cd5\u957f\u5927\u6210\u4eba\u3002\u8fd9\u4e00\u9886\u57df\u4f9d\u7136\u5b58\u5728\u7740\u4e25\u91cd\u672a\u88ab\u6ee1\u8db3\u7684\u533b\u7597\u9700\u6c42\u3002<\/span><\/p>\n


<\/p>\n

Amicus\u516c\u53f8\u6536\u83b7\u768410\u9879\u57fa\u56e0\u7597\u6cd5\u90fd\u662f\u9776\u5411LSD\u7684\u7814\u7a76\u9879\u76ee\u3002\u5b83\u4eec\u901a\u8fc7\u4f7f\u7528AAV\u8f7d\u4f53\u5c06\u529f\u80fd\u6b63\u5e38\u7684\u57fa\u56e0\u901a\u8fc7\u9798\u5185\u6ce8\u5c04\uff08intrathecal injection\uff09\u8fd0\u9001\u5230\u4e2d\u67a2\u795e\u7ecf\u7cfb\u7edf\u5185\u90e8\uff0c\u5e0c\u671b\u80fd\u591f\u8fbe\u5230\u4e00\u6b21\u6cbb\u7597\u5c31\u80fd\u957f\u671f\uff0c\u751a\u81f3\u7ec8\u8eab\u7f13\u89e3\u60a3\u8005\u75c7\u72b6\u7684\u6548\u679c<\/strong>\u3002\u8fd9\u4e9b\u9879\u76ee\u4f7f\u7528\u7684AAV\u75c5\u6bd2\u8f7d\u4f53\u7684\u5b89\u5168\u6027\u548c\u6709\u6548\u6027\u5df2\u7ecf\u5728\u5176\u5b83\u57fa\u56e0\u7597\u6cd5\u7684\u4e34\u5e8a\u8bd5\u9a8c\u4e2d\u5f97\u5230\u9a8c\u8bc1\u3002<\/span><\/p>\n


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\"\u901f\u9012<\/p>\n

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\u8fd910\u4e2a\u7814\u7a76\u9879\u76ee\u4e2d\uff0c\u6cbb\u7597\u56e0\u4e3aCLN6<\/em>\u548cCLN3<\/em>\u7684\u57fa\u56e0\u7a81\u53d8\u5bfc\u81f4\u7684Batten\u75c5\u7684\u57fa\u56e0\u7597\u6cd5\u5df2\u7ecf\u8fdb\u5165\u4e34\u5e8a\u9636\u6bb5\uff0c\u5176\u4e2d\u6cbb\u7597CLN6 Batten\u75c5\u7684\u57fa\u56e0\u7597\u6cd5\u5df2\u7ecf\u83b7\u5f97\u4e86\u79ef\u6781\u7684\u521d\u6b65\u4e34\u5e8a\u7ed3\u679c\u3002<\/span><\/p>\n


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Amicus\u516c\u53f8\u662f\u4e00\u5bb6\u81f4\u529b\u4e8e\u7814\u53d1\u6cbb\u7597\u7f55\u89c1\u75c5\u7684\u521b\u65b0\u7597\u6cd5\u7684\u751f\u7269\u533b\u836f\u516c\u53f8\u3002\u8be5\u516c\u53f8\u7814\u53d1\u7684<\/span>migalastat\u8fd1\u65e5\u521a\u83b7\u5f97\u7f8e\u56fdFDA\u7684\u6279\u51c6<\/span><\/a>\uff0c\u7528\u4e8e\u6cbb\u7597\u7f55\u89c1\u9057\u4f20\u75c5\u6cd5\u5e03\u91cc\u75c5\uff08Fabry disease\uff09\u3002\u8fd9\u4e00\u5408\u4f5c\u663e\u8457\u6269\u5c55\u4e86\u8be5\u516c\u53f8\u7684\u836f\u7269\u7814\u53d1\u7ba1\u7ebf\uff0c\u5e76\u4e14\u5c06\u7814\u53d1\u91cd\u5fc3\u8f6c\u79fb\u5230\u53ef\u80fd\u6cbb\u6108LSD\u75be\u75c5\u7684\u57fa\u56e0\u7597\u6cd5\u9886\u57df\u3002<\/strong><\/span><\/p>\n


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\u6839\u636e\u5408\u4f5c\u534f\u8bae\uff0cAmicus\u516c\u53f8\u5c06\u4ed8\u51fa1\u4ebf\u7f8e\u5143\u7684\u5148\u671f\u4ed8\u6b3e\u3002\u6839\u636e\u7814\u53d1\u548c\u76d1\u7ba1\u91cc\u7a0b\u7891\uff0cCelenex\u53ef\u80fd\u83b7\u5f97\u9ad8\u8fbe2.77\u4ebf\u7f8e\u5143\u7684\u540e\u7eed\u4ed8\u6b3e\u3002<\/span><\/p>\n


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\u201c\u83b7\u5f97\u8fd9\u4e9b\u57fa\u56e0\u7597\u6cd5\u9879\u76ee\u4e3a\u6539\u53d8\u4e0a\u5343\u540dLSD\u513f\u7ae5\u60a3\u8005\u7684\u751f\u6d3b\u63d0\u4f9b\u4e86\u4e00\u4e2a\u7edd\u4f73\u7684\u673a\u4f1a\uff0c\u76ee\u524d\u4ed6\u4eec\u51e0\u4e4e\u6ca1\u6709\u4efb\u4f55\u7597\u6cd5\u9009\u62e9\uff0c\u201dAmicus\u516c\u53f8\u7684\u4e3b\u5e2d\u517cCEO John F. Crowley\u5148\u751f\u8bf4\uff1a\u201c\u5168\u56fd\u513f\u7ae5\u533b\u9662\u7684Brian Kaspar\u548cKathrin Meyer\u535a\u58eb\uff0c\u4ee5\u53ca\u4fc4\u4ea5\u4fc4\u5dde\u7acb\u5927\u5b66Arthur Burghes\u535a\u58eb\u5728\u8fd9\u4e9b\u9879\u76ee\u4e0a\u7684\u7a81\u7834\u6027\u5de5\u4f5c\uff0c\u5e26\u6765\u4e86\u975e\u5e38\u51fa\u8272\u7684\u4e34\u5e8a\u524d\u7814\u7a76\u7ed3\u679c\u3002\u5728CLN6 Batten\u75c5\u65b9\u9762\uff0c\u4ece\u513f\u7ae5\u60a3\u8005\u4e2d\u83b7\u5f97\u7684\u521d\u6b65\u7ed3\u679c\u4e5f\u975e\u5e38\u4ee4\u4eba\u9f13\u821e\u3002\u201d<\/span><\/p>\n


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Kathrin Meyer\u535a\u58eb\u8865\u5145\u9053\uff1a\u201c\u6211\u575a\u4fe1Amicus\u516c\u53f8\u662f\u63a8\u8fdb\u8fd9\u4e9b\u7814\u7a76\u9879\u76ee\u7684\u6700\u4f73\u79d1\u5b66\u548c\u4e34\u5e8a\u5408\u4f5c\u4f19\u4f34\u3002\u6211\u671f\u5f85\u4e0eAmicus\u7684\u56e2\u961f\u7ee7\u7eed\u5408\u4f5c\uff0c\u5c06\u8fd9\u4e9b\u6f5c\u5728\u7597\u6cd5\u5c3d\u5feb\u5e26\u7ed9\u66f4\u591a\u513f\u7ae5\u60a3\u8005\u3002\u5b83\u4eec\u53ef\u80fd\u7ed9\u60a3\u8005\u4eec\u7684\u751f\u6d3b\u5e26\u6765\u7ffb\u5929\u8986\u5730\u7684\u6539\u5584\u3002\u201d<\/span><\/p>\n


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\u53c2\u8003\u8d44\u6599\uff1a<\/span><\/p>\n

[1] Amicus Therapeutics Acquires Gene Therapy Portfolio of Ten Clinical and Pre-Clinical Stage AAV Programs in Neurologic Lysosomal Storage Disorders. Retrieved September 20, 2018, from http:\/\/ir.amicusrx.com\/news-releases\/news-release-details\/amicus-therapeutics-acquires-gene-therapy-portfolio-ten-clinical <\/span><\/p>\n

[2] Amicus Therapeutics. Retrieved September 20, 2018, from https:\/\/www.amicusrx.com\/<\/span><\/p>\n


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\u672c\u6587\u6765\u81ea\u836f\u660e\u5eb7\u5fb7\u5fae\u4fe1\u56e2\u961f\uff0c\u6b22\u8fce\u8f6c\u53d1\u5230\u670b\u53cb\u5708\uff0c\u8c22\u7edd\u8f6c\u8f7d\u5230\u5176\u4ed6\u5e73\u53f0\uff1b\u5982\u6709\u5f00\u8bbe\u767d\u540d\u5355\u9700\u6c42\uff0c\u8bf7\u5728\u6587\u7ae0\u5e95\u90e8\u7559\u8a00\uff1b\u5982\u6709\u5176\u4ed6\u5408\u4f5c\u9700\u6c42\uff0c\u8bf7\u8054\u7cfbwuxi_media@wuxiapptec.com<\/span>
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