{"id":55309,"date":"2018-09-26T01:00:00","date_gmt":"2018-09-30T02:03:35","guid":{"rendered":"http:\/\/www.05921.net\/index.php\/2018\/09\/30\/%e4%b8%8d%e6%9c%8d%e5%9f%ba%e5%9b%a0%e6%9d%a5%e6%88%98%ef%bc%81%e5%b0%8f%e5%88%86%e5%ad%90%e6%96%b0%e8%8d%af%e8%bf%9b%e4%b8%89%e6%9c%9f%e4%b8%b4%e5%ba%8a\/"},"modified":"2018-09-30T10:03:37","modified_gmt":"2018-09-30T02:03:37","slug":"%e4%b8%8d%e6%9c%8d%e5%9f%ba%e5%9b%a0%e6%9d%a5%e6%88%98%ef%bc%81%e5%b0%8f%e5%88%86%e5%ad%90%e6%96%b0%e8%8d%af%e8%bf%9b%e4%b8%89%e6%9c%9f%e4%b8%b4%e5%ba%8a","status":"publish","type":"post","link":"https:\/\/www.05921.net\/index.php\/55309\/","title":{"rendered":"\u4e0d\u670d\u57fa\u56e0\u6765\u6218\uff01\u5c0f\u5206\u5b50\u65b0\u836f\u8fdb\u4e09\u671f\u4e34\u5e8a"},"content":{"rendered":"
\n

\u79cb\u5b63\uff0c\u662f\u6536\u83b7\u7684\u5b63\u8282\u3002<\/span><\/p>\n


<\/p>\n

\u6628\u5929\u7684Sarepta\u516c\u53f8\u7684\u57fa\u56e0\u7597\u6cd5\u4e34\u5e8a\u8bd5\u9a8c\u518d\u5f00\uff0c\u4eca\u5929\uff0c\u53e6\u4e00\u5bb6\u516c\u53f8\u7684\u53e3\u670d\u65b0\u836f\u4e5f\u8fdb\u5165\u6700\u540e\u7684\u8bd5\u9a8c\u3002<\/span><\/p>\n


<\/p>\n

\u4f4d\u4e8e\u6ce2\u58eb\u987f\u9644\u8fd1\u5251\u6865\u5e02\u7684Catabasis\u516c\u53f8\uff0c\u4e13\u6ce8\u808c\u8425\u517b\u4e0d\u826f\u548c\u80ba\u7ea4\u7ef4\u5316\u7684\u65b0\u836f\u7814\u53d1\uff0c\u516c\u53f8\u521a\u521a\u5ba3\u5e03\uff0cCAT-1004\uff0cEdasalonexent \u8fdb\u5165\u7b2c\u4e09\u671f\u4e34\u5e8a\u8bd5\u9a8c\u3002<\/span><\/p>\n


<\/p>\n

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<\/p>\n

Edasalonexent\u7684\u4f5c\u7528\u673a\u7406\u662f\uff0c\u6291\u5236<\/span>NF-Kb\uff08Kb\u6838\u9178\u56e0\u5b50\uff09<\/span>\uff0cKb\u6838\u9178\u56e0\u5b50\u662f\u6fc0\u6d3b\u708e\u75c7\u57fa\u56e0\u8868\u8fbe\u7684\u4e00\u4e2a\u8f6c\u5f55\u8c03\u8282\u5668, \u5b83\u662f\u708e\u75c7\u53d1\u751f\u7684\u4fe1\u53f7\u3002<\/span><\/p>\n


<\/p>\n

\u901a\u8fc7\u6291\u5236\u8fd9\u4e2a\u56e0\u5b50\uff0c\u53ef\u4ee5<\/span>\u4fdd\u6301\u808c\u8089\u5065\u5eb7\uff0c\u5ef6\u7f13\u808c\u840e\u7f29\u7684\u8fdb\u5c55<\/span>\u3002<\/span><\/p>\n


<\/p>\n

\u5728\u51e0\u5929\u540e\u5f00\u59cb\u7684\u4e09\u671f\u8bd5\u9a8c\u4e2d\uff0c<\/span>\u516c\u53f8\u8ba1\u5212\u62db\u52df125\u540d4-7\u5c81\u7684DMD\u60a3\u8005<\/span>\uff0c\u8fd9\u4e2a<\/span>PolarisDMD<\/span><\/strong>\u7684\u8bd5\u9a8c\uff0c\u5c06\u5728\u7f8e\u56fd\u5f00\u59cb\uff0c\u7136\u540e\u5728\u660e\u5e74\u5e74\u521d\u5f00\u653e\u5176\u4ed6\u5728\u5176\u4ed6\u56fd\u5bb6\u7684\u62db\u52df\uff0c\u5305\u62ec\uff1a\u6fb3\u5927\u5229\u4e9a\uff0c\u52a0\u62ff\u5927\uff0c\u6b27\u6d32\u548c\u4ee5\u8272\u5217\u7b49\u3002<\/span><\/p>\n


<\/span><\/p>\n

\u8ba1\u5212\u5728\u5168\u90e840\u4e2a\u4e34\u5e8a\u8bd5\u9a8c\u5730\u70b9\u8fdb\u884c\u8fd9\u4e2a\u4e09\u671f\u4e34\u5e8a\u8bd5\u9a8c\u3002<\/span><\/p>\n

\"\u4e0d\u670d\u57fa\u56e0\u6765\u6218\uff01\u5c0f\u5206\u5b50\u65b0\u836f\u8fdb\u4e09\u671f\u4e34\u5e8a\"<\/p>\n

<\/span>
<\/p>\n

\u5728\u6b64\u524d\u8fdb\u884c\u7684\u4e8c\u671f\u4e34\u5e8a\u8bd5\u9a8c\uff08MoveDMD\uff09\u4e2d\uff0c4-7\u5c81\u7684<\/span>\u4efb\u4f55\u7a81\u53d8\u7684DMD\u7537\u7ae5<\/span>\u53c2\u4e0e\u4e86<\/span>\u6cbb\u7597\uff0c\u4ee5\u8bc4\u4f30edasalonexent\u7684\u5b89\u5168\u6027\u548c\u6709\u6548\u6027\u3002<\/span>
<\/p>\n


<\/p>\n

\u7ed3\u679c\u663e\u793a\uff0c\u4e0e\u5b89\u6170\u5242\u7ec4\u76f8\u6bd4\uff0c\u572860\u5468\uff08\u4e00\u5e74\uff09\u7684\u6cbb\u7597\u4e2d\uff0c\u8fd9\u4e2a\u65b0\u836f\u53ef\u4ee5\u4fdd\u5b58\u808c\u8089\u529f\u80fd\uff0c\u660e\u663e\u5ef6\u7f13DMD\u75be\u75c5\u8fdb\u7a0b\uff0c\u516c\u53f8\u8868\u793a\u3002<\/span><\/p>\n


<\/p>\n

\u5728\u968f\u540e\u7684\u89c2\u5bdf\u4e2d\uff0c\u670d\u7528100\u6beb\u514b\/\u516c\u65a4\/\u65e5\u7684\u6cbb\u7597\u540e, \u4e0e\u5728\u63a5\u53d7\u6cbb\u7597\u524d\u7684\u63a7\u5236\u671f\u7684\u53d8\u5316\u7387\u76f8\u6bd4, CAT-1004\u53ef\u4ee5\u6301\u7eed\u6539\u8fdb\u808c\u8089\u529f\u80fd\u7684\u6240\u6709\u56db\u9879\u8bc4\u4f30\u3002<\/span><\/p>\n


<\/p>\n

\u8fd9\u4e9b\u8bc4\u4f30\u662f\uff1a\u5317\u6781\u661f\u6d4b\u8bd5\uff1b\u7ad9\u7acb\u6240\u9700\u65f6\u95f4\uff1b4\u53f0\u9636\u6500\u722c\uff1b10\u7c73\u8d70\/\u8dd1\u3002<\/span><\/p>\n


<\/p>\n

\u8fd9\u9879\u8bd5\u9a8c\u8fd8\u53d1\u73b0\uff0c edasalonexent \u6709<\/span>\u663e\u8457\u7684\u957f\u671f\u51cf\u5c11\u808c\u8089\u9176\u548c c-\u53cd\u5e94\u86cb\u767d (CRP)\u7684\u4f5c\u7528<\/span>\u3002\u7531\u4e8e\u8010\u53d7\u6027\u826f\u597d, \u5728\u8bd5\u9a8c\u4e2d\u6ca1\u6709\u89c2\u5bdf\u5230\u5b89\u5168\u4fe1\u53f7\u4e8b\u4ef6\uff0c\u652f\u6301\u957f\u671f\u670d\u7528\u548c<\/span>\u6cbb\u7597\u3002<\/span><\/p>\n


<\/span><\/p>\n

Catabasis\u516c\u53f8\u8fd8\u5728\u7814\u53d1CAT-1004\u6cbb\u7597\u8d1d\u514b\u808c\u8425\u517b\u4e0d\u826f\uff08BMD\uff09\u3002\u516c\u53f8\u7684\u53e6\u4e00\u4e2a\u65b0\u836f\uff0cCAT-5571\u7814\u7a76\u6cbb\u7597\u80ba\u7ea4\u7ef4\u5316\uff08CF\uff09\u3002<\/span><\/p>\n


<\/p>\n

\"\u4e0d\u670d\u57fa\u56e0\u6765\u6218\uff01\u5c0f\u5206\u5b50\u65b0\u836f\u8fdb\u4e09\u671f\u4e34\u5e8a\"<\/p>\n


<\/span><\/p>\n

“\u5728 MoveDMD \u8bd5\u9a8c\u4e2d, edasalonexent \u6cbb\u7597\u663e\u793a\u80fd\u7a33\u5b9a\u808c\u8089\u529f\u80fd\uff0c\u5177\u6709\u826f\u597d\u7684\u5b89\u5168\u6027, \u60a3\u8005\u548c\u5bb6\u5ead\u90fd\u6e34\u671b\u5bf9\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u7684\u65b0\u7597\u6cd5, \u8fd9\u4e2a\u836f\u5728\u4e34\u5e8a\u4e0a\u663e\u793a\u4e86\u80fd\u5ef6\u7f13\u75be\u75c5\u8fdb\u5c55.” \u8bd5\u9a8c\u603b\u8d1f\u8d23\u4eba\uff0cNemours\u513f\u7ae5\u533b\u9662\u7cfb\u7edf\u795e\u7ecf\u79d1\u533b\u751f\u7406\u67e5\u5fb7 \u82ac\u514b\uff08Richard Finkle)\u5728\u58f0\u660e\u4e2d\u8868\u793a\u3002<\/span><\/p>\n


<\/p>\n

“\u6211\u4eec\u70ed\u5207\u5730\u770b\u5230 edasalonexent\u8fdb\u51653\u671f PolarisDMD \u8bd5\u9a8c, \u4f5c\u4e3a\u4e00\u79cd\u53e3\u670d\u6cbb\u7597, \u53ef\u4ee5\u6cbb\u7597\u6240\u6709\u53d7\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u5f71\u54cd\u7684\u4eba\u3002\u201cDMD\u60a3\u8005\u7ec4\u7ec7\uff0cPPMD\u7684\u521b\u59cb\u4eba\u548c\u603b\u88c1Pat Furlong\u5728\u58f0\u660e\u4e2d\u8868\u793a\u3002<\/span><\/p>\n


<\/p>\n

\u8bd5\u9a8c\u7684\u8bbe\u8ba1\u4e0e\u98df\u54c1\u836f\u54c1\u76d1\u7ba1\u5c40\uff08FDA\uff09\u7684\u8ba8\u8bba\u540e\u516c\u5e03\u3002\u7ed3\u679c\u8ba1\u5212\u5728\u8fd1\u4e24\u5e74\u540e\uff0c2020\u5e74\u7b2c\u4e8c\u5b63\u5ea6\u53d1\u5e03\u3002<\/span><\/p>\n


<\/p>\n

CAT-1004\u5df2\u7ecf\u83b7\u5f97\u5b64\u513f\u836f\uff0c\u5feb\u901f\u5ba1\u8bc4\u548c\u7a00\u5c11\u513f\u7ae5\u7528\u836f\u6307\u5b9a\uff0c\u5728\u6b27\u6d32\uff0c\u6b27\u76df\u6388\u4e88\u5b64\u513f\u836f\u8d44\u683c\u3002<\/span><\/p>\n


<\/p>\n

\u4f60\u5bf9\u8fd9\u4e2a\u53e3\u670d\u5c0f\u5206\u5b50\u65b0\u836f\u600e\u4e48\u770b\uff1f<\/em><\/span><\/p>\n

\u8bf7\u7559\u8a00\u5206\u4eab\u770b\u6cd5<\/em><\/span>\u3002<\/span><\/p>\n


<\/p>\n

\u4f60\u53ef\u80fd\u559c\u6b22\uff1a<\/span><\/p>\n

\u57fa\u56e0\u6cbb\u7597\u808c\u840e\u7f29 \u8bd5\u9a8c\u518d\u5f00<\/span><\/a>
<\/p>\n

DMD\u5468\u62a5\uff1a\u65b0\u836f\u4e2d\u56fd\u4e34\u5e8a\u8bd5\u9a8c \u5355\u6297\u57fa\u56e0\u6709\u559c\u6709\u5fe7<\/span><\/a><\/p>\n

\u4e34\u95e8\u4e00\u811a \u98de\u4e86\uff01DMD\u65b0\u836f\u53d7\u632b \u836f\u4f01\u8df3\u6c34<\/span><\/a>
<\/p>\n

4.5\u4ebf\uff01\u5b89\u65af\u6cf0\u6765\u8981\u731b\u8e22\u7f55\u89c1\u75c5DMD<\/span><\/a>
<\/p>\n


<\/p>\n

\u670d\u52a1\u65b0\u836f\u7814\u53d1 \u628a\u63e1\u6295\u8d44\u8d8b\u52bf<\/span><\/em><\/span><\/p>\n

\u6bcf\u5929\u7528\u65b0\u8bf4\u8bdd \u51b3\u7b56\u4e0d\u53ef\u6216\u7f3a<\/span><\/em><\/span><\/p>\n

\u5065\u70b9\u5b50ihealth<\/em><\/strong>\u662f\u533b\u836f\u884c\u4e1a\u9886\u5148\u7684\u6295\u8d44\u987e\u95ee\u8d44\u8baf\u5e73\u53f0\uff0c\u4e13\u6ce8<\/em>\u7f55\u89c1\u75c5\uff0c<\/strong>\u751f\u7269\u7c7b\u4f3c\u836f\uff0c<\/strong><\/em>\u65e5\u97e9\u5370\u5ea6<\/strong><\/em>\u5065\u5eb7\u884c\u4e1a\u7684\u6700\u65b0\u52a8\u5411\u3002<\/em><\/span><\/p>\n

\u4f60\u4e5f\u53ef\u4ee5\u5728\u4efb\u4f55\u5065\u70b9\u5b50\u6587\u7ae0\u540e\u7559\u8a00\u3002\u8bf7\u7b80\u77ed\u8bf4\u660e\u5177\u4f53\u9700\u6c42\u3002\u6211\u4eec\u4e00\u822c\u572824\u5c0f\u65f6\u4e4b\u5185\u8054\u7cfb\u3002<\/em><\/span><\/p>\n

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

\u79cb\u5b63\uff0c\u662f\u6536\u83b7\u7684\u5b63\u8282\u3002 \u6628\u5929\u7684Sarepta\u516c\u53f8\u7684\u57fa\u56e0\u7597\u6cd5\u4e34\u5e8a\u8bd5\u9a8c\u518d\u5f00\uff0c\u4eca\u5929\uff0c\u53e6\u4e00\u5bb6\u516c\u53f8\u7684\u53e3\u670d\u65b0\u836f\u4e5f\u8fdb\u5165\u6700\u540e\u7684\u8bd5 […]<\/p>\n","protected":false},"author":1,"featured_media":55310,"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\/55309"}],"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=55309"}],"version-history":[{"count":1,"href":"https:\/\/www.05921.net\/index.php\/wp-json\/wp\/v2\/posts\/55309\/revisions"}],"predecessor-version":[{"id":55313,"href":"https:\/\/www.05921.net\/index.php\/wp-json\/wp\/v2\/posts\/55309\/revisions\/55313"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.05921.net\/index.php\/wp-json\/wp\/v2\/media\/55310"}],"wp:attachment":[{"href":"https:\/\/www.05921.net\/index.php\/wp-json\/wp\/v2\/media?parent=55309"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.05921.net\/index.php\/wp-json\/wp\/v2\/categories?post=55309"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.05921.net\/index.php\/wp-json\/wp\/v2\/tags?post=55309"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}