{"id":112729,"date":"2019-06-11T11:53:08","date_gmt":"2019-06-11T03:52:18","guid":{"rendered":"http:\/\/www.05921.net\/index.php\/112729\/"},"modified":"2019-06-11T11:52:21","modified_gmt":"2019-06-11T03:52:21","slug":"2b92376e92","status":"publish","type":"post","link":"https:\/\/www.05921.net\/index.php\/112729\/","title":{"rendered":"1400 \u4e07\u4e00\u9488\uff0c\u8fd9\u662f\u4e16\u754c\u4e0a\u5df2\u77e5\u6700\u8d35\u7684\u836f\u7269\u7597\u6cd5"},"content":{"rendered":"
\n

\u8fd9\u662f\u5173\u4e8e\u51e0\u4e2a\u7edd\u671b\u7684\u5bb6\u5ead\u5f97\u5230\u300c\u91cd\u751f\u300d\u7684\u6545\u4e8b\u3002<\/span><\/p>\n

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Matteo Almeida \u8fd8\u5728\u5988\u5988Nicole Almeida \u809a\u5b50\u91cc\u7684\u65f6\u5019\uff0c\u5c31\u88ab\u533b\u751f\u65ad\u8a00\uff1a\u8fd9\u4e2a\u5b69\u5b50\u53ef\u80fd\u4e0d\u4f1a\u6d3b\u8fc7\u4ed6\u7684\u7b2c\u4e00\u4e2a\u751f\u65e5\u3002<\/span><\/p>\n

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\u5f53\u533b\u751f\u5411 Nicole \u89e3\u91ca Matteo \u7684\u75c5\u60c5\u7684\u65f6\u5019\uff0c\u5979\u5c31\u77e5\u9053\uff0c\u8fd9\u5e76\u4e0d\u662f\u5371\u8a00\u8038\u542c\uff0c\u5979\u7684\u5b69\u5b50\u7f79\u60a3\u7684\u662f\u4e00\u4e2a\u300c\u7edd\u75c7\u300d\uff0c\u800c\u8fd9\u4e2a\u7edd\u75c7\uff0c\u662f\u4e24\u5c81\u4ee5\u4e0b\u5b69\u5b50\u6b7b\u4ea1\u7684\u91cd\u8981\u56e0\u7d20\u4e4b\u4e00\u2014\u2014\u810a\u9ad3\u6027\u808c\u840e\u7f29\u75c7<\/span>\uff08Spinal Muscular Atrophy\uff0c\u7b80\u79f0 SMA\uff09<\/span>\u3002<\/span><\/p>\n


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\"1400<\/p>\n

\u56fe\u6e90\uff1a nbcnews<\/span><\/p>\n


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\u810a\u9ad3\u6027\u808c\u840e\u7f29\u75c7<\/span>\uff08SMA\uff09<\/span>\u662f\u4e00\u7ec4\u9057\u4f20\u6027\u75be\u75c5\uff0c\u6700\u5e38\u89c1\u7684 SMA \u662f\u7531\u4e0e\u67d3\u8272\u4f53 5q \u4e0a\u5b58\u6d3b\u8fd0\u52a8\u795e\u7ecf\u5143 1 \u57fa\u56e0<\/span>\uff08SMN1\uff09<\/span>\u7684\u4e24\u4e2a\u62f7\u8d1d\u4e2d\u7684\u7f3a\u9677\u5f15\u8d77\u7684\u3002<\/span><\/p>\n


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\u8be5\u57fa\u56e0\u4ea7\u751f\u5b58\u6d3b\u8fd0\u52a8\u795e\u7ecf\u5143<\/span>\uff08SMN\uff09<\/span>\u86cb\u767d\uff0c\u5176\u7ef4\u6301\u8fd0\u52a8\u795e\u7ecf\u5143\u7684\u5065\u5eb7\u548c\u6b63\u5e38\u529f\u80fd\u3002<\/span><\/p>\n

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\u7531 SMN1 \u57fa\u56e0\u7a81\u53d8\u5f15\u8d77\u7684 SMA \u901a\u5e38\u6839\u636e\u53d1\u75c5\u5e74\u9f84\u548c\u4e25\u91cd\u7a0b\u5ea6\u5206\u4e3a\u51e0\u79cd\u4e9a\u578b\uff1b\u5a74\u513f\u53d1\u75c5\u7684 SMA \u662f\u6700\u4e25\u91cd\u548c\u6700\u5e38\u89c1\u7684\u4e9a\u578b\u3002<\/span><\/p>\n

 <\/p>\n

\u7f79\u60a3 SMA \u7684\u5b69\u5b50\uff0c\u8fd0\u52a8\u795e\u7ecf\u5143\u2014\u8111\u5e72\u548c\u810a\u9ad3\u4e2d\u7684\u795e\u7ecf\u7ec6\u80de\u4f1a\u88ab\u9010\u6b65\u7834\u574f\uff0c\u8fd0\u52a8\u795e\u7ecf\u5143\u63a7\u5236\u624b\u81c2\uff0c\u817f\uff0c\u80f8\u90e8\uff0c\u9762\u90e8\uff0c\u5589\u5499\u548c\u820c\u5934\u7684\u8fd0\u52a8\u3002<\/span><\/p>\n


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\u56e0\u6b64\uff0c\u60a3\u513f\u5c06\u4f1a\u6162\u6162\u5931\u53bb\u8bf4\u8bdd\uff0c\u8d70\u8def\u7684\u80fd\u529b\uff0c\u5230\u6700\u540e\uff0c\u4ed6\u4eec\u751a\u81f3\u65e0\u6cd5\u547c\u5438\u548c\u541e\u54bd\uff0c\u5e76\u6700\u7ec8\u4f1a\u88ab\u75be\u75c5\u593a\u53bb\u6027\u547d\u3002<\/span><\/p>\n

 <\/p>\n

Nicole \u4e0d\u613f\u610f\u653e\u5f03\u8fd9\u4e2a\u5c0f\u751f\u547d\uff0c\u5979\u62b1\u7740\u5bf9\u672a\u77e5\u5de8\u5927\u7684\u6050\u60e7\uff0c\u5f00\u59cb\u81ea\u5df1\u7740\u624b\u5f00\u59cb\u7814\u7a76 SMA\uff0c\u5bfb\u627e\u53ef\u80fd\u7684\u6cbb\u7597\u65b9\u6cd5\u3002<\/span><\/p>\n


<\/p>\n

\u7687\u5929\u4e0d\u8d1f\u6709\u5fc3\u4eba\uff0c\u901a\u8fc7\u4e00\u4e9b\u5fd7\u613f\u7f51\u7ad9\uff0c\u5979\u83b7\u77e5\u6709\u4e24\u9879\u6b63\u5728\u8fdb\u884c\u7684\u4e34\u5e8a\u8bd5\u9a8c\uff0c\u79d1\u5b66\u5bb6\u6b63\u5728\u5bfb\u627e\u53ef\u4ee5\u53c2\u4e0e\u6d4b\u8bd5\u7684\u5b9e\u9a8c\u5bf9\u8c61\u3002<\/span><\/p>\n

 <\/p>\n

\u800c Matteo \u7684\u60c5\u51b5\uff0c\u6b63\u7b26\u5408\u5165\u7ec4\u5b9e\u9a8c\u7684\u6761\u4ef6\u3002<\/span><\/p>\n


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\u5728\u5b69\u5b50\u51fa\u751f\u7684\u7b2c 27 \u65e5\uff0cNicole \u5e26\u4ed6\u63a5\u53d7\u4e86\u8fd9\u6b21\u57fa\u56e0\u6cbb\u7597\u3002<\/span><\/p>\n


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\u7ed3\u679c\u8fd1\u4e4e\u300c\u795e\u8ff9\u300d\uff0c\u76f4\u5230\u73b0\u5728\uff0c\u5728\u5c06\u8fd1\u56db\u5e74\u540e\uff0c\u8fd9\u4e2a\u5c0f\u7537\u5b69\u5065\u5eb7\u5f3a\u58ee\uff0c\u6ca1\u6709\u4efb\u4f55 SMA \u8ff9\u8c61\u3002<\/span><\/p>\n


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\"1400Matteo Almeida \u5e86\u795d\u4ed6\u7684\u4e09\u5c81\u751f\u65e5<\/span><\/p>\n

\u56fe\u7247\u6765\u6e90\uff1awww.nbcnews.com<\/span><\/p>\n


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\u8fd9\u6837\u7684\u6545\u4e8b\u7edd\u5bf9\u4e0d\u662f\u4ec5\u6709\u4e00\u4f8b\u3002<\/span><\/p>\n

 <\/span><\/p>\n

\u6839\u636e\u5fd7\u613f\u7ec4\u7ec7\u300cSMA CURE\u300d\u7684\u7edf\u8ba1\u53d1\u73b0\uff0c\u5927\u7ea6\u5728 11,000 \u540d\u5a74\u513f\u4e2d\u4f1a\u6709 1 \u4e2a\u5a74\u513f\u88ab\u8bca\u65ad\u4e3a SMA\uff0c\u800c\u57fa\u56e0\u643a\u5e26\u6570\u66f4\u662f\u5e9e\u5927\uff0c\u4ed6\u4eec\u7684\u7edf\u8ba1\u8ba4\u4e3a\uff0c\u6bcf 50 \u540d\u7f8e\u56fd\u4eba\u4e2d\u81f3\u5c11\u6709 1 \u540d\u662f SMA \u57fa\u56e0\u7684\u643a\u5e26\u8005\u3002<\/span><\/p>\n

 <\/span><\/p>\n

\u4e09\u5c81\u7684 Donovan Weisgarber \u662f\u53e6\u5916\u4e00\u4e2a\u6545\u4e8b\u7684\u4e3b\u89d2\u3002<\/span><\/p>\n

 <\/p>\n

\u5b69\u5b50\u521a\u51fa\u751f\u7684\u65f6\u5019\uff0c\u770b\u8d77\u6765\u6b63\u5e38\u5065\u5eb7\uff0c\u548c\u5176\u4ed6\u65b0\u751f\u513f\u5e76\u6ca1\u6709\u4ec0\u4e48\u4e0d\u540c\uff0c\u4f46\u4ec5\u4ec5\u662f\u51e0\u5468\u4e4b\u5185\uff0c\u4ed6\u5c31\u51fa\u73b0\u4e86\u4e00\u4e9b\u8db3\u4ee5\u8ba9\u7236\u6bcd\u6ce8\u610f\u5230\u7684\u5f02\u5e38\u4e86\uff0c\u4ed6\u9996\u5148\u8868\u73b0\u5f97\u975e\u5e38\u70e6\u8e81\uff0c\u6709\u5404\u79cd\u5947\u602a\u7684\u8815\u52a8\uff0c\u4f46\u968f\u4e4b\u800c\u6765\u7684\uff0c\u5374\u662f\u8d8a\u6765\u8d8a\u5730\u865a\u5f31\u3002<\/span><\/p>\n

 <\/p>\n

\u548c Matteo \u4e00\u6837\uff0cDonovan \u88ab\u8ba4\u4e3a\u4e0d\u4f1a\u6d3b\u8fc7\u4ed6\u7684\u7b2c\u4e8c\u4e2a\u751f\u65e5\u3002<\/span><\/p>\n


<\/p>\n

\u4f46\u8fd9\u4f3c\u4e4e\u662f\u547d\u8fd0\u7ed9\u4ed6\u4eec\u7684\u4e00\u4e2a\u5c0f\u6311\u6218\uff0c\u4ed6\u4eec\u8d62\u4e86\u3002<\/span><\/p>\n


<\/p>\n

\u4e09\u5e74\u540e\u7684\u4eca\u5929\uff0cDonovan \u4f9d\u7136\u9700\u8981\u4e00\u4e2a\u8f6e\u6905\u6765\u8f85\u52a9\u884c\u8d70\uff0c\u996e\u98df\u4e5f\u9700\u8981\u9f3b\u9972\u7ba1\u534f\u52a9\uff0c\u4f46\u4ed6\u4f9d\u7136\u6d3b\u7740\u3002<\/span><\/p>\n


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\u53e6\u4e00\u4e2a\u300c\u5e78\u8fd0\u300d\u7684\u5c0f\u5b69 Malachi Anderson\uff0c\u5df2\u7ecf\u5c06\u8fd1\u56db\u5c81\u4e86\u4ed6\u56db\u4e2a\u6708\u5927\u7684\u65f6\u5019\u5c31\u88ab\u8bca\u65ad\u51fa\u60a3\u6709\u7684\u810a\u9ad3\u6027\u808c\u840e\u7f29\uff0c\u5728\u4ed6 12 \u4e2a\u6708\u7684\u65f6\u5019\uff0c\u63a5\u53d7\u4e86\u65b0\u7684\u57fa\u56e0\u7597\u6cd5\u6cbb\u7597\u3002<\/span><\/p>\n


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\"1400<\/p>\n

\u56fe\u7247\u6765\u6e90\uff1aThe New York Times<\/span><\/p>\n


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\u8fd9\u662f\u56e0\u4e3a\uff0c\u4ed6\u4eec\u540c\u6837\u5e78\u8fd0\u5730\u9047\u5230\u4e86 Zolgensma\uff0c\u4eca\u5929\u7684\u5929\u4ef7\u4e3b\u89d2\u3002<\/span><\/p>\n

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<\/p>\n

\u5929\u4ef7\u4e3b\u89d2\u7684\u524d\u4e16\u4eca\u751f<\/span><\/strong><\/span><\/p>\n


<\/p>\n

Zolgensma \u9002\u7528\u4e8e\u6cbb\u7597 SMA \u5c0f\u4e8e 2 \u5c81\u7684\u513f\u7ae5\u3002 <\/span><\/p>\n

 <\/span><\/p>\n

\u836f\u5382\u81ea\u7136\u662f\u9700\u8981\u5411\u516c\u4f17\u8bc1\u660e\u8fd9\u4e2a\u836f\u7269\u7684\u5b89\u5168\u6027\u548c\u6709\u6548\u6027\u7684\u3002<\/span><\/p>\n


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\u4e34\u5e8a\u8bd5\u9a8c\u662f\u5728 36 \u4e2a 2 \u5468\u5230 8 \u4e2a\u6708\u5927\u7684\u5a74\u513f\u4e2d\u6d4b\u8bd5\u7684\uff0c\u6240\u6709\u5a74\u513f\u90fd\u88ab\u786e\u8bca\u4e3a SMA\u3002<\/span><\/p>\n

 <\/span><\/p>\n

\u6709\u6548\u6027\u7684\u4e3b\u8981\u8bc1\u636e\u662f\u57fa\u4e8e\u6b63\u5728\u8fdb\u884c\u7684\u4e34\u5e8a\u8bd5\u9a8c\u4e2d\u4f7f\u7528\u8fc7\u836f\u7269\u6cbb\u7597\u7684 21 \u540d\u60a3\u8005\u7684\u7ed3\u679c\u3002<\/span><\/p>\n


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\u5728\u8fd9\u9879\u8bd5\u9a8c\u4e2d\u7684 19 \u540d\u60a3\u8005\uff0c\u5e74\u9f84\u8303\u56f4\u4e3a 9.4 \u81f3 18.5 \u4e2a\u6708\uff0c\u8fd9 19 \u540d\u60a3\u8005\u4e2d\u6709 13 \u540d\u81f3\u5c11 14 \u4e2a\u6708\u5927\u3002<\/span><\/p>\n

 <\/span><\/p>\n

\u4e0e\u60a3\u6709\u5a74\u513f\u671f SMA \u7684\u60a3\u8005\u7684\u81ea\u7136\u75c5\u53f2\u76f8\u6bd4\uff0c\u7528\u836f\u6cbb\u7597\u7684\u60a3\u8005\u4e5f\u8868\u73b0\u51fa\u5176\u8fbe\u5230\u53d1\u80b2\u8fd0\u52a8\u91cc\u7a0b\u7891\u7684\u80fd\u529b\u7684\u663e\u7740\u6539\u5584<\/span>\uff08\u4f8b\u5982\uff0c\u5934\u90e8\u63a7\u5236\u548c\u65e0\u652f\u6491\u5750\u4e0b\u7684\u80fd\u529b\uff09<\/span>\u3002<\/span><\/p>\n

 <\/p>\n

\u5b83\u7684\u539f\u7406\uff0c\u662f\u7528\u75c5\u6bd2\u8f7d\u4f53\uff0c\u628a\u57fa\u56e0\u8fd0\u9001\u5230\u6709\u300c\u9519\u8bef\u300d\u7684\u57fa\u56e0\u5904\uff0c\u8f7d\u4f53\u597d\u7684\u8fd0\u52a8\u795e\u7ecf\u5143\u57fa\u56e0\uff0c\u62f7\u8d1d\u9776\u8fd0\u52a8\u795e\u7ecf\u5143\u7ec6\u80de\u4e2d\uff0c\u7136\u540e\u8ba9\u8fd9\u4e9b\u8fd0\u52a8\u795e\u7ecf\u5143\u7ec6\u80de\uff0c\u80fd\u7ee7\u7eed\u590d\u5236\uff0c\u751f\u4ea7\u51fa\u300c\u597d\u300d\u7684\u8fd0\u52a8\u795e\u7ecf\u5143\u3002<\/span><\/p>\n

 <\/p>\n

\u5bf9\u4e8e\u60a3\u8005\u6765\u8bf4\uff0c\u6574\u4e2a\u7597\u7a0b\uff0c\u53ef\u80fd\u5c31\u662f\u63a5\u53d7\u4e00\u6b21\u9759\u8109\u5185\u7ed9\u836f\uff0c\u5c31\u53ef\u4ee5\u6539\u5584\u808c\u8089\u8fd0\u52a8\u548c\u529f\u80fd\u548c\u5b58\u6d3b\u4e86\u3002<\/span><\/p>\n

 <\/p>\n

Zolgensma \u6700\u5e38\u89c1\u7684\u526f\u4f5c\u7528\u662f\u809d\u9176\u5347\u9ad8\u548c\u5455\u5410\uff0c\u60a3\u6709\u809d\u810f\u635f\u5bb3\u7684\u60a3\u8005\u4f1a\u6709\u4e25\u91cd\u809d\u635f\u4f24\u751a\u81f3\u809d\u8870\u7aed\u7684\u53ef\u80fd\u3002<\/span><\/p>\n


<\/p>\n

\u56e0\u6b64\uff0c\u5728\u5f00\u5c55\u6cbb\u7597\u524d\uff0c\u5e94\u5b8c\u6210\u4e34\u5e8a\u68c0\u67e5\u548c\u8bc4\u4f30\u809d\u529f\u80fd\u7684\u5b9e\u9a8c\u5ba4\u68c0\u67e5\uff0c\u5e76\u5e94\u5728 Zolgensma \u7ed9\u836f\u540e\u76d1\u6d4b\u60a3\u8005\u7684\u809d\u529f\u80fd\u81f3\u5c11 3 \u4e2a\u6708\u3002<\/span><\/p>\n

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<\/p>\n

\u4e0d\u662f\u7b2c\u4e00\u6b3e\uff0c\u80af\u5b9a\u662f\u6700\u8d35\u7684\u6b3e<\/span><\/strong><\/span><\/p>\n

 <\/p>\n

\u6cbb\u7597\u9057\u4f20\u6027\u75be\u75c5\u7684\u57fa\u56e0\u7597\u6cd5\u5176\u5b9e\u65e9\u5728 2017 \u5e74\u5c31\u5df2\u7ecf\u9762\u4e16\u4e86\u3002<\/span><\/p>\n

 <\/p>\n

\u8fd9\u79cd\u88ab\u79f0\u4e3a Luxturna \u7684\u57fa\u56e0\u6cbb\u7597\u836f\u7269\uff0c\u5176\u5b9e\u662f\u4e00\u79cd\u8f6c\u57fa\u56e0\u75c5\u6bd2\uff0c\u53ef\u4ee5\u5c06\u5065\u5eb7\u7684\u57fa\u56e0\u8fd0\u9001\u5230\u51fa\u751f\u65f6\u60a3\u6709\u89c6\u7f51\u819c\u8425\u517b\u4e0d\u826f\u7684\u60a3\u8005\u7684\u773c\u775b\u4e2d\u3002<\/span><\/p>\n

 <\/p>\n

\u89c6\u7f51\u819c\u8425\u517b\u4e0d\u826f\u662f\u4e00\u79cd\u7f55\u89c1\u7684\u75be\u75c5\uff0c\u7f79\u60a3\u75be\u75c5\u7684\u5b69\u5b50\uff0c\u89c6\u7f51\u819c\u7ec6\u80de\u4e5f\u53d1\u80b2\u4e0d\u826f\uff0c\u56e0\u6b64\u5b69\u5b50\u5929\u751f\u5c31\u7f3a\u4e4f\u5065\u5eb7\u89c6\u529b\u6240\u9700\u57fa\u7840\u3002<\/span><\/p>\n

 <\/p>\n

\u4f46 Luxturna \u7684\u6210\u7ee9\u770b\u8d77\u6765\u5f88\u7a81\u51fa\u548c\u663e\u8457\uff0c\u5728\u5bf9\u60a3\u8005\u7684\u6d4b\u8bd5\u4e2d\uff0c\u6062\u590d\u60a3\u8005\u53ef\u4ee5\u770b\u5230\u4ed6\u4eec\u4ee5\u524d\u4ece\u672a\u89c1\u8fc7\u7684\u4e1c\u897f\u7684\u80fd\u529b\uff0c\u4f8b\u5982\u6708\u4eae\uff0c\u70df\u82b1\u548c\u4ed6\u4eec\u7236\u6bcd\u7684\u8138\u3002<\/span><\/p>\n

 <\/p>\n

\u6cbb\u7597\u8fd8\u4f7f\u60a3\u8005\u80fd\u591f\u5b8c\u6210\u8bb8\u591a\u4e0d\u53ef\u80fd\u7684\u4e8b\u60c5\uff0c\u4f8b\u5982\u9605\u8bfb\uff0c\u4f53\u80b2\u8fd0\u52a8\uff0c\u9a91\u81ea\u884c\u8f66\u4ee5\u53ca\u81ea\u5df1\u665a\u4e0a\u5916\u51fa\u3002<\/span><\/p>\n

 <\/span><\/p>\n

\u5f53\u65f6\u7684\u8fd9\u9879\u836f\u7269\uff0c\u62a5\u4ef7\u662f 47.5 \u4e07\u7f8e\u5143<\/span>\uff08\u7ea6 327.65 \u4e07\u5143\u4eba\u6c11\u5e01\uff09<\/span>\u4e00\u53ea\u773c\u775b\u3002<\/span><\/p>\n


<\/p>\n

\u4f46\u5bf9\u6bd4\u8fd9\u4e2a\u8d39\u7528\uff0c\u4eca\u5929\u7684\u4e3b\u89d2 Zolgensma \u5237\u65b0\u4e86\u5927\u5bb6\u7684\u8ba4\u8bc6\uff1a\u4e00\u6b21\u6cbb\u7597\u9700\u8981\u8d39\u7528\u4e3a 212.5 \u4e07\u7f8e\u5143<\/span>\uff08\u7ea6\u4e3a 1465.83 \u4e07\u4eba\u6c11\u5e01\uff09<\/span>\u3002<\/span><\/p>\n


<\/p>\n

\"1400<\/p>\n

\u5929\u4ef7Zolgensma<\/span><\/p>\n

\u56fe\u7247\u6765\u6e90\uff1a\u8bfa\u534e<\/span><\/p>\n


<\/p>\n

\u8bfa\u534e\u516c\u53f8\u9996\u5e2d\u6267\u884c\u5b98 Vas Narasimhan \u5728\u4e0e\u8bb0\u8005\u7684\u7535\u8bdd\u4f1a\u8bae\u4e0a\u8bf4\uff1a\u300c\u6211\u4eec\u89c9\u5f97\u6211\u4eec\u6b63\u8d70\u5728\u4e00\u6761\u9053\u8def\u4e0a\uff0c\u6211\u4eec\u5e0c\u671b\u6709\u4e00\u5929\u80fd\u591f\u8ba9 SMA \u51e0\u4e4e\u6d88\u706d<\/span>\u300d<\/span>\u3002<\/span><\/p>\n

 <\/p>\n

\u8bb8\u591a\u7f55\u89c1\u75be\u75c5\u836f\u7269\u6700\u8fd1\u4e0a\u5e02\uff0c\u6700\u8fd1\u6bcf\u5e74\u82b1\u8d39\u6570\u5341\u4e07\u7f8e\u5143\u3002\u4f46\u5f88\u5c11\u6709\u4eba\u8d85\u8fc7 100 \u4e07\u7f8e\u5143\u7684\u95e8\u69db\u3002<\/span><\/p>\n


<\/p>\n


<\/p>\n

\u5929\u4ef7\u836f\u7269\u7684\u4f26\u7406\u8ba8\u8bba<\/span><\/strong><\/span><\/p>\n

 <\/p>\n

\u597d\u836f\u8981\u8d35\u4ef7\u3002\u8fd9\u4f3c\u4e4e\u662f\u5929\u7136\u7684\u9053\u7406\u3002<\/span><\/p>\n

 <\/p>\n

\u4f46\u8fd9\u4e2a\u836f\u7269\u4e4b\u300c\u5929\u4ef7\u7a0b\u5ea6\u300d\u786e\u5b9e\u8ba9\u4eba\u548b\u820c\uff0c210 \u4e07\u7f8e\u5143\uff0c\u51e0\u8fd1 1400 \u591a\u4e07\u4eba\u6c11\u5e01\u4e00\u5242\u7684\u8d39\u7528\uff0c\u8ba9\u5b83\u81ea\u8bde\u751f\u8d77\uff0c\u5c31\u6302\u4e0a\u4e86\u300c\u5168\u7403\u6700\u8d35\u836f\u7269\u300d\u7684\u6842\u51a0\u3002<\/span><\/p>\n

 <\/p>\n

\u518d\u52a0\u4e0a\u75be\u75c5\u672c\u8eab\u7684\u81f4\u547d\u548c\u4e25\u91cd\u7a0b\u5ea6\uff0c\u4eba\u4eec\u5bf9\u5b83\u7684\u8bc4\u4ef7\u8912\u8d2c\u4e0d\u4e00\u3002<\/span><\/p>\n

 <\/p>\n

\u6709\u4eba\u8ba4\u4e3a\uff0c\u8fd9\u662f\u5c5e\u4e8e\u5168\u4eba\u7c7b\u7684\u79d1\u6280\u6210\u679c\uff0c\u4ece\u9053\u5fb7\u4e0a\u6765\u8bf4\uff0c\u5e94\u8be5\u6709\u66f4\u5408\u7406\u7684\u5b9a\u4ef7\u8ba9\u5b83\u666e\u60e0\u66f4\u591a\u60a3\u91cd\u75c5\u7684\u5b69\u5b50\u3002<\/span><\/p>\n


<\/p>\n

\u6709\u4eba\u5219\u8ba4\u4e3a\uff0c\u6ca1\u6709\u5929\u4ef7\u7684\u56de\u62a5\uff0c\u836f\u5382\u7edd\u65e0\u5f00\u53d1\u7684\u52a8\u529b\uff0c\u8fd9\u6b3e\u836f\uff0c\u53ef\u80fd\u8fde\u4e0d\u4f1a\u88ab\u7814\u53d1\uff0c\u751a\u81f3\u8fde\u4e00\u4e2a\u5b69\u5b50\u90fd\u65e0\u6cd5\u60e0\u53ca\u3002<\/span><\/p>\n

 <\/p>\n

\u800c\u836f\u5382\u7684 CEO\uff0c\u5219\u7528\u5728\u7f8e\u56fd\u8fdb\u884c\u5668\u5b98\u79fb\u690d\u624b\u672f\u7684\u8d39\u7528\u4f5c\u4e3a\u5bf9\u6bd4\uff0c\u53cd\u9a73\u4e86\u6279\u8bc4\u7684\u4eba\u4eec\uff1a<\/span><\/p>\n

 <\/p>\n

\u300c<\/span>\uff08\u5728\u7f8e\u56fd\uff09<\/span>\u79fb\u690d\u624b\u672f\u52a8\u8f84\u9700\u8981\u82b1\u8d39 300\uff5e500 \u4e07\u7f8e\u5143\uff0c\u8fdc\u9ad8\u4e8e\u4e00\u5242\u57fa\u56e0\u836f\u7269\uff0c\u4f46\u8bb2\u5230\u7597\u6548\uff0c\u5374\u672a\u5fc5\u6709\u57fa\u56e0\u836f\u7269\u5bf9\u75be\u75c5\u7684\u6539\u5584\u8fd9\u4e48\u6709\u4fdd\u8bc1<\/span>\uff08\u6bd4\u5982\u79fb\u690d\u5931\u8d25\u7b49\uff09<\/span>\u3002\u96be\u9053\u5927\u5bb6\u5e94\u8be5\u53cd\u601d\u7684\uff0c\u4e0d\u662f\u533b\u7597\u7cfb\u7edf\u7684\u8fd0\u4f5c\u65b9\u6cd5\u5417\uff1f\u300d<\/span><\/p>\n

 <\/span><\/p>\n

\u4f60\u5462\uff1f\u4f60\u53c8\u600e\u6837\u8ba4\u4e3a\u5462\uff1f<\/span><\/p>\n


<\/p>\n

\u9898\u56fe\uff1aThe New York Times<\/span><\/span><\/p>\n

\u8d23\u7f16\uff1a\u674e\u5b50\u6021<\/span><\/p>\n

\"1400<\/span><\/p>\n


<\/p>\n

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

1. FDA \u62a5\u9053\uff1a<\/span>https:\/\/www.fda.gov\/news-events\/press-announcements\/fda-approves-innovative-gene-therapy-treat-pediatric-patients-spinal-muscular-atrophy-rare-disease<\/span><\/p>\n

2.The New York Times\uff1a<\/span>https:\/\/www.nytimes.com\/2019\/05\/24\/health\/zolgensma-gene-therapy-drug.html<\/span><\/p>\n

3. \u9a6c\u6cf0\u5965\u7684\u6545\u4e8b\u62a5\u9053\uff1a<\/span>https:\/\/www.nbcnews.com\/health\/health-news\/2-1-million-drug-treat-rare-genetic-disease-approved-fda-n1009956<\/span><\/p>\n

4. Donovan Weisgarber \u6545\u4e8b\u62a5\u9053\uff1a<\/span>https:\/\/www.npr.org\/sections\/health-shots\/2019\/05\/24\/725404168\/at-2-125-million-new-gene-therapy-is-the-most-expensive-drug-ever<\/span><\/p>\n

5. NIH \u5173\u4e8e SMA \u7684\u4e8b\u5b9e\u9648\u8ff0\uff1a<\/span>https:\/\/www.ninds.nih.gov\/Disorders\/Patient-Caregiver-Education\/Fact-Sheets\/Spinal-Muscular-Atrophy-Fact-Sheet<\/span><\/p>\n

6. \u7b2c\u4e00\u6b3e\u57fa\u56e0\u6cbb\u7597\u836f\u7269\u4e0a\u5e02\u7684\u65b0\u95fb\uff1a<\/span>https:\/\/www.npr.org\/sections\/health-shots\/2017\/12\/19\/571962226\/first-gene-therapy-for-inherited-disease-gets-fda-approval<\/span><\/p>\n


<\/p>\n<\/p><\/div>\n","protected":false},"excerpt":{"rendered":"

\u8fd9\u662f\u5173\u4e8e\u51e0\u4e2a\u7edd\u671b\u7684\u5bb6\u5ead\u5f97\u5230\u300c\u91cd\u751f\u300d\u7684\u6545\u4e8b\u3002   Matteo Almeida \u8fd8\u5728\u5988\u5988Nicole […]<\/p>\n","protected":false},"author":1,"featured_media":112730,"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\/112729"}],"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=112729"}],"version-history":[{"count":1,"href":"https:\/\/www.05921.net\/index.php\/wp-json\/wp\/v2\/posts\/112729\/revisions"}],"predecessor-version":[{"id":112736,"href":"https:\/\/www.05921.net\/index.php\/wp-json\/wp\/v2\/posts\/112729\/revisions\/112736"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.05921.net\/index.php\/wp-json\/wp\/v2\/media\/112730"}],"wp:attachment":[{"href":"https:\/\/www.05921.net\/index.php\/wp-json\/wp\/v2\/media?parent=112729"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.05921.net\/index.php\/wp-json\/wp\/v2\/categories?post=112729"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.05921.net\/index.php\/wp-json\/wp\/v2\/tags?post=112729"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}