{"id":117316,"date":"2019-06-11T12:43:54","date_gmt":"2019-06-11T04:43:04","guid":{"rendered":"http:\/\/www.05921.net\/index.php\/117316\/"},"modified":"2019-06-11T12:43:05","modified_gmt":"2019-06-11T04:43:05","slug":"8da822fdbc","status":"publish","type":"post","link":"https:\/\/www.05921.net\/index.php\/117316\/","title":{"rendered":"\u6700\u8d35\u836f\u7269\u76d8\u70b9"},"content":{"rendered":"
\n

\"\u6700\u8d35\u836f\u7269\u76d8\u70b9\"<\/p>\n


<\/p>\n

\u258d<\/span><\/strong><\/span><\/strong>\u6765\u6e90\uff1a\u8d5b\u67cf\u84dd    \u6574\u7406\uff1a\u534a\u590f<\/span><\/p>\n

 <\/span><\/p>\n

\u8fd1\u65e5\uff0c\u7f8e\u56fdFDA\u6279\u51c6\u4e86\u8bfa\u534e\u516c\u53f8\u7528\u4e8e\u4e00\u6b21\u6027\u6cbb\u7597\u810a\u9ad3\u6027\u808c\u840e\u7f29\uff08SMA\uff09\u7684\u57fa\u56e0\u7597\u6cd5 Zolgensma \u4e0a\u5e02\u3002<\/span><\/p>\n

 <\/span><\/p>\n

\u4f5c\u4e3a\u4e00\u6b3e\u5907\u53d7\u77a9\u76ee\u7684\u57fa\u56e0\u7597\u6cd5\uff0cZolgensma \u53ef\u6839\u672c\u6027\u5730\u89e3\u51b3 1 \u578b SMA \u7684\u9057\u4f20\u75c5\u56e0\uff0c\u5728\u4e00\u6b21\u6027\u6cbb\u6108\u7f55\u89c1\u9057\u4f20\u75c5\u53f2\u4e0a\u4e5f\u5177\u6709\u7edd\u5bf9\u7684\u91cc\u7a0b\u7891\u8fdb\u5c55\u3002<\/span><\/p>\n

 <\/span><\/p>\n

\u636e\u5a92\u4f53\u62a5\u9053\uff0c\u8be5\u836f\u4e00\u9488\u836f\u6807\u4ef7210\u4e07\u7f8e\u5143\uff0c\u6298\u5408\u7ea61448\u4e07\u4eba\u6c11\u5e01\uff0c\u582a\u79f0\u53f2\u4e0a\u6700\u8d35\u836f\u7269\u3002<\/span><\/p>\n

 <\/span><\/p>\n

\u258d<\/span><\/strong><\/span><\/strong><\/span>\u6551\u547d\u836f=\u5929\u4ef7\u836f\uff1f<\/strong><\/span><\/p>\n

 <\/span><\/p>\n

\u636e\u6089\uff0cSMA \u662f\u6700\u5e38\u89c1\u7684\u81f4\u6b7b\u6027\u795e\u7ecf\u808c\u8089\u75be\u75c5\u4e4b\u4e00\uff0c\u4e5f\u662f\u5bfc\u81f4\u5a74\u513f\u6b7b\u4ea1\u7684\u5934\u53f7\u9057\u4f20\u75c5\u56e0\uff0c\u7531\u5355\u57fa\u56e0\u8fd0\u52a8\u795e\u7ecf\u5143\u5b58\u6d3b\u57fa\u56e0 SMN1 \u7f3a\u9677\u5f15\u8d77\u3002\u6839\u636e\u75be\u75c5\u7684\u53d1\u751f\u65f6\u95f4\u548c\u4e25\u91cd\u7a0b\u5ea6\uff0cSMA \u53ef\u5206\u4e3a 1 \u578b\u30012 \u578b\u30013 \u578b\u548c 4 \u578b\uff0c\u5927\u7ea6 60% \u7684 SMA \u60a3\u8005\u662f 1 \u578b\uff0c\u4e5f\u662f\u6700\u4e25\u91cd\u7684\u4e00\u79cd\u7c7b\u578b\u3002<\/span><\/p>\n

 <\/span><\/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

 <\/span><\/p>\n

\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

 <\/span><\/p>\n

\u6709\u6570\u636e\u4f30\u8ba1\uff0cSMA \u53d1\u75c5\u7387\u4e3a 1\/6000~1\/10000\uff0c\u4e5f\u5c31\u662f\u8bf4\u6bcf 10000 \u540d\u65b0\u751f\u513f\u4e2d\u5c31\u6709\u4e00\u4eba\u53d7\u6b64\u5f71\u54cd\uff0c\u5176\u4e2d 90% \u7684\u60a3\u75c5\u5a74\u513f\u6d3b\u4e0d\u8fc7 2 \u5c81\u3002<\/span><\/p>\n

 <\/span><\/p>\n

\u867d\u7136\u6cbb\u7597SMA\u7684\u836f\u7269Zolgensma\u5df2\u6210\u529f\u9762\u5e02\uff0c\u4f46\u5e76\u4e0d\u662f\u4eba\u4eba\u90fd\u8d1f\u62c5\u5f97\u8d77\uff0c\u4e00\u652f\u836f\u7269\u5c31\u8981\u5929\u4ef7210\u4e07\u7f8e\u5143\uff08\u7ea61448\u4e07\u5143\uff09\uff0c\u582a\u79f0\u6709\u53f2\u4ee5\u6765\u6700\u6602\u8d35\u7684\u836f\u7269\u3002<\/span><\/p>\n

 <\/span><\/p>\n

\u8fc7\u53bb\u5e02\u9762\u4e0a\u53ea\u6709\u4e00\u79cd\u6cbb\u7597\u6b64\u75be\u75c5\u7684\u836f\u7269\uff0c\u5982\u4eca\u63a8\u51faZolgensma\uff0c\u65e0\u7591\u662f\u7ed9\u4e86\u75c5\u7ae5\u7236\u6bcd\u6781\u5927\u5e0c\u671b\uff0c\u4f46\u76f8\u5173\u4e13\u5bb6\u4e5f\u8d28\u7591\uff1a\u201c\u8bb8\u591a\u836f\u7269\u7684\u95ee\u9898\u5728\u4e8e\u5b83\u4eec\u592a\u8d35\uff0c\u6709\u9700\u8981\u7684\u4eba\u6839\u672c\u8d1f\u62c5\u4e0d\u8d77\u3002\u201d<\/span><\/p>\n

 <\/span><\/p>\n

\u7f51\u53cb\u7eb7\u7eb7\u8d28\u7591\uff1a\u8fd9\u662f\u5728\u62ff\u5b69\u5b50\u7684\u547d\u8981\u631f\u5bb6\u957f\uff0c\u8ba8\u8981\u8d4e\u91d1\u3002\u4f46\u533b\u836f\u516c\u53f8\u5219\u79f0\u6cbb\u7597\u8be5\u75c5\u53ea\u9700\u4e00\u652f\u836f\uff0c\u4e14\u5728\u533b\u4fdd\u8986\u76d6\u8303\u56f4\u5185\uff0c\u8fd8\u53ef\u4ee5\u5206\u671f\u4ed8\u6b3e\u3002<\/span><\/p>\n

 <\/span><\/p>\n

\u8be5\u516c\u53f8CEO\u8868\u793a\uff0c\u7531\u4e8e\u8fd9\u79cd\u7597\u6cd5\u53ea\u8fdb\u884c\u4e00\u6b21\uff0c\u6240\u4ee5\u8d39\u7528\u6bd4\u76ee\u524d\u7684\u7597\u6cd5\u90fd\u8981\u4fbf\u5b9c\uff0c\u8be5\u7597\u6cd5\u7ea6\u4e3a\u957f\u671f\u6cbb\u7597\u8d39\u7528\u7684\u4e00\u534a\uff0c\u60a3\u8005\u82e5\u5b58\u6d3b\u4e94\u5e74\uff0c\u76f8\u5f53\u4e8e\u6bcf\u5e74\u82b1\u8d3942.5\u4e07\u7f8e\u5143\uff0c\u800c\u73b0\u6709\u7684\u8be5\u75be\u75c5\u7597\u6cd5\uff0c\u7b2c\u4e00\u5e74\u8d39\u7528\u5c31\u970075\u4e07\u7f8e\u5143\u3002\u800c\u4e14\u5728\u533b\u4fdd\u8303\u56f4\u5185\uff0c\u5c31\u50cf\u8d37\u6b3e\u4e00\u6837\uff0c\u4fdd\u9669\u516c\u53f8\u4f1a\u5728\u4e94\u5e74\u5185\u5206\u671f\u4ed8\u6b3e\u3002<\/span><\/p>\n

 <\/span><\/p>\n

\u901a\u8fc7\u8be5\u7597\u6cd5\u540e\u75ca\u6108\u513f\u7ae5\u7684\u5bb6\u957f\u5219\u8868\u793a\uff0c\u751f\u547d\u65e0\u6cd5\u7528\u91d1\u94b1\u8861\u91cf\u3002<\/span><\/p>\n

 <\/span><\/p>\n

\u663e\u7136\uff0c\u8ba9\u6bcf\u4e00\u4f4d\u60a3\u8005\u90fd\u53d8\u6709\u94b1\uff0c\u6216\u8005\u8ba9\u836f\u4f01\u8d54\u672c\u5927\u964d\u4ef7\uff0c\u90fd\u662f\u4e0d\u592a\u73b0\u5b9e\u7684\u60f3\u6cd5\u3002\u4f46\u8fd9\u5e76\u4e0d\u610f\u5473\u7740\u5c31\u6ca1\u6709\u529e\u6cd5\u89e3\u51b3\u6551\u547d\u836f\u7684\u5929\u4ef7\u8d39\u7528\u95ee\u9898\u3002\u6362\u4e2a\u89d2\u5ea6\u770b\uff0c\u4e00\u4e2a\u6ca1\u6709\u5b58\u6b3e\u4f46\u4e70\u4e86\u5065\u5eb7\u9669\u7684\u764c\u75c7\u60a3\u8005\uff0c\u5374\u53ef\u4ee5\u83b7\u5f97\u4e00\u7b14\u6bd4\u81ea\u5df1\u4e70\u4fdd\u9669\u6295\u5165\u591a\u5f88\u591a\u7684\u53ef\u89c2\u8d44\u91d1\u7528\u4e8e\u81ea\u5df1\u7684\u75be\u75c5\u6cbb\u7597\u3002<\/span><\/p>\n

 <\/span><\/p>\n

\u258d<\/span><\/strong><\/span><\/strong><\/span>\u6602\u8d35\u836f\u7269\u76d8\u70b9<\/strong><\/span><\/p>\n

 <\/span><\/p>\n

\u9664\u4e86\u4e0a\u8ff0\u7528\u4e8e\u4e00\u6b21\u6027\u6cbb\u7597\u810a\u9ad3\u6027\u808c\u840e\u7f29\uff08SMA\uff09\u7684\u57fa\u56e0\u7597\u6cd5 Zolgensma\uff0c\u8fd8\u6709\u5f88\u591a\u836f\u7269\u7684\u4e5f\u662f\u975e\u5e38\u6602\u8d35\u7684\uff0c\u8ba9\u6211\u4eec\u4e00\u540c\u76d8\u70b9\u4e00\u4e0b\u3002<\/span><\/p>\n

 <\/span><\/p>\n

1\u3001Actimmune  52322\u7f8e\u5143\/\u6708<\/span><\/p>\n

 <\/span><\/p>\n

\u8be5\u836f\u9002\u7528\u4e8e\u6cbb\u7597\u9aa8\u786c\u5316\u75c7\uff08Osteopetrosis\uff09\u548c\u6162\u6027\u8089\u82bd\u80bf\u75c5\uff08Chronic Granulomatous Disease\uff09\u4e24\u79cd\u7f55\u89c1\u75c5\u3002\u60a3\u8005\u4e00\u822c\u6bcf\u5468\u6ce8\u5c043\u6b21Actimmune\uff0c\u6bcf\u6708\u5e73\u5747\u6ce8\u5c0412\u5242\uff0c\u6bcf\u5242\u7684\u4ef7\u683c\u4e3a4360\u7f8e\u5143\u3002<\/span><\/p>\n

 <\/span><\/p>\n

2\u3001Myalept  46328\u7f8e\u5143\/\u6708<\/span><\/p>\n

 <\/span><\/p>\n

Myalept\u7531Aegerion\u516c\u53f8\u7814\u53d1\uff0c\u662f\u4e00\u6b3e\u7528\u4e8e\u6cbb\u7597\u5168\u8eab\u6027\u8102\u80aa\u8425\u517b\u4e0d\u826f\uff08Generalized Lipodystrophy\uff09\u5b64\u513f\u836f\uff0c\u4e5f\u662f\u76ee\u524d\u63a7\u5236\u8fd9\u79cd\u7f55\u89c1\u75c5\u7684\u552f\u4e00\u836f\u7269\u3002\u60a3\u8005\u901a\u5e38\u6bcf\u6708\u4f7f\u752810\u5242\uff0c\u6bcf\u5242\u6807\u4ef7\u7ea64632\u7f8e\u5143\u3002<\/span><\/p>\n

 <\/span><\/p>\n

3\u3001Daraprim  45000\u7f8e\u5143\/\u6708<\/span><\/p>\n

 <\/span><\/p>\n

\u8be5\u836f\u662f\u4e00\u79cd\u4e13\u95e8\u6cbb\u7597\u5bc4\u751f\u866b\uff08\u5982\u5f13\u5f62\u866b\u75c5\uff09\u611f\u67d3\u7684\u836f\uff0c\u7531Vyera\u5236\u836f\u516c\u53f8\uff08\u524d\u8eab\u662f\u56fe\u7075\u5236\u836f\uff09\u9500\u552e\u3002\u503c\u5f97\u63d0\u53ca\u7684\u662f\uff0cDaraprim\u7684\u4ef7\u683c\u662f\u57282015\u5e74\uff0c\u7531\u516c\u53f8\u524dCEO\u2014Martin Shkreli\u5c06\u5176\u4ef7\u683c\u4ece\u6bcf\u724713.50\u7f8e\u5143\u63d0\u9ad8\u5230750\u7f8e\u5143\u5de6\u53f3\uff0c\u4ece\u800c\u5f15\u53d1\u4e86\u4e00\u573a\u836f\u7269\u5b9a\u4ef7\u98ce\u66b4\u3002\u968f\u540e\u53d7\u5230\u4e25\u683c\u5ba1\u67e5\uff0c\u4f46\u5176\u6807\u4ef7\u4ecd\u5c45\u9ad8\u4e0d\u4e0b\u3002<\/span><\/p>\n

 <\/span><\/p>\n

4\u3001Cinryze  44141\u7f8e\u5143\/\u6708<\/span><\/p>\n

 <\/span><\/p>\n

\u8be5\u836f\u7531Shire\u516c\u53f8\u751f\u4ea7\uff0c\u7528\u4e8e\u6cbb\u7597\u9057\u4f20\u6027\u8840\u7ba1\u6027\u6c34\u80bf\uff08hereditary angioedema\uff0c\u4e00\u79cd\u7f55\u89c1\u7684\u5371\u53ca\u751f\u547d\u7684\u9057\u4f20\u6027\u75be\u75c5\uff09\uff0c\u6bcf\u6708\u6807\u51c6\u7528\u836f\u91cf(16\u5242)\uff0c\u6807\u4ef7\u662f44140\u7f8e\u5143\u3002\u636e\u6089\uff0cShire\u516c\u53f8\u63d0\u4f9bOnePath Co-Pay Assistance Program\u63f4\u52a9\u8ba1\u5212\uff0c\u6709\u5546\u4e1a\u4fdd\u9669\u7684\u60a3\u8005\u53ef\u83b7\u5f97\u4f4e\u81f30\u7f8e\u5143\u7684\u4ef7\u683c\u3002<\/span><\/p>\n

 <\/span><\/p>\n

5\u3001Takhzyro  44140\u7f8e\u5143\/\u6708<\/span><\/p>\n

 <\/span><\/p>\n

Takhzyro\u4ee5 \u6807\u4ef744140\u7f8e\u5143\/\u6708\u4f4d\u5c45\u7b2c\u4e94\uff0c\u540c\u6837\u4e3aShire\u516c\u53f8\u7814\u53d1\uff0c\u5e76\u4e8e2018\u5e748\u6708\u83b7FDA\u6279\u51c6\u6cbb\u7597\u9057\u4f20\u6027\u8840\u7ba1\u6027\u6c34\u80bf\uff08hereditary angioedema\uff09\u3002\u60a3\u8005\u901a\u5e38\u6bcf\u6708\u9700\u8981\u6ce8\u5c04\u4e24\u5242\uff0c\u6bcf\u5242\u6807\u4ef722070\u7f8e\u5143\u3002<\/span><\/p>\n

 <\/span><\/p>\n

6\u3001Chenodal  42570\u7f8e\u5143\/\u6708<\/span><\/p>\n

 <\/span><\/p>\n

\u8be5\u836f\u7531Retrophin\u516c\u53f8\u9500\u552e\uff0c\u662f\u4e00\u79cd\u6cbb\u7597\u80c6\u7ed3\u77f3\u7684\u836f\u7269\u3002Chenodal\u7684\u6bcf\u6708\u8d39\u7528\u662f42,570\u7f8e\u5143\uff08\u6bcf\u7247\u836f\u4ef7\u4e3a473\u7f8e\u5143\uff0c\u60a3\u8005\u901a\u5e38\u9700\u8981\u670d\u752890\u7247\uff09\u3002<\/span><\/p>\n

 <\/span><\/p>\n

\u636e\u6089\uff0cChenodal\u7684\u4ef7\u683c\u57282014\u5e74\u589e\u957f\u4e865\u500d\uff0c\u5176\u80cc\u540e\u64cd\u7eb5\u8005\u662f\u4f7fDaraprim\u4ef7\u683c\u66b4\u6da8\u7684Martin Shkreli\u3002\u8be5\u836f\u867d\u5df2\u8fc7\u4e13\u5229\u4fdd\u62a4\u671f\uff0c\u4f46\u7531\u4e8e\u201c\u5c01\u95ed\u5206\u9500\u7cfb\u7edf\u201d\u7684\u4fdd\u62a4\uff0c\u5176\u5b83\u5236\u836f\u4f01\u4e1a\u7814\u53d1\u4ea6\u4e0d\u80fd\u751f\u4ea7\u8fd9\u79cd\u836f\u7269\u3002<\/span><\/p>\n

 <\/span><\/p>\n

7\u3001Juxtapidl  40671\u7f8e\u5143\/\u6708<\/span><\/p>\n

 <\/span><\/p>\n

Juxtapid\u7531Aegerion\u5236\u836f\u516c\u53f8\u751f\u4ea7\uff0c\u9002\u7528\u4e8e\u6cbb\u7597\u7eaf\u5408\u5b50\u5bb6\u65cf\u6027\u9ad8\u80c6\u56fa\u9187\u8840\u75c7\uff08Homozygous FamilialHypercholesterolemia\uff0c\u4e00\u79cd\u7531\u57fa\u56e0\u7a81\u53d8\u5bfc\u81f4\u7684\u5fc3\u8840\u7ba1\u75be\u75c5\uff09\uff0c\u901a\u5e38\u9700\u60a3\u8005\u6bcf\u6708\u670d\u752828\u7c92\u836f\u3002<\/span><\/p>\n

 <\/span><\/p>\n

8\u3001H.P. Acthar  38892\u7f8e\u5143\/\u6708<\/span><\/p>\n

 <\/span><\/p>\n

\u8be5\u836f\u7531Malnkkrdt\u5236\u836f\u516c\u53f8\u751f\u4ea7\uff0c\u9002\u7528\u4e8e\u591a\u79cd\u75be\u75c5\uff0c\u5305\u62ec\u7c7b\u98ce\u6e7f\u6027\u5173\u8282\u708e\u3001\u591a\u53d1\u6027\u786c\u5316\u75c7\u3001\u5a74\u513f\u75c9\u631b\u3001\u773c\u79d1\u75be\u75c5\u3001\u94f6\u5c51\u75c5\u6027\u5173\u8282\u708e\u7b49\u3002\u60a3\u8005\u901a\u5e38\u6bcf\u6708\u6ce8\u5c04\u4e00\u5242\u5373\u53ef\u3002<\/span><\/p>\n

 <\/span><\/p>\n

9\u3001Tegsedi  34600\u7f8e\u5143\/\u6708<\/span><\/p>\n

 <\/span><\/p>\n

Tegsedi\u57282018\u5e746\u6708\u83b7\u6279\u4e0a\u5e02\uff0c\u7528\u4e8e\u6cbb\u7597\u5bb6\u65cf\u6027\u6dc0\u7c89\u6837\u7269\u591a\u53d1\u6027\u795e\u7ecf\u75c5\uff08Hereditary Transthyretin Amyloidosis\uff0c\u4e00\u79cd\u7f55\u89c1\u75be\u75c5\uff09\u5f15\u8d77\u7684\u795e\u7ecf\u635f\u4f24\u3002\u60a3\u8005\u9700\u8981\u63a5\u53d7\u9002\u5f53\u7684\u7ed9\u836f\u6280\u672f\u57f9\u8bad\uff0c\u901a\u5e38\u6bcf\u6708\u4f7f\u7528\u91cf\u4e3a4\u5242\uff0c\u6bcf\u5242\u6807\u4ef7\u4e3a8650\u7f8e\u5143\u3002<\/span><\/p>\n

 <\/span><\/p>\n

10\u3001Ravicti  33572\u7f8e\u5143\/\u6708<\/span><\/p>\n

 <\/span><\/p>\n

\u8be5\u836f\u7531Horizon\u516c\u53f8\u751f\u4ea7\uff0c\u7528\u4e8e\u6cbb\u7597\u6cbb\u7597\u5c3f\u7d20\u5faa\u73af\u969c\u788d\uff08Urea Cycle Disorders\uff0c\u4e00\u79cd\u4f1a\u5bfc\u81f4\u8840\u6db2\u4e2d\u6c28\u542b\u91cf\u8fc7\u9ad8\u7684\u57fa\u56e0\u75be\u75c5\uff09\u3002\u9002\u7528\u4eba\u7fa4\u53ef\u4e3a2\u4e2a\u6708\u4ee5\u4e0a\u7684\u513f\u7ae5\uff0c\u60a3\u8005\u901a\u5e38\u6bcf\u4e2a\u6708\u9700\u8981\u4f7f\u75287\u74f6\u3002<\/span><\/p>\n

 <\/span><\/p>\n

11\u3001Vitrakvi  32800\u7f8e\u5143\/\u6708<\/span><\/p>\n

 <\/span><\/p>\n

\u8be5\u836f\u4e8e2018\u5e7411\u670826\u65e5\u83b7FDA\u6279\u51c6\u4e0a\u5e02\uff0c\u4e3b\u8981\u7528\u4e8e\u6cbb\u7597\u60a3\u6709NTRK\u57fa\u56e0\u878d\u5408\u7684\u5c40\u90e8\u665a\u671f\u6216\u8f6c\u79fb\u6027\u5b9e\u4f53\u7624\u7684\u6210\u4eba\u548c\u513f\u7ae5\u60a3\u8005\uff0c\u800c\u4e14\u5b83\u7684\u6709\u6548\u7387\u9ad8\u8fbe75%\uff0c\u662fFDA\u6279\u51c6\u7684\u7b2c\u4e00\u4e2a\u4e0d\u533a\u5206\u80bf\u7624\u6765\u6e90\u7528\u4e8e\u521d\u59cb\u6cbb\u7597\u7684\u65b9\u6cd5\u3002<\/span><\/p>\n

 <\/span><\/p>\n

\u62dc\u8033\u66fe\u8868\u793a\uff0c\u53e3\u670d\u80f6\u56ca\u5f62\u5f0f\u7684Larotrectinib\u91c7\u8d2d\u4ef7\u683c\u4e3a\u6bcf\u670832,800\u7f8e\u5143\uff0830\u5929100mg\uff09\uff0c\u6bcf\u5e74\u53ef\u80fd\u82b1\u8d39393600\u7f8e\u5143\uff08\u7ea6270\u591a\u4e07\u4eba\u6c11\u5e01\uff09\uff0c\u800c\u67d0\u4e9b\u513f\u7ae5\u60a3\u8005\u7684\u53e3\u670d\u6db2\u4f53\u5236\u5242\u4e3a\u6bcf\u67081.1\u4e07\u7f8e\u5143\u3002\u8fd9\u6837\u7684\u5929\u4ef7\u5bf9\u7f55\u89c1\u75c5\u60a3\u8005\u6765\u8bb2\uff0c\u4e0d\u662f\u5403\u6389\u4e00\u5957\u623f\u5b50\u5c31\u80fd\u4e70\u5355\u3002\u9274\u4e8e\u6b64\uff0c\u62dc\u8033\u516c\u53f8\u4e5f\u8868\u793a\u8d44\u52a9\u7684\u6148\u5584\u673a\u6784\u5c06\u514d\u8d39\u63d0\u4f9b\u8be5\u836f\u7269\u3002<\/span><\/p>\n

 <\/span><\/p>\n

12\u3001Firazyr  32468\u7f8e\u5143\/\u6708<\/span><\/p>\n

 <\/span><\/p>\n

Firazyr\u662fShire\u516c\u53f8\u7b2c\u4e09\u6b3e\u4e0a\u699c\u7684\u836f\u7269\uff0c\u7528\u4e8e\u6cbb\u7597\u9057\u4f20\u6027\u8840\u7ba1\u6027\u6c34\u80bf\u3002\u7136\u800c\uff0c\u4e0e\u4e0a\u8ff0Cinryze\u6709\u6240\u4e0d\u540c\uff0cCinryze\u7528\u4e8e\u53d1\u4f5c\u524d\u7684\u80bf\u80c0\u9884\u9632\uff0c\u800cFirazyr\u7528\u4e8e\u53d1\u4f5c\u540e\u3002\u7531\u4e8e\u60a3\u8005\u5e73\u5747\u6bcf\u6708\u906d\u53d72-4\u6b21\u53d1\u4f5c\uff0c\u5927\u591a\u6570\u60a3\u8005\u6bcf\u6708\u9700\u75281\u76d2\uff083\u9488\uff09Firazyr\uff0c\u5373\u6cbb\u7597\u6210\u672c\u8fbe32468\u7f8e\u5143\u3002<\/span><\/p>\n

 <\/span><\/p>\n

13\u3001Cuprimine  31426\u7f8e\u5143\/\u6708<\/span><\/p>\n

 <\/span><\/p>\n

\u8be5\u836f\u662f\u699c\u5355\u4e2d\u201c\u8d44\u5386\u201d\u6700\u8001\u7684\u4e00\u6b3e\u836f\u7269\uff0c\u9002\u7528\u4e8e\u7531Wilson\u75be\u75c5\uff08Wilson\u2019s Disease\uff09\u6cbb\u7597\u5f15\u53d1\u7684\u94dc\u7d2f\u79ef\u3002\u60a3\u8005\u996d\u540e\u670d\u7528\u4e00\u7c92\u80f6\u56ca\uff0c\u6bcf\u7c92\u7684\u6807\u4ef7\u662f\u8d85\u8fc7261\u7f8e\u5143\u3002<\/span><\/p>\n

 <\/span><\/p>\n

14\u3001Sovaldi  28000\u7f8e\u5143\/\u6708<\/span><\/p>\n

 <\/span><\/p>\n

Sovaldi\u7531\u5409\u5229\u5fb7\u516c\u53f8\u751f\u4ea7\uff0c\u7528\u4e8e\u6cbb\u7597\u4e19\u809d\u3002\u60a3\u8005\u9700\u670d\u836f12\u5468\uff0c\u4ef7\u683c\u4e3a1000\u7f8e\u5143\u4e00\u7247\uff0c\u6bcf\u6708\u7684\u7528\u836f\u8d39\u7528\u4e3a28000\u7f8e\u5143\u3002<\/span><\/p>\n

 <\/span><\/p>\n

15\u3001Viekira Pak  27773\u7f8e\u5143\/\u6708<\/span><\/p>\n

 <\/span><\/p>\n

\u8be5\u836f\u7684\u4f18\u52bf\u5728\u4e8e\u6cbb\u7597\u7f8e\u56fd\u6700\u5e38\u89c1\u7684\u4e19\u809d\u7c7b\u578b\uff0c\u9002\u7528\u4eba\u7fa4\u5e7f\u6cdb\u3002\u4f46\u56e0\u5176\u5305\u88c5\u7531\u591a\u79cd\u836f\u7247\u7ec4\u6210\uff0c\u60a3\u8005\u670d\u7528\u4e0d\u65b9\u4fbf\uff0c\u52a0\u4e4b\u5e02\u573a\u6d8c\u5165\u8d8a\u6765\u8d8a\u591a\u7684\u809d\u708e\u836f\u7269\uff0cViekira Pak\u5df2\u7136\u4e0d\u518d\u662f\u6cbb\u7597\u4e19\u578b\u809d\u708e\u7684\u9996\u9009\u7528\u836f\u3002<\/span><\/p>\n

 <\/span><\/p>\n

16\u3001Viekira XR  27773\u7f8e\u5143\/\u6708<\/span><\/p>\n

 <\/span><\/p>\n

ViekiraXR\u662fViekira Pak\u7684\u7f13\u91ca\u5242\u578b\uff0c\u4e5f\u7528\u4e8e\u57fa\u56e01\u578b\u4e19\u809d\u60a3\u8005\u7684\u6cbb\u7597\uff0c\u4e0eViekira Pak\u4e0d\u540c\u7684\u662f\uff0c\u60a3\u8005\u6bcf\u5929\u53ea\u9700\u670d\u7528\u4e00\u7247\u5373\u53ef\u3002\u800c\u540e\uff0c\u827e\u4f2f\u7ef4\u63a8\u51fa\u4e86Mavyret\uff0c\u53ef\u57288\u5468\u5185\u6cbb\u6108\u809d\u708e\uff0c\u4f4d\u5c45\u672c\u6b21\u6392\u884c\u699c\u7684\u7b2c\u4e8c\u4f4d\u3002\u636e\u827e\u4f2f\u7ef42018\u5e74\u8d22\u62a5\uff0cMavyret\u5168\u5e74\u521b\u653634.38\u4ebf\u7f8e\u5143\u3002<\/span><\/p>\n

 <\/span><\/p>\n

 <\/span><\/p>\n

17\u3001Orfadin  27247\u7f8e\u5143\/\u6708<\/span><\/p>\n

 <\/span><\/p>\n

Orfadin\u9002\u7528\u4e8e1\u578b\u9057\u4f20\u6027\u916a\u6c28\u9178\u8840\u75c7\uff08HT-1\uff0c\u4e00\u79cd\u7531\u916a\u6c28\u9178\u4ee3\u8c22\u969c\u788d\u6240\u81f4\u7684\u7f55\u89c1\u9057\u4f20\u6027\u75be\u75c5\uff09\u3002\u8be5\u836f\u7684\u4f7f\u7528\u5242\u91cf\u56e0\u4eba\u800c\u5f02\uff0c\u4f46\u5927\u591a\u6570\u60a3\u8005\u6bcf\u6708\u9700\u670d\u752830\u7c9220\u6beb\u514b\u7684\u80f6\u56ca\uff0c\u6bcf\u7c92\u5b9a\u4ef7\u4e3a908\u7f8e\u5143\u3002<\/span><\/p>\n

 <\/span><\/p>\n

18\u3001Tibsovo 26115\u7f8e\u5143\/\u6708<\/span><\/p>\n

 <\/span><\/p>\n

Tibsovo\u7531Agios Pharmaceuticals\u7814\u53d1\uff0c\u4e8e2018\u5e747\u6708\u83b7\u6279\u4e0a\u5e02\uff0c\u662f\u9996\u4e2a\u9488\u5bf9IDH1\u9176\u7684\u9776\u5411\u6291\u5236\u5242\uff0c\u9002\u7528\u4e8e\u6cbb\u7597\u7279\u5b9a\u57fa\u56e0\u7a81\u53d8\u60a3\u8005\u7684\u6025\u6027\u9ad3\u6027\u767d\u8840\u75c5\uff08Acute Myeloid Leukemia\uff09\u7684\u836f\u7269\u3002<\/span><\/p>\n

 <\/span><\/p>\n

19\u3001Cerdelga  26000\u7f8e\u5143\/\u6708<\/span><\/p>\n

 <\/span><\/p>\n

Cerdelga\u662f\u7531\u8d5b\u8bfa\u83f2\u65d7\u4e0b\u5b50\u516c\u53f8\u5065\u8d5e(Genzyme)\u7814\u53d1\uff0c\u7528\u4f5c\u7279\u5b9a1\u578b\u6208\u8c22\u75c5(Gaucher disease)\u6210\u4eba\u60a3\u8005\u552f\u4e00\u7684\u4e00\u7ebf\u53e3\u670d\u7597\u6cd5\uff0c\u662f\u7ee7Cerezyme(\u6ce8\u5c04\u7528\u4f0a\u7c73\u82f7\u9176\uff0cimiglucerase)\u7684\u53e6\u4e00\u6b3e\u6cbb\u7597\u6208\u8c22\u75c5\u7684\u53e3\u670d\u5236\u5242\u3002<\/span><\/p>\n

 <\/span><\/p>\n

\u636e\u6089\uff0cCerezyme\u66fe\u662f\u5168\u7403\u6700\u8d35\u7684\u836f\uff0c\u60a3\u8005\u6bcf\u5e74\u7684\u6cbb\u7597\u6210\u672c\u9ad8\u8fbe30\u4e07\u7f8e\u5143\u3002\u62e5\u6709\u540c\u6837\u9002\u5e94\u75c7\u7684Cerdelga\u4e0e\u524d\u8005\u6807\u4ef7\u76f8\u5dee\u65e0\u51e0\u3002\u800c\u590f\u5c14(Shire)\u7684Vpriv\u548c\u8f89\u745e(Pfizer)\u7684Elelyso\u4e24\u6b3e\u7528\u4e8e\u6cbb\u7597\u6208\u8c22\u75c5\u7684\u6ce8\u5c04\u6db2\u6807\u4ef7\u4e0d\u5982Cerdelga\u9ad8\u3002<\/span><\/p>\n

 <\/span><\/p>\n

20\u3001Remodulin  25466\u7f8e\u5143\/\u6708<\/span><\/p>\n

 <\/span><\/p>\n

Remodulin\u7531United Therapeutics\u7814\u53d1\uff0c\u4ee5\u6807\u4ef725466\u7f8e\u5143\/\u6708\u6392\u540d\u7b2c\u4e8c\u5341\u4f4d\u3002\u8be5\u836f\u7528\u4e8e\u6cbb\u7597\u80ba\u52a8\u8109\u9ad8\u538b\u3002\u60a3\u8005\u901a\u5e38\u9700\u8981\u6bcf\u6708\u6ce8\u5c042\u5242\uff0c\u6bcf\u5242\u5b9a\u4ef712733\u7f8e\u5143\u3002<\/span><\/p>\n


<\/p>\n

\u4f1a\u8bae\u901a\u77e5\uff1a<\/span><\/strong>\u533b\u4fdd\u6539\u9769\u3001\u5408\u7406\u7528\u836f\u3001\u4e34\u5e8a\u8bc4\u4ef7\u4e0b\uff0c<\/span>\u533b\u751f\u6743\u529b\u8fd8\u6709\u591a\u5927\uff1f<\/span>\u836f\u4f01\u600e\u6837\u7cbe\u51c6\u628a\u63e1\u533b\u751f\u53ca\u4e34\u5e8a\u79d1\u5ba4\u7684\u975e\u4e34\u5e8a\u9700\u6c42\uff0c<\/span>\u600e\u6837\u505a\u597d\u836f\u5b66\u90e8\/\u836f\u5242\u79d1\u5b66\u672f\u63a8\u5e7f<\/span>\uff0c<\/span>\u5982\u4f55\u901a\u8fc7\u884c\u653f\u79d1\u5ba4\u52a9\u529b\u516c\u7acb\u533b\u9662\u6539\u9769\uff1f<\/span>\u65b0\u5f62\u52bf\u4e0b\uff0c<\/span>\u836f\u4f01\u4e34\u5e8aKA\u600e\u4e48\u505a\uff1f<\/span>6\u670829\u65e5-30\u65e5\uff0c\u6210\u90fd\uff0c\u50cf\u9662\u957f\u4e00\u6837\u601d\u8003\u2122\uff1a\u533b\u9662\u975e\u4e34\u5e8a\u90e8\u95e8\u8425\u9500\u7814\u4fee\u73ed\uff0c\u67e5\u9605\u8be6\u60c5<\/strong><\/a><\/span><\/p>\n

\"\u6700\u8d35\u836f\u7269\u76d8\u70b9\"<\/span><\/p>\n

\"\u6700\u8d35\u836f\u7269\u76d8\u70b9\"<\/span><\/p>\n


<\/p>\n

\"\u6700\u8d35\u836f\u7269\u76d8\u70b9\"<\/p>\n<\/p><\/div>\n","protected":false},"excerpt":{"rendered":"

\u258d\u6765\u6e90\uff1a\u8d5b\u67cf\u84dd    \u6574\u7406\uff1a\u534a\u590f   \u8fd1\u65e5\uff0c\u7f8e\u56fdFDA\u6279\u51c6\u4e86\u8bfa\u534e\u516c\u53f8\u7528 […]<\/p>\n","protected":false},"author":1,"featured_media":117317,"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\/117316"}],"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=117316"}],"version-history":[{"count":1,"href":"https:\/\/www.05921.net\/index.php\/wp-json\/wp\/v2\/posts\/117316\/revisions"}],"predecessor-version":[{"id":117322,"href":"https:\/\/www.05921.net\/index.php\/wp-json\/wp\/v2\/posts\/117316\/revisions\/117322"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.05921.net\/index.php\/wp-json\/wp\/v2\/media\/117317"}],"wp:attachment":[{"href":"https:\/\/www.05921.net\/index.php\/wp-json\/wp\/v2\/media?parent=117316"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.05921.net\/index.php\/wp-json\/wp\/v2\/categories?post=117316"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.05921.net\/index.php\/wp-json\/wp\/v2\/tags?post=117316"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}