{"id":266949,"date":"2019-05-28T00:17:55","date_gmt":"2019-05-28T07:17:55","guid":{"rendered":"https:\/\/nai500.com\/?p=266949"},"modified":"2019-05-28T00:17:55","modified_gmt":"2019-05-28T07:17:55","slug":"%e5%83%b9%e5%80%bc%e6%8a%95%e8%b3%87%ef%bc%9a%e8%ad%b7%e5%9f%8e%e6%b2%b3%e6%9c%80%e6%b7%b1%e6%9c%80%e5%af%ac%e7%9a%843%e5%ae%b6%e5%a4%a7%e5%9e%8b%e7%94%9f%e7%89%a9%e7%a7%91%e6%8a%80%e5%85%ac%e5%8f%b8","status":"publish","type":"post","link":"https:\/\/nai500.com\/zh-hant\/blog\/2019\/05\/%e5%83%b9%e5%80%bc%e6%8a%95%e8%b3%87%ef%bc%9a%e8%ad%b7%e5%9f%8e%e6%b2%b3%e6%9c%80%e6%b7%b1%e6%9c%80%e5%af%ac%e7%9a%843%e5%ae%b6%e5%a4%a7%e5%9e%8b%e7%94%9f%e7%89%a9%e7%a7%91%e6%8a%80%e5%85%ac%e5%8f%b8\/","title":{"rendered":"\u50f9\u503c\u6295\u8cc7\uff1a\u8b77\u57ce\u6cb3\u6700\u6df1\u6700\u5bec\u76843\u5bb6\u5927\u578b\u751f\u7269\u79d1\u6280\u516c\u53f8"},"content":{"rendered":"

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Alexion Pharmaceuticals Inc\uff08NASDAQ: ALXN\uff09<\/h4>\n

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2007\u5e74\uff0c\u7f8e\u570b\u98df\u54c1\u85e5\u54c1\u76e3\u7ba1\u5c40\uff08FDA\uff09\u9996\u6b21\u6279\u51c6Soliris\u6cbb\u7642\u9663\u767c\u6027\u7761\u7720\u6027\u8840\u7d05\u86cb\u767d\u5c3f\u75c7\uff08PNH\uff09\u3002\u5230\u76ee\u524d\u7232\u6b62\uff0c\u9019\u4ecd\u662f\u9019\u4e00\u6975\u5ea6\u7f55\u898b\u907a\u50b3\u6027\u8840\u6db2\u75be\u75c5\u7684\u552f\u4e00\u7372\u6279\u85e5\u7269\u3002\u5728\u6b64\u5f8c2011\u5e74\u548c2017\u5e74\uff0cSoliris\u5169\u7a2e\u65b0\u7684\u9069\u61c9\u75c7\u2014\u2014\u975e\u5178\u578b\u6eb6\u8840\u6027\u5c3f\u6bd2\u7d9c\u5408\u5f81\uff08aHUS\uff09\u4ee5\u53ca\u5168\u8eab\u578b\u91cd\u75c7\u808c\u7121\u529b\uff08gMG\uff09\u2014\u2014\u4e5f\u5148\u5f8c\u7372\u6279\u3002<\/p>\n

\u66f4\u7232\u53ef\u559c\u7684\u662f\uff0cAlexion\u53c8\u63a8\u51fa\u4e86\u65b0\u7684\u7523\u54c1Ultomiris\u3002\u8a72\u85e5\u7269\u7684PNH\u9069\u61c9\u75c7\u5df2\u57282018\u5e7412\u6708\u7372\u6279\uff0c\u65e9\u4e8e\u9810\u671f\u3002\u6b64\u5916\uff0caHUS\u9069\u61c9\u75c7\u6709\u671b\u5728\u4eca\u5e74\u7372\u6279\u3002\u5e02\u5834\u7814\u7a76\u516c\u53f8EvaluatePharma\u5c07Ultomiris\u5217\u72322019\u5e74\u7684\u6700\u5927\u65b0\u85e5\uff0c\u5e77\u9810\u8a08\u92b7\u552e\u984d\u52302024\u5e74\u5c07\u9ad8\u9054$35\u5104\u3002<\/p>\n

Biomarin Pharmaceutical Inc\uff08NASDAQ: BMRN\uff09<\/h4>\n

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\u8a72\u516c\u53f8\u6700\u66a2\u92b7\u7684\u7523\u54c1Vimizim\u53bb\u5e74\u5275\u6536\u5360\u5176\u7e3d\u6536\u5165\uff08$14.7\u5104\uff09\u7684\u8fd1\u4e09\u5206\u4e4b\u4e00\uff0c\u8a72\u85e5\u7269\u662f\u9996\u500b\u4e5f\u662f\u552f\u4e00\u7372\u6279\u7684\u7f55\u898b\u907a\u50b3\u75c5Morquio A\u9176\u66ff\u4ee3\u7642\u6cd5\u3002<\/p>\n

\u82ef\u4e19\u916e\u5c3f\u75c7\uff08PKU\uff09\u6cbb\u7642\u85e5\u7269Kuvan\u5360\u516c\u53f8\u7e3d\u6536\u5165\u7684\u6bd4\u91cd\u7d04\u723229%\uff0c\u7a0d\u6b21\u4e8eVimizim\u3002\u76ee\u524d\u8a72\u4ee3\u8b1d\u75c5\u7684\u7372\u6279\u8655\u65b9\u85e5\u50c5\u6709\u5169\u7a2e\u3002\u6392\u540d\u7b2c3\u7684Naglazyme\u662f\u9996\u500b\uff0c\u5e77\u4e14\u5230\u76ee\u524d\u7232\u6b62\u552f\u4e00\u7372\u5f97FDA\u6279\u51c6\u7684\u6cbb\u7642\u7f55\u898b\u907a\u50b3\u75c5\u2014\u2014VI\u578b\u9ecf\u591a\u7cd6\u75c5\uff08MPS VI\uff09\u2014\u2014\u7684\u9176\u66ff\u4ee3\u7642\u6cd5\u3002<\/p>\n

\u6b64\u5916\uff0cBioMarin\u7684\u5176\u4ed6\u85e5\u7269\u4e5f\u64c1\u6709\u8b77\u57ce\u6cb3\u3002Aldurazyme\u662f\u552f\u4e00\u7372\u6279\u7684MPS I\u6cbb\u7642\u624b\u6bb5\uff0cBrineura\u662f\u552f\u4e00\u7372\u6279\u7684CLN2\u9176\u66ff\u4ee3\u7642\u6cd5\u3002\u53bb\u5e74\uff0cPalynziq\u7372\u6279\u6cbb\u7642PKU\u3002<\/p>\n

\u798f\u6cf0\u88fd\u85e5Vertex Pharmaceuticals\uff08NASDAQ: VRTX\uff09<\/h4>\n

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\u6b64\u5916\uff0c\u798f\u6cf0\u88fd\u85e5\u5728\u4e0b\u4e00\u4ee3CF\u6cbb\u7642\uff08\u4e09\u806f\u7642\u6cd5\uff09\u9818\u57df\u4e5f\u5148\u4eba\u4e00\u6b65\u3002\u8a72\u751f\u7269\u79d1\u6280\u516c\u53f8\u9810\u8a082019\u5e74\u4e09\u5b63\u5ea6\u5411FDA\u63d0\u4ea4\u9996\u500bCF\u4e09\u806f\u7642\u6cd5\u4e0a\u5e02\u7533\u8acb\uff0c\u5e74\u5e95\u63d0\u4ea4\u6b50\u6d32\u7533\u8acb\u3002\u827e\u4f2f\u7dadAbbVie\uff08NYSE: ABBV\uff09\u7684CF\u4e09\u806f\u7642\u6cd5\u4e5f\u6709\u671b\u53d6\u5f97\u6210\u529f\uff0c\u4f46\u9032\u5ea6\u9060\u9060\u843d\u5f8c\u4e8e\u798f\u6cf0\u88fd\u85e5\u3002\u6b64\u5916\uff0c\u5728\u53bb\u5e74\u7684\u4e8c\u671f\u81e8\u5e8a\u7814\u7a76\u7576\u4e2d\uff0c\u827e\u4f2f\u7dad\u4e09\u806f\u7642\u6cd5\u7576\u4e2d\u7684\u4e00\u6b3e\u95dc\u9375\u85e5\u7269\u5e77\u672a\u53d6\u5f97\u9810\u671f\u7684\u7d50\u679c\u3002<\/p>\n

\u8207\u6b64\u540c\u6642\uff0c\u798f\u6cf0\u88fd\u85e5\u9084\u6709\u671b\u9032\u8ecd\u5176\u4ed6\u9818\u57df\u5e77\u638c\u63e1\u65b0\u7684\u7e31\u6df1\u9632\u79a6\u3002\u516c\u53f8\u806f\u5408CRISPR Therapeutics AG\uff08NASDAQ: CRSP\uff09\u958b\u767c\u91dd\u5c0d\u7f55\u898b\u8840\u6db2\u75c5\u2014\u2014\u03b2-\u5730\u4e2d\u6d77\u8ca7\u8840\u4ee5\u53ca\u942e\u72c0\u7d30\u80de\u75c5\u2014\u2014\u7684\u57fa\u56e0\u7de8\u8f2f\u7642\u6cd5\u3002\u6b64\u5916\uff0c\u516c\u53f8\u6b63\u5728\u8a55\u4f30\u4e00\u6b3e\u5019\u9078\u85e5\uff08\u521d\u671f\u81e8\u5e8a\u7814\u7a76\uff09\u6cbb\u7642\u7f55\u898b\u907a\u50b3\u75c5\u2014\u2014\u03b11-\u6297\u80f0\u86cb\u767d\u9176\uff08AAT\uff09\u7f3a\u4e4f\u75c7\u2014\u2014\u7684\u6548\u679c\u3002<\/p>\n

\u4f30\u503c\u6c34\u5e73<\/h4>\n

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