{"id":266732,"date":"2019-05-24T21:00:21","date_gmt":"2019-05-25T04:00:21","guid":{"rendered":"https:\/\/nai500.com\/?p=266732"},"modified":"2019-05-24T21:00:21","modified_gmt":"2019-05-25T04:00:21","slug":"sma%e8%8d%af%e7%89%a9zolgensma%e8%8e%b7%e6%89%b9%ef%bc%8c%e5%9f%ba%e5%9b%a0%e7%96%97%e6%b3%95%e5%bc%80%e5%90%af%e5%85%a8%e6%96%b0%e6%97%b6%e4%bb%a3","status":"publish","type":"post","link":"https:\/\/nai500.com\/zh-hans\/blog\/2019\/05\/sma%e8%8d%af%e7%89%a9zolgensma%e8%8e%b7%e6%89%b9%ef%bc%8c%e5%9f%ba%e5%9b%a0%e7%96%97%e6%b3%95%e5%bc%80%e5%90%af%e5%85%a8%e6%96%b0%e6%97%b6%e4%bb%a3\/","title":{"rendered":"SMA\u836f\u7269Zolgensma\u83b7\u6279\uff0c\u57fa\u56e0\u7597\u6cd5\u5f00\u542f\u5168\u65b0\u65f6\u4ee3"},"content":{"rendered":"

\u76ee\u524d\u5168\u7403\u6709\u8d85\u8fc76,000\u79cd\u9057\u4f20\u75c5\uff0c\u5e76\u4e14\u76ee\u524d\u591a\u6570\u90fd\u8fd8\u6ca1\u6709\u83b7\u6279\u7684\u6cbb\u7597\u624b\u6bb5\u3002\u73b0\u5b9e\u8ba9\u4eba\u6cae\u4e27\uff0c\u4f46\u5e0c\u671b\u4ecd\u5728\u3002\u4f5c\u4e3a\u9057\u4f20\u75c5\u7684\u6839\u6cbb\u624b\u6bb5\uff0c\u57fa\u56e0\u7597\u6cd5\u6b63\u5728\u6162\u6162\u5c55\u73b0\u51fa\u5b83\u7684\u5de8\u5927\u6f5c\u529b\u3002<\/p>\n

\u5468\u4e94\uff0c\u7f8e\u56fd\u98df\u54c1\u836f\u54c1\u76d1\u7ba1\u5c40\uff08FDA\uff09\u6279\u51c6\u8bfa\u534eNovartis\uff08NYSE: NVS\uff09\u65d7\u4e0b\u7684\u795e\u7ecf\u808c\u8089\u75be\u75c5\u57fa\u56e0\u7597\u6cd5Zolgensma\u5e76\u5c06\u5728\u7f8e\u56fd\u4e0a\u5e02\u9500\u552e\uff0c\u8fd9\u4e5f\u662f\u7b2c2\u4e2a\u83b7\u5f97FDA\u6279\u51c6\u7684\u57fa\u56e0\u7597\u6cd5\uff08\u76f8\u5173\u65b0\u95fb\uff1aFDA\u6279\u51c6\u8bfa\u534eSMA\u57fa\u56e0\u7597\u6cd5\uff0c\u552e\u4ef7\u4e3a$212.5\u4e07<\/a>\uff09\u3002Zolgensma\u83b7\u51c6\u6cbb\u7597\u8fd0\u52a8\u795e\u7ecf\u5143\u751f\u5b58\u57fa\u56e01\uff08SMN1\uff09\u53cc\u7b49\u4f4d\u57fa\u56e0\u7a81\u53d8\uff0c\u5e74\u9f84\u57282\u5c81\u4ee5\u4e0b\u7684\u810a\u9ad3\u6027\u808c\u840e\u7f29\u75c7\uff08SMA\uff09\u5c0f\u513f\u60a3\u8005\u3002FDA\u4e34\u65f6\u5c40\u957fNed Sharpless\u8868\u793a\uff0c\u4eca\u5929\u8fd9\u4e00\u6279\u6587\u6807\u5fd7\u7740\u57fa\u56e0\u548c\u7ec6\u80de\u7597\u6cd5\u7684\u53e6\u4e00\u4e2a\u91cc\u7a0b\u7891\u3002<\/p>\n

\u8be5\u7597\u6cd5\u4e00\u6b21\u6027\u9759\u8109\u6ce8\u5c04\u7684\u4ef7\u683c\u4e3a$212.5\u4e07\uff0c\u521b\u5386\u53f2\u4e4b\u6700\uff0c5\u5e74\u5e73\u644a\u540e\u6bcf\u5e74\u7684\u8d39\u7528\u4e3a$42.5\u4e07\u3002\u4e4d\u770b\u4e4b\u4e0b\uff0c\u8fd9\u4e00\u5b9a\u4ef7\u5b9e\u5728\u9ad8\u5f97\u79bb\u8c31\uff0c\u4f46\u76f8\u8f83\u5f53\u524dSMA\u60a3\u8005\u7684\u6cbb\u7597\u8d39\u7528\uff0cZolgensma\u7684\u6210\u672c\u4f18\u52bf\u4ecd\u7136\u975e\u5e38\u660e\u663e\uff08\u76f8\u5173\u65b0\u95fb\uff1a\u8bfa\u534e\u57fa\u56e0\u7597\u6cd5Zolgensma\u7684\u6210\u672c\u6548\u76ca\u60ca\u4eba<\/a>\uff09\u3002\u6bd4\u5982\u8bf4\uff0c\u6b64\u524d\u83b7\u6279\u7684SMA\u65b0\u836fSpinraza\u7b2c\u4e00\u5e74\u7684\u6210\u672c\u4e3a$75\u4e07\uff0c\u540e\u7eed\u6bcf\u5e74\u7684\u8d39\u7528\u4ecd\u9ad8\u8fbe$37.5\u4e07\u3002<\/p>\n

SMA\u662f\u4e00\u79cd\u7f55\u89c1\u7684\u9057\u4f20\u75c5\uff0c\u7531\u4e8eSMN1\u57fa\u56e0\u7684\u7f3a\u5931\u6216\u7a81\u53d8\uff0c\u60a3\u8005\u65e0\u6cd5\u751f\u6210\u5bf9\u4e8e\u8fd0\u52a8\u795e\u7ecf\u5143\u5b58\u6d3b\u81f3\u5173\u91cd\u8981\u7684\u8fd0\u52a8\u795e\u7ecf\u5143\u751f\u5b58\u86cb\u767d\uff08SMN\uff09\u3002\u7f79\u60a3\u6b64\u75c5\u7684\u5a74\u513f\u5c06\u5931\u53bb\u72ec\u7acb\u8fdb\u98df\u548c\u547c\u5438\u7684\u80fd\u529b\uff0c\u6bcf10\u4e2a\u8bca\u65ad\u4e3a\u4e00\u578bSMA\u7684\u5a74\u513f\u5f53\u4e2d\uff0c\u5927\u7ea69\u4e2a\u4f1a\u5728\u4e24\u5468\u5c81\u4e4b\u524d\u53bb\u4e16\uff0c\u6216\u8005\u9700\u8981\u957f\u671f\u7684\u547c\u5438\u673a\u652f\u6301\u3002\u7f8e\u56fd\u6bcf\u5e74\u6709\u8d85\u8fc7450\u540dSMA\u5a74\u513f\u51fa\u751f\uff0c\u52a0\u4e0a\u6b27\u6d32\uff0c\u8fd9\u4e00\u6570\u5b57\u7ea6\u4e3a1,000\u4eba\u3002\u65e9\u671f\u8bca\u65ad\u548c\u5e72\u9884\u662f\u5bf9\u6297\u8fd9\u79cd\u75be\u75c5\u7684\u5173\u952e\uff0c\u56e0\u6b64\u65b0\u751f\u513f\u7684SMA\u7b5b\u67e5\u53d8\u5f97\u8d8a\u6765\u8d8a\u666e\u904d\u3002<\/p>\n

\u6700\u65e9\uff0cSMA\u7684\u6cbb\u7597\u65b9\u6848\u4e3b\u8981\u662f\u652f\u6301\u7597\u6cd5\u3002\u4e0d\u8fc7\u57282016\u5e74\uff0cBiogen Inc\uff08NASDAQ: BIIB\uff09\u548cIonis Pharmaceuticals Inc\uff08NASDAQ: IONS\uff09\u7684Spinraza\u83b7\u5f97FDA\u7684\u6279\u51c6\uff0c\u6210\u4e3a\u5168\u7403\u9996\u4e2a\u810a\u808c\u840e\u7f29\u75c7\u6cbb\u7597\u836f\u7269\uff08\u76f8\u5173\u65b0\u95fb\uff1a\u767e\u5065Spinraza\u5728\u4e2d\u56fd\u4e0a\u5e02\u9500\u552e<\/a>\uff09\u3002\u6b64\u540e\uff0c\u8be5\u836f\u7269\u6210\u4e3a\u4e86SMA\u60a3\u8005\u7684\u5e38\u7528\u836f\u7269\uff0c\u4f46\u4ecd\u5b58\u5728\u8bf8\u591a\u7f3a\u9677\u3002<\/p>\n

Spinraza\u662f\u4e00\u79cd\u53cd\u4e49\u5be1\u6838\u82f7\u9178\uff08ASO\uff09\uff0c\u65e8\u5728\u4fee\u590d\u540e\u5907\u57fa\u56e0SMN2\u7684\u526a\u63a5\u9519\u8bef\uff0c\u4ece\u800c\u6062\u590d\u66f4\u52a0\u5b8c\u6574\u7684SMN\u86cb\u767d\u751f\u6210\u3002\u7136\u800c\uff0c\u8be5\u7597\u6cd5\u9700\u8981\u5c06\u836f\u7269\u6ce8\u5c04\u8fdb\u810a\u9ad3\u5468\u56f4\u7684\u6db2\u4f53\u5f53\u4e2d\uff0c\u5e76\u4e14\u7ed9\u836f\u9996\u5e74\u9700\u6ce8\u5c044\u6b21\uff0c\u6b64\u540e\u6bcf\u5e74\u6ce8\u5c043\u6b21\u3002\u6b64\u5916\uff0c\u5728\u4e34\u5e8a\u8bd5\u9a8c\u5f53\u4e2d\uff0c\u4ec540%\u7684\u60a3\u8005\u5b9e\u73b0\u4e86\u8fd0\u52a8\u91cc\u7a0b\u7891\u7684\u6539\u5584\u3002<\/p>\n

\u76f8\u6bd4\u4e4b\u4e0b\uff0cZolgensma\u7684\u4f5c\u7528\u673a\u5236\u5b8c\u5168\u4e0d\u540c\u4e14\u66f4\u4e3a\u6709\u6548\u3002<\/p>\n

\u4f5c\u4e3a\u4e00\u79cd\u57fa\u56e0\u7597\u6cd5\uff0cZolgensma\u4f7f\u7528AAV9\u75c5\u6bd2\u8f7d\u4f53\u5c06\u5065\u5eb7\u7684\u4eba\u7c7bSMN\u57fa\u56e0\u9012\u9001\u81f3\u60a3\u8005\u4f53\u5185\uff0c\u8ba9\u60a3\u8005\u53ef\u4ee5\u751f\u6210\u6b63\u5e38\u7684SMN\u86cb\u767d\uff0c\u4ece\u800c\u6539\u5584\u8fd0\u52a8\u795e\u7ecf\u5143\u529f\u80fd\u548c\u751f\u5b58\u3002\u6b64\u5916\uff0c\u548cSpinraza\u4e0d\u540c\u7684\u662f\uff0c\u60a3\u8005\u53ea\u9700\u4e00\u6b21\u7ed9\u836f\u3002\u4e34\u5e8a\u8bd5\u9a8c\u7ed3\u679c\u663e\u793a\uff0cZolgensma\u7684\u6cbb\u7597\u6548\u679c\u975e\u5e38\u51fa\u8272\u3002\u6cbb\u75971\u4e2a\u6708\u4ee5\u5185\uff0c\u60a3\u8005\u7684\u8fd0\u52a8\u529f\u80fd\u83b7\u5f97\u5feb\u901f\u6539\u5584\uff0c\u5e76\u4e14\u6cbb\u7597\u5229\u76ca\u6301\u7eed\u4e24\u5e74\u4ee5\u4e0a\u3002<\/p>\n

\u622a\u81f32019\u5e743\u6708\uff0cStr1ve\u4e34\u5e8a\u8bd5\u9a8c\u62db\u52df\u768421\u540d\u60a3\u8005\u5f53\u4e2d\uff0c19\u4eba\u4ecd\u7136\u5b58\u6d3b\u4e14\u65e0\u9700\u957f\u671f\u7684\u547c\u5438\u673a\u901a\u6c14\u652f\u6301\uff0c13\u4eba\u7684\u5b58\u6d3b\u65f6\u95f4\u5df2\u8fbe14\u4e2a\u6708\u3002\u6b64\u5916\uff0c10\u540d\u60a3\u8005\u80fd\u591f\u5728\u5e73\u574712.1\u4e2a\u6708\u7684\u65f6\u5019\u72ec\u7acb\u5750\u7acb\u81f3\u5c1130\u79d2\u3002\u8bfa\u534e\u7684\u7814\u7a76\u8fd8\u663e\u793a\uff0c\u7b26\u5408Str1ve\u62db\u52df\u6807\u51c6\u7684\u60a3\u8005\uff0c\u9884\u671f\u751f\u5b58\u671f\u8d85\u8fc714\u4e2a\u6708\u7684\u6bd4\u4f8b\u4ec5\u670925%\u5de6\u53f3\u3002<\/p>\n

\u53e6\u4e00\u9879\u540d\u4e3aStart\u7684\u8bd5\u9a8c\u62db\u52df\u4e8615\u540d\u60a3\u8005\uff0c\u5176\u4e2d12\u4eba\u9009\u5165\u9ad8\u5242\u91cf\u7ec4\u3002\u7ed3\u679c\u663e\u793a\uff0c\u6cbb\u7597\u7b2c24\u4e2a\u6708\uff0c\u9ad8\u5242\u91cf\u7ec4\u7684\u6240\u6709\u60a3\u8005\u5747\u5b58\u6d3b\u4e14\u65e0\u9700\u957f\u671f\u901a\u6c14\uff0c\u5e76\u4e149\u540d\u60a3\u8005\u80fd\u591f\u72ec\u7acb\u5750\u7acb\u81f3\u5c1130\u79d2\u3002<\/p>\n","protected":false},"excerpt":{"rendered":"

\u76ee\u524d\u5168\u7403\u6709\u8d85\u8fc76,000\u79cd\u9057\u4f20\u75c5\uff0c\u5e76\u4e14\u76ee\u524d\u591a\u6570\u90fd\u8fd8\u6ca1\u6709\u83b7\u6279\u7684\u6cbb\u7597\u624b\u6bb5\u3002\u73b0\u5b9e\u8ba9\u4eba\u6cae\u4e27\uff0c\u4f46\u5e0c\u671b\u4ecd\u5728\u3002\u4f5c\u4e3a\u9057\u4f20\u75c5\u7684\u6839 […]<\/p>\n","protected":false},"author":27,"featured_media":266733,"comment_status":"closed","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"footnotes":"","_links_to":"","_links_to_target":""},"categories":[430],"tags":[455,747,925,459],"class_list":["post-266732","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-trends-strategies-zh-hans","tag-455","tag-747","tag-925","tag-459","sectors-life-science-zh-hans","locations-us-zh-hans","exchange-nasdaq-zh-hans","exchange-nyse-zh-hans"],"acf":[],"_links":{"self":[{"href":"https:\/\/nai500.com\/zh-hans\/wp-json\/wp\/v2\/posts\/266732","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/nai500.com\/zh-hans\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/nai500.com\/zh-hans\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/nai500.com\/zh-hans\/wp-json\/wp\/v2\/users\/27"}],"replies":[{"embeddable":true,"href":"https:\/\/nai500.com\/zh-hans\/wp-json\/wp\/v2\/comments?post=266732"}],"version-history":[{"count":0,"href":"https:\/\/nai500.com\/zh-hans\/wp-json\/wp\/v2\/posts\/266732\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/nai500.com\/zh-hans\/wp-json\/wp\/v2\/media\/266733"}],"wp:attachment":[{"href":"https:\/\/nai500.com\/zh-hans\/wp-json\/wp\/v2\/media?parent=266732"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/nai500.com\/zh-hans\/wp-json\/wp\/v2\/categories?post=266732"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/nai500.com\/zh-hans\/wp-json\/wp\/v2\/tags?post=266732"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}