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Riley\u76845\u5c81\u5973\u513fOlivia\u5728\u88ab\u8bca\u65ad\u51fa\u60a3\u6709MLD\u540e\uff0c\u73b0\u5728\u4f4f\u5728\u4e34\u7ec8\u5173\u6000\u533b\u9662\u3002Riley\u8868\u793a\uff0c\u6700\u65e9\u53d1\u73b0\u6709\u95ee\u9898\u7684\u8ff9\u8c61\u662fOlivia\u5927\u7ea62\u5c81\u65f6\u3002\u5979\u5f00\u59cb\u884c\u8d70\u56f0\u96be\uff0c\u5f53\u5979\u770b\u7535\u89c6\u65f6\uff0c\u5934\u90e8\u7ecf\u5e38\u503e\u659c\u3002Riley\u8bf4\uff1a\u201c\u6211\u4eec\u5f53\u65f6\u4ee5\u4e3a\u53ea\u9700\u8981\u4e00\u4e9b\u7269\u7406\u6cbb\u7597\uff0c\u7136\u540e\u5979\u7684\u773c\u775b\u5f00\u59cb\u9707\u98a4\u3002\u90a3\u65f6\u6211\u4eec\u5c31\u610f\u8bc6\u5230\u53ef\u80fd\u5b58\u5728\u5176\u4ed6\u95ee\u9898\u3002\u201d\u533b\u751f\u8bca\u65ad\u51faOlivia\u60a3\u6709MLD\u65f6\uff0c\u57fa\u56e0\u6cbb\u7597\u5df2\u7ecf\u6765\u4e0d\u53ca\u4e86\u3002\u4f46\u662f\uff0c\u77e5\u9053\u8fd9\u79cd\u60c5\u51b5\u662f\u9057\u4f20\u6027\u7684\uff0c\u4ed6\u4eec\u5c31\u5bf9\u4ed6\u4eec\u7684\u5c0f\u5973\u513fKeira\u8fdb\u884c\u4e86\u68c0\u67e5\u548c\u8bca\u65ad\u3002\u73b0\u5e744\u5c81\u7684Keira\u662f\u4e16\u754c\u4e0a\u7b2c32\u4e2a\u63a5\u53d7\u8fd9\u79cd\u65b0\u7597\u6cd5\u7684\u5b69\u5b50\uff0c\u8fd9\u79cd\u7597\u6cd5\u5728\u5b69\u5b50\u51fa\u73b0\u75c7\u72b6\u4e4b\u524d\u6700\u4e3a\u6709\u6548\u3002\u5c3d\u7ba1\u8be5\u516c\u53f8\u63d0\u4f9b\u57fa\u56e0\u6cbb\u7597\uff0c\u4f46\u4ed6\u4eec\u5728\u56fd\u5916\u751f\u6d3b\u548c\u652f\u4ed8Keira\u7684\u533b\u7597\u8d39\u7528\u5c06\u82b1\u8d39\u7ea650\u4e07\u7f8e\u5143\u3002Riley\u8868\u793a\uff1a\u201cFDA\u6279\u51c6\u7684\u597d\u5904\u662f\uff0c\u5982\u679c\u5b69\u5b50\u5728\u51fa\u73b0\u75c7\u72b6\u4e4b\u524d\u88ab\u8bca\u65ad\u51fa\u60a3\u6709\u8be5\u75c5\uff0c\u4ed6\u4eec\u5c31\u6709\u673a\u4f1a\u8fc7\u4e0a\u6b63\u5e38\u7684\u751f\u6d3b\u3002\u201d\u8be5\u6cbb\u7597\u65b9\u6848\u5728\u7f8e\u56fd\u5c1a\u672a\u63a8\u51fa\uff0c\u56e0\u6b64\u8fd9\u5bb6\u5ead\u4e8e2020\u5e74\u901a\u8fc7\u4f17\u7b79\u7b79\u96c6\u8d44\u91d1\uff0c\u5e76\u6682\u65f6\u642c\u5230\u610f\u5927\u5229\u63a5\u53d7\u6cbb\u7597\u3002<\/p>\n
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\u6797\u5fb7\u6307\u51fa\uff0c\u5373\u4f7f\u5b9a\u4ef7\u8fbe\u5230\u6700\u9ad8\u6c34\u5e73\uff0c\u8fd9\u79cd\u6cbb\u7597\u4e5f\u4e0d\u4f1a\u6210\u4e3a\u7545\u9500\u54c1\uff0c\u56e0\u4e3a\u8fd9\u79cd\u75be\u75c5\u975e\u5e38\u7f55\u89c1\u3002\u4e00\u4e9b\u7528\u4e8e\u6cbb\u7597\u7f55\u89c1\u75be\u75c5\u7684\u57fa\u56e0\u7597\u6cd5\u5df2\u7ecf\u6d88\u5931\uff0c\u56e0\u4e3a\u5236\u9020\u8fd9\u4e9b\u7597\u6cd5\u7684\u516c\u53f8\u65e0\u6cd5\u83b7\u5f97\u8db3\u591f\u7684\u5229\u6da6\u4ee5\u7ef4\u6301\u8fd0\u8425\u3002Orchard Therapeutics\u516c\u53f8\u4e4b\u524d\u5f00\u53d1\u7684\u4e00\u79cd\u57fa\u56e0\u7597\u6cd5\u5df2\u7ecf\u96be\u4ee5\u76c8\u5229\uff0c\u4f7f\u60a3\u8005\u611f\u5230\u65e0\u5948\u3002\u6797\u5fb7\u8868\u793a\uff0cICER\u5ba1\u67e5\u8fd9\u79cd\u836f\u7269\u7684\u539f\u56e0\u4e4b\u4e00\u662f\uff0c\u5b83\u7528\u4e8e\u6cbb\u7597\u4e00\u79cd\u201c\u8d85\u7ea7\u7f55\u89c1\u75be\u75c5\u201d\uff0c\u4f46\u8be5\u516c\u53f8\u8ba4\u4e3a\u5b83\u53ef\u4ee5\u4ee5\u5177\u6709\u6210\u672c\u6548\u76ca\u7684\u4ef7\u683c\u63d0\u4f9b\u8fd9\u79cd\u836f\u7269\u3002\u201c\u5982\u679c\u4ed6\u4eec\u7684\u4ef7\u683c\u4f4e\u4e8e390\u4e07\u7f8e\u5143\uff0c\u90a3\u5b9e\u9645\u4e0a\u5c06\u662f\u4e00\u4e2a\u597d\u8ff9\u8c61\uff0c\u5373\u4f7f\u5bf9\u4e8e\u6700\u7f55\u89c1\u7684\u7f55\u89c1\u75be\u75c5\uff0c\u4f60\u4e5f\u53ef\u4ee5\u627e\u5230\u4e00\u5bb6\u8ba4\u4e3a\u53ef\u4ee5\u4ee5\u4e0d\u8fc7\u5206\u7684\u4ef7\u683c\u63d0\u4f9b\u6cbb\u7597\u7684\u5236\u836f\u516c\u53f8\u3002\u201d<\/p>\n
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Orchard Therapeutics\u8868\u793a\uff0c\u8fd9\u4e00\u8fdb\u5c55\u7ecf\u5386\u4e86\u51e0\u5341\u5e74\u7684\u5386\u7a0b\u3002Orchard Therapeutics\u7684\u8054\u5408\u521b\u59cb\u4eba\u517c\u9996\u5e2d\u6267\u884c\u5b98\u9c8d\u6bd4\u00b7\u52a0\u65af\u5e15\uff08Bobby Gaspar\uff09\u535a\u58eb\u5728\u65b0\u95fb\u53d1\u5e03\u4f1a\u4e0a\u8bf4\uff1a\u201cFDA\u6279\u51c6Lenmeldy\u4e3a\u7f8e\u56fd\u65e9\u53d1MLD\u513f\u7ae5\u5f00\u8f9f\u4e86\u5de8\u5927\u7684\u65b0\u53ef\u80fd\u6027\uff0c\u8fd9\u4e9b\u513f\u7ae5\u4ee5\u524d\u9664\u4e86\u652f\u6301\u548c\u4e34\u7ec8\u5173\u6000\u4e4b\u5916\u6ca1\u6709\u4efb\u4f55\u6cbb\u7597\u9009\u62e9\u3002\u201d\u829d\u52a0\u54e5Ann\uff06Robert H. Lurie\u513f\u7ae5\u533b\u9662\u7684\u9057\u4f20\u5b66\u3001\u57fa\u56e0\u7ec4\u5b66\u548c\u4ee3\u8c22\u7684\u4e3b\u6cbb\u533b\u5e08\u82ad\u82ad\u62c9\u00b7\u4f2f\u987f\uff08Barbara Burton\uff09\u535a\u58eb\u8868\u793a\uff0c\u8fd9\u5bf9\u533b\u751f\u6765\u8bf4\u662f\u4e00\u4e2a\u5386\u53f2\u6027\u65f6\u523b\u3002\u201c\u957f\u671f\u4ee5\u6765\uff0c\u6211\u548c\u6211\u7684\u540c\u4e8b\u4eec\u4e00\u76f4\u5728\u5bb6\u5ead\u6700\u8106\u5f31\u7684\u65f6\u5019\u5b89\u6170\u4ed6\u4eec\u2014\u2014\u901a\u5e38\u662f\u5728\u7ecf\u5386\u4e86\u8270\u96be\u7684\u8bca\u65ad\u8fc7\u7a0b\u4e4b\u540e\uff0c\u9762\u5bf9\u53ef\u6015\u7684\u9884\u540e\uff0c\u88ab\u544a\u77e5\u6ca1\u6709\u6cbb\u7597\u65b9\u6cd5\uff0c\u7136\u540e\u4e0d\u5f97\u4e0d\u770b\u7740\u4ed6\u4eec\u5e74\u5e7c\u7684\u5b69\u5b50\u6084\u6084\u79bb\u5f00\u3002\u201d\u5979\u8bf4\u9053\u3002\u201c\u6709\u4e86\u8fd9\u9879\u6279\u51c6\uff0c\u6211\u4eec\u73b0\u5728\u79bb\u786e\u4fdd\u540e\u4ee3\u7684\u513f\u7ae5\u3001\u5bb6\u5ead\u548c\u533b\u7597\u4fdd\u5065\u4e13\u4e1a\u4eba\u5458\u4e0d\u518d\u9700\u8981\u4eb2\u8eab\u4f53\u9a8c\u8fd9\u79cd\u75be\u75c5\u7ed9\u672a\u7ecf\u6cbb\u7597\u7684\u60a3\u8005\u5e26\u6765\u7684\u53ef\u6015\u75c7\u72b6\u53c8\u8fd1\u4e86\u4e00\u6b65\u3002\u201d<\/p>\n
Riley\u8868\u793a\uff0c\u76ee\u524dOlivia\u7684\u60c5\u51b5\u7a33\u5b9a\uff0c\u4f46\u5979\u62c5\u5fc3Keira\u53ef\u80fd\u4f1a\u5e26\u56de\u7ec6\u83cc\u3002\u201c\u666e\u901a\u611f\u5192\u53ef\u80fd\u4f1a\u81f4\u547d\uff0c\u201d Riley\u8bf4\uff0c\u201c\u6240\u4ee5\u6bcf\u6b21\u5979\u59b9\u59b9\u4ece\u5b66\u6821\u5e26\u56de\u611f\u5192\u65f6\uff0c\u90a3\u53ef\u80fd\u662f\u6f5c\u5728\u5bfc\u81f4\u5979\u79bb\u4e16\u7684\u539f\u56e0\u3002\u201dOlivia\u4e0d\u80fd\u884c\u8d70\u6216\u8bf4\u8bdd\uff0c\u5979\u901a\u8fc7\u7ba1\u9053\u5582\u98df\u3002 Riley\u8bf4\u5979\u9700\u8981\u5168\u5929\u5019\u7684\u62a4\u7406\u3002\u6bcf\u5468\u4ed6\u4eec\u6709\u7269\u7406\u548c\u804c\u4e1a\u6cbb\u7597\u5e08\u6765\u5bb6\u91cc\uff0c\u4ee5\u201c\u8ba9\u5979\u7684\u601d\u7ef4\u6d3b\u52a8\u8d77\u6765\u3002\u5979\u786e\u5b9e\u7406\u89e3\u53d1\u751f\u4e86\u4ec0\u4e48\u3002\u201d\u5979\u8bf4\uff0c\u5f53\u4ed6\u4eec\u770b\u7740Olivia\u6162\u6162\u6d88\u5931\u65f6\uff0c\u4ed6\u4eec\u610f\u8bc6\u5230\u5979\u7684\u751f\u547d\u5177\u6709\u6df1\u8fdc\u7684\u610f\u4e49\u3002\u4f46\u4ed6\u4eec\u5e0c\u671bLenmeldy\u7684\u6279\u51c6\u610f\u5473\u7740\u4e0d\u4f1a\u518d\u6709\u5176\u4ed6\u60a3\u6709MLD\u7684\u5bb6\u5ead\u4e3a\u4e86\u62ef\u6551\u53e6\u4e00\u4e2a\u5b69\u5b50\u800c\u5931\u53bb\u4e00\u4e2a\u5b69\u5b50\u3002<\/p>\n
\uff08\u56fe\u7247\u6e90\u4e8eCNN\uff09<\/p>\n
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