{"id":8368,"date":"2024-07-03T15:45:49","date_gmt":"2024-07-03T15:45:49","guid":{"rendered":"https:\/\/www.lgmd-info.org\/?page_id=8368"},"modified":"2026-04-21T17:51:39","modified_gmt":"2026-04-21T17:51:39","slug":"lgmd-milestones-a-decade-of-difference","status":"publish","type":"page","link":"https:\/\/www.lgmd-info.org\/zh\/knowledge-base\/lgmd-milestones-a-decade-of-difference\/","title":{"rendered":"LGMD Milestones – Over a Decade of Difference"},"content":{"rendered":"

\u63a2\u7d22\u5341\u5e74\u6765\u9632\u6cbb\u80a2\u8170\u808c\u8425\u517b\u4e0d\u826f\u75c7 (LGMD) \u7684\u91cd\u8981\u91cc\u7a0b\u7891\u3002\u4ece\u5f00\u521b\u6027\u7684\u57fa\u56e0\u6d4b\u5e8f\u548c\u5ba3\u4f20\u6d3b\u52a8\uff0c\u5230\u56fd\u9645\u4f1a\u8bae\u548c\u4e34\u5e8a\u8bd5\u9a8c\uff0c\u4e86\u89e3\u4e3a\u6539\u5584 LGMD \u60a3\u8005\u7684\u751f\u6d3b\u800c\u53d6\u5f97\u7684\u91cd\u5927\u8fdb\u5c55\u548c\u6b63\u5728\u8fdb\u884c\u7684\u52aa\u529b\u3002<\/h4><\/div><\/div><\/div><\/div><\/div>

2014<\/h2><\/div><\/div><\/div>
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2014 \u5e74\uff0c\u514d\u8d39 \u65b0\u4e00\u4ee3\u57fa\u56e0\u6d4b\u5e8f<\/strong> \u901a\u8fc7\u4e00\u9879 LGMD \u5ba3\u4f20\u8ba1\u5212\uff0c\u5411\u7f8e\u56fd\u60a3\u8005\u63d0\u4f9b\u4e86\u9488\u5bf9 LGMD\uff0835 \u4e2a\u57fa\u56e0\uff09\u7684\u57fa\u56e0\u68c0\u6d4b\uff0c\u4ece\u800c\u4f7f\u6570\u5343\u4eba\u83b7\u5f97\u4e86\u514d\u8d39\u68c0\u6d4b\u548c\u57fa\u56e0\u8bca\u65ad\u3002<\/p>\n<\/div>

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\"2014<\/span><\/div>

LGMD-Info.org\u3001<\/strong> \u7684 \"LGMD \u793e\u533a\u7684\u4fe1\u606f\u67a2\u7ebd\"<\/strong> \u521b\u5efa\u4e86\u3002<\/p>\n<\/div>

<\/div><\/div>
\"2014<\/span><\/div>

\"(\u300a\u4e16\u754c\u4eba\u6743\u5ba3\u8a00\u300b) \u7528\u4e8e LGMD \u7684\u77f3\u7070\u7eff<\/strong> \u5ba3\u4f20\u6d3b\u52a8\uff0c\u5e76\u5c06\u7ee7\u7eed\u6210\u4e3a\u6211\u4eec\u7684\u6807\u5fd7\u6027\u989c\u8272\u3002<\/p>\n<\/div>

<\/div><\/div>
\"2014<\/span><\/div>

\"(\u300a\u4e16\u754c\u4eba\u6743\u5ba3\u8a00\u300b) \u5e72\u6270\u7d20\u75c5\u4e34\u5e8a\u6548\u679c\u7814\u7a76<\/strong> (COS\uff09\uff0c\u6807\u5fd7\u7740\u56fd\u9645\u793e\u4f1a\u4e3a\u4e86\u89e3\u8be5\u75be\u75c5\u505a\u51fa\u4e86\u91cd\u5927\u52aa\u529b\u3002<\/p>\n<\/div><\/div><\/div><\/div><\/div>

2015<\/h2><\/div><\/div><\/div>
<\/div><\/div><\/div><\/div>
<\/div><\/div><\/div>
\"2015awarenessday\"<\/span><\/div>

\u7b2c\u4e00\u4e2a LGMD \u5ba3\u4f20\u65e5<\/strong><\/a> \u4e8e 2015 \u5e74 9 \u6708 30 \u65e5\u4e3e\u884c\u3002<\/p>\n<\/div>

<\/div><\/div>
\"2015<\/a><\/div><\/div>
\"2015<\/a><\/div><\/div>
\"2015<\/a><\/div><\/div>
\"2015<\/a><\/div><\/div>
<\/div><\/div><\/div>

\u7b2c\u4e00\u4e2a \u5dde\u516c\u544a<\/strong> \u6536\u5230\u4e86\u5ba3\u5e03 9 \u6708 30 \u65e5\u4e3a\u5a01\u65af\u5eb7\u661f\u5dde\u3001\u534e\u76db\u987f\u5dde\u3001\u5bc6\u82cf\u91cc\u5dde\u548c\u79d1\u7f57\u62c9\u591a\u5dde\u7684\u80a2\u8170\u808c\u8425\u517b\u4e0d\u826f\u75c7\u5ba3\u4f20\u65e5\u7684\u901a\u77e5\u3002<\/p>\n<\/div>

<\/div><\/div>
\"Jainfoundation\"<\/span><\/div>

\"(\u300a\u4e16\u754c\u4eba\u6743\u5ba3\u8a00\u300b) Dysferlin \u79d1\u5b66\u4f1a\u8bae<\/strong> \u7814\u7a76\u4eba\u5458\u3001\u4e34\u5e8a\u533b\u751f\u548c\u884c\u4e1a\u4e13\u5bb6\u9f50\u805a\u52a0\u62ff\u5927\u591a\u4f26\u591a\uff0c\u5206\u4eab\u7814\u7a76\u548c\u6cbb\u7597\u80a2\u8170\u808c\u8425\u517b\u4e0d\u826f\u75c7\uff08dysferlinopathy\uff09\u7684\u89c1\u89e3\u548c\u8fdb\u5c55\u3002<\/p>\n<\/div>

<\/div><\/div>

\"(\u300a\u4e16\u754c\u4eba\u6743\u5ba3\u8a00\u300b) 2B \u589e\u5f3a\u60a3\u8005\u6743\u80fd\u4f1a\u8bae<\/strong> \u5728\u5f97\u514b\u8428\u65af\u5dde\u8fbe\u62c9\u65af\u5e02\u4e3e\u884c\uff0c\u4e3a\u80a2\u4f53\u8170\u808c\u840e\u7f29\u75c7\u60a3\u8005\u63d0\u4f9b\u4e86\u4e00\u4e2a\u4ea4\u6d41\u3001\u5206\u4eab\u7ecf\u9a8c\u548c\u4ece\u8be5\u9886\u57df\u4e13\u5bb6\u90a3\u91cc\u83b7\u5f97\u5b9d\u8d35\u89c1\u89e3\u7684\u5e73\u53f0\u3002<\/p>\n<\/div><\/div><\/div><\/div><\/div>

2016<\/h2><\/div><\/div><\/div>
<\/div><\/div><\/div><\/div>
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\"2016<\/span><\/div>

2016 \u5e74 \u7b2c 219 \u5c4a ENMC Titinopathies \u56fd\u9645\u7814\u8ba8\u4f1a\uff1a<\/strong> \u5728\u8377\u5170\u6d77\u59c6\u65af\u514b\u5c14\u514b\uff08Heemskerk\uff09\u5efa\u7acb\u4e86\u4e00\u4e2a\u5173\u4e8e titin \u7a81\u53d8\u548c\u8868\u578b\u7684\u56fd\u9645\u6570\u636e\u5e93\u3002<\/p>\n<\/div>

<\/div><\/div>
\"2016<\/span><\/div>

\"(\u300a\u4e16\u754c\u4eba\u6743\u5ba3\u8a00\u300b) \u8428\u66fc\u838e-J-\u5e03\u62c9\u4f50\u57fa\u91d1\u4f1a<\/strong> \u66f4\u540d\u4e3a CureLGMD2i<\/strong><\/a>.\u8fd9\u4e00\u53d8\u5316\u53cd\u6620\u4e86\u57fa\u91d1\u4f1a\u81f4\u529b\u4e8e\u5bfb\u627e 2i \u578b\u80a2\u4f53\u8170\u808c\u840e\u7f29\u75c7\uff08LGMD2i\uff09\u7684\u6cbb\u7597\u65b9\u6cd5\u3002<\/p>\n<\/div>

CureLGMD2i \u548c LGMD2i \u7814\u7a76\u57fa\u91d1\u4e3a LGMD2I\/R9 \u7684\u9996\u6b21\u4e34\u5e8a\u8bd5\u9a8c\u63d0\u4f9b\u4e86\u652f\u6301\uff0c\u8be5\u8bd5\u9a8c\u540d\u4e3a \"PF-06252616\u5728LGMD2I\u975e\u5367\u5e8a\u60a3\u8005\u4e2d\u7684\u8bd5\u9a8c\"\uff0c\u300a\u7f8e\u56fd\u533b\u5b66\u6742\u5fd7\u300b\uff0c2011\u5e74\u7b2c2\u671f\u3002<\/strong> \u7531\u51ef\u745f\u7433-\u74e6\u683c\u7eb3\u535a\u58eb\u9886\u5bfc\u3002<\/p>\n<\/div>

<\/div><\/div>
\"2016<\/span><\/div>

Dysferlin \u6ce8\u518c\u8868<\/strong> \u63a8\u51fa\u4e86\u4e00\u4e2a\u65b0\u5e73\u53f0\uff0c\u4e13\u95e8\u9488\u5bf9\u7ecf\u57fa\u56e0\u8bc1\u5b9e\u7684\u94c1\u86cb\u767d\u5f02\u5e38\u75c5\u53d8\u60a3\u8005\u3002<\/p>\n<\/div>

<\/div><\/div>

\u53cc\u8f7d\u4f53\u57fa\u56e0\u7597\u6cd5<\/strong> \u7684\u65b0\u836f\u7533\u8bf7\u83b7\u5f97\u4e86\u7f8e\u56fd\u98df\u54c1\u53ca\u836f\u7269\u7ba1\u7406\u5c40\uff08FDA\uff09\u7684\u6279\u51c6\u3002<\/p>\n<\/div>

<\/div><\/div>

\u7b2c\u4e8c\u4e2a 2B \u589e\u5f3a\u60a3\u8005\u6743\u80fd\u4f1a\u8bae<\/strong> \u5728\u5f97\u514b\u8428\u65af\u5dde\u8fbe\u62c9\u65af\u4e3e\u884c\u3002<\/p>\n<\/div><\/div><\/div><\/div><\/div>

2017<\/h2><\/div><\/div><\/div>
<\/div><\/div><\/div><\/div>
<\/div><\/div><\/div>
\"2017myonexuslogo\"<\/span><\/div>

Myonexus Therapeutics<\/strong> \u6210\u7acb\u4e8e 2017 \u5e74\uff0c\u65e8\u5728\u5f00\u53d1\u6cbb\u7597\u80a2\u8170\u808c\u8425\u517b\u4e0d\u826f\u75c7\uff08LGMD\uff09\u4e94\u79cd\u4e9a\u578b\u7684\u57fa\u56e0\u7597\u6cd5\uff1aLGMD2B\/R2\u3001LGMD2C\/R5\u3001LGMD2D\/R5\u3001LGMD2E\/R4 \u548c LGMD2L\/R12\u3002\u8fd9\u5bb6\u5904\u4e8e\u4e34\u5e8a\u9636\u6bb5\u7684\u751f\u7269\u6280\u672f\u516c\u53f8\u4e0e\u5168\u7f8e\u513f\u7ae5\u533b\u9662\uff08Nationwide Children's Hospital\uff09\u5408\u4f5c\uff0c\u5229\u7528\u57fa\u4e8e AAVrh.74 \u8f7d\u4f53\u7cfb\u7edf\uff08AAVrh.74\uff09\u7684\u521b\u65b0\u57fa\u56e0\u6cbb\u7597\u5e73\u53f0\uff0c\u81f4\u529b\u4e8e\u4e3a\u8fd9\u4e9b\u5f62\u5f0f\u7684 LGMD \u521b\u9020\u6709\u53f2\u4ee5\u6765\u7b2c\u4e00\u79cd\u77eb\u6b63\u6cbb\u7597\u65b9\u6cd5\u3002Sarepta Therapeutics<\/a>) (\u808c\u8089\u840e\u7f29\u75c7\u65b0\u95fb<\/a>) (\u751f\u7269\u7a7a\u95f4<\/a>).<\/p>\n<\/div>

<\/div><\/div>

2017 \u5e74 3 \u6708 17-19 \u65e5\u5728\u8377\u5170\u7eb3\u4e39\u4e3e\u884c\u7684 ENMC \u7b2c 229 \u671f\u56fd\u9645\u7814\u8ba8\u4f1a\u5236\u5b9a\u4e86\u4e00\u9879 \u80a2\u8170\u808c\u8425\u517b\u4e0d\u826f\u75c7\uff08LGMDs\uff09\u7684\u65b0\u547d\u540d\u548c\u5206\u7c7b\u7cfb\u7edf<\/strong>.\u4e3a\u4e86\u66f4\u65b0 LGMD \u7684\u5b9a\u4e49\u5e76\u89e3\u51b3\u9690\u6027\u4e9a\u578b\u7f16\u53f7\u8fbe\u5230 Z\uff08LGMD 2Z\uff09\u7684\u95ee\u9898\uff0c\u6709\u5fc5\u8981\u8fdb\u884c\u4fee\u8ba2\u3002\u65b0\u7cfb\u7edf\u7684\u76ee\u7684\u662f\u4f7f LGMD \u6210\u4e3a\u4e00\u4e2a\u66f4\u52a0\u72ec\u7279\u7684\u5b9e\u4f53\uff0c\u5404\u4e9a\u578b\u4e4b\u95f4\u5177\u6709\u5171\u6027\u3002<\/p>\n<\/div>

<\/div><\/div>

2017 \u5e74 \u9996\u6b21\u9488\u5bf9\u94c1\u86cb\u767d\u969c\u788d\u75c7\u7684\u808c\u5185\uff08IM\uff09\u53cc\u8f7d\u4f53\u57fa\u56e0\u6cbb\u7597\u4e34\u5e8a\u8bd5\u9a8c<\/strong> \u542f\u52a8\uff0c\u6807\u5fd7\u7740\u5728\u6cbb\u7597\u8fd9\u79cd LGMD \u65b9\u9762\u53d6\u5f97\u4e86\u91cd\u5927\u8fdb\u5c55\u3002<\/p>\n<\/div><\/div><\/div><\/div><\/div>

2018<\/h2><\/div><\/div><\/div>
<\/div><\/div><\/div><\/div>
<\/div><\/div><\/div>
\"2018grasp\"<\/span><\/div>

2018 \u5e74 G<\/strong>\u9057\u4f20 R<\/strong>\u51b3\u8bae\u548c A<\/strong>\u8bc4\u4f30 S<\/strong>\u6a44\u6984\u5f62 P<\/strong>LGMD\u4e2d\u7684notypes (GRASP-LGMD)<\/strong> \u6210\u7acb\u8be5\u8054\u76df\u7684\u76ee\u7684\u662f\u52a0\u901f LGMDs \u7684\u6cbb\u7597\u5f00\u53d1\u3002<\/p>\n<\/div>

<\/div><\/div>
\"2018<\/span><\/div>

\"(\u300a\u4e16\u754c\u4eba\u6743\u5ba3\u8a00\u300b) LGMD2L \u57fa\u91d1\u4f1a<\/strong><\/a> \u6210\u7acb\u3002<\/p>\n<\/div>

<\/div><\/div>
\"2018<\/span><\/div>

\"(\u300a\u4e16\u754c\u4eba\u6743\u5ba3\u8a00\u300b) \u6cbb\u7597\u9499\u86cb\u767d\u9176\u8054\u76df 3<\/strong><\/a> \u5ba3\u5e03\u5176 \u57fa\u56e0\u6cbb\u7597\u8ba1\u5212<\/strong>\u8be5\u9879\u76ee\u65e8\u5728\u52a0\u901f\u5f00\u53d1\u6cbb\u7597 2A\/R1 \u578b\u8170\u808c\u8425\u517b\u4e0d\u826f\u75c7\uff08LGMD2A\/R1\uff09\u7684\u57fa\u56e0\u7597\u6cd5\u3002<\/p>\n<\/div>

<\/div><\/div>
\"2018lgmd2a<\/span><\/div>

\"(\u300a\u4e16\u754c\u4eba\u6743\u5ba3\u8a00\u300b) LGMD2A\/R1 \u79d1\u5b66\u4f1a\u8bae<\/strong> \u5728\u5f17\u5409\u5c3c\u4e9a\u5dde\u963f\u7075\u987f\u4e3e\u884c\uff0c\u4e13\u5bb6\u548c\u7814\u7a76\u4eba\u5458\u6c47\u805a\u4e00\u5802\uff0c\u5171\u540c\u63a2\u8ba8\u9632\u6cbb 2A\/R1 \u578b\u80a2\u8170\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684\u6700\u65b0\u8fdb\u5c55\u548c\u7b56\u7565\u3002<\/p>\n<\/div>

<\/div><\/div>
\"2018<\/span><\/div>

\"(\u300a\u4e16\u754c\u4eba\u6743\u5ba3\u8a00\u300b) LGMD-1D DNAJB6 \u57fa\u91d1\u4f1a<\/strong><\/a> \u6210\u7acb\u3002<\/p>\n<\/div>

<\/div><\/div>

Sarepta Therapeutics \u516c\u53f8\u8fdb\u884c\u4e86\u9996\u6b21 \u57fa\u56e0\u6cbb\u7597\u7684\u5168\u8eab\u7ed9\u836f<\/strong> \u6cbb\u7597\u80a2\u8170\u808c\u8425\u517b\u4e0d\u826f\u75c7\uff0c\u7279\u522b\u662f\u9488\u5bf9\u03b2-\u808c\u7cd6\u86cb\u767d\u57fa\u56e0\uff08LGMD2E\uff09\u3002<\/p>\n<\/div><\/div><\/div><\/div><\/div>

2019<\/h2><\/div><\/div><\/div>
<\/div><\/div><\/div><\/div>
<\/div><\/div><\/div>

2019 \u5e74 \u56fd\u9645 LGMD \u793e\u533a\u54a8\u8be2\u59d4\u5458\u4f1a (CAB)<\/strong> \u6210\u7acb\u8be5\u7ec4\u7ec7\u7684\u76ee\u7684\u662f\u4e3a\u5168\u7403\u80a2\u8170\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7fa4\u4f53\u63d0\u4f9b\u4e00\u4e2a\u7edf\u4e00\u7684\u58f0\u97f3\uff0c\u5e76\u4fc3\u8fdb\u60a3\u8005\u3001\u7814\u7a76\u4eba\u5458\u548c\u533b\u7597\u670d\u52a1\u63d0\u4f9b\u8005\u4e4b\u95f4\u7684\u5408\u4f5c\u3002<\/p>\n<\/div>

<\/div><\/div>
\"2019limeemoji\"<\/span><\/div>

\"(\u300a\u4e16\u754c\u4eba\u6743\u5ba3\u8a00\u300b) \"LIMEmoji\u6311\u6218\"<\/strong> \u4ee5\u5e2e\u52a9\u63d0\u9ad8\u5bf9 LGMD \u7684\u8ba4\u8bc6\u3002<\/p>\n<\/div>

<\/div><\/div>

\u542f\u52a8\u4e86\u4e00\u9879\u4e34\u5e8a\u7814\u7a76\uff0c\u4ee5\u8bc4\u4f30 \u6bcf\u5468\u670d\u7528\u4e00\u6b21\u7c7b\u56fa\u9187\u7684\u5f00\u653e\u6807\u7b7e\u5b89\u5168\u6027\u548c\u7597\u6548<\/strong> \u6cbb\u7597\u80a2\u8170\u808c\u8425\u517b\u4e0d\u826f\u75c7\uff08LGMD\uff09\u60a3\u8005\u3002<\/p>\n<\/div>

<\/div><\/div>
\"2019conference\"<\/span><\/div>

\u7b2c\u4e00\u4e2a \u56fd\u9645 LGMD \u4f1a\u8bae<\/strong> \u5728\u4f0a\u5229\u8bfa\u4f0a\u5dde\u829d\u52a0\u54e5\u4e3e\u884c\uff0c\u6765\u81ea\u4e16\u754c\u5404\u5730\u7684\u60a3\u8005\u3001\u5bb6\u5c5e\u3001\u7814\u7a76\u4eba\u5458\u548c\u4e34\u5e8a\u533b\u751f\u6c47\u805a\u4e00\u5802\uff0c\u5206\u4eab\u77e5\u8bc6\uff0c\u4fc3\u8fdb\u5bf9\u80a2\u4f53\u8170\u808c\u840e\u7f29\u75c7 (LGMD) \u7684\u4e86\u89e3\u548c\u6cbb\u7597\u3002<\/p>\n<\/div>

<\/div><\/div>
\"2109india\"<\/span><\/div>

\"(\u300a\u4e16\u754c\u4eba\u6743\u5ba3\u8a00\u300b) 2B \u589e\u5f3a\u60a3\u8005\u6743\u80fd\u4f1a\u8bae<\/strong> \u4f1a\u8bae\u5728\u5370\u5ea6\u5b5f\u4e70\u4e3e\u884c\uff0c\u4e3a\u80a2\u4f53\u8170\u808c\u840e\u7f29\u75c7\u60a3\u8005\u63d0\u4f9b\u4e86\u4e00\u4e2a\u4ea4\u6d41\u5e73\u53f0\uff0c\u8ba9\u4ed6\u4eec\u4e0e\u8be5\u9886\u57df\u7684\u6743\u5a01\u4e13\u5bb6\u8054\u7cfb\u3001\u5206\u4eab\u7ecf\u9a8c\u5e76\u83b7\u5f97\u5b9d\u8d35\u7684\u89c1\u89e3\u3002<\/p>\n<\/div><\/div><\/div><\/div><\/div>

2020<\/h2><\/div><\/div><\/div>
<\/div><\/div><\/div><\/div>
<\/div><\/div><\/div>
\"2020laf\"<\/span><\/div>

2020 \u5e74 LGMD \u610f\u8bc6\u57fa\u91d1\u4f1a<\/strong><\/a> \u6210\u7acb\u8be5\u7ec4\u7ec7\u7684\u76ee\u7684\u662f\u63d0\u9ad8\u8ba4\u8bc6\u3001\u63d0\u4f9b\u6559\u80b2\u548c\u652f\u6301\u7814\u7a76\u5de5\u4f5c\uff0c\u4ee5\u6539\u5584\u80a2\u8170\u808c\u8425\u517b\u4e0d\u826f\u75c7 (LGMD) \u60a3\u8005\u7684\u751f\u6d3b\u3002<\/p>\n<\/div>

<\/div><\/div>
\"2020girdie\"<\/span><\/div>

\u5409\u5c14\u8fea\uff0cLGMD \u5927\u4f7f\u3001<\/strong><\/a> \u88ab\u4ecb\u7ecd\u7ed9\u4e86 LGMD \u793e\u533a\u3002<\/p>\n<\/div>

<\/div><\/div>

A \u75c5\u4eba\u503e\u542c\u4f1a<\/strong> \u80a2\u8170\u808c\u8425\u517b\u4e0d\u826f\u75c7 (LGMD) \u7684\u60a3\u8005\u548c\u62a4\u7406\u4eba\u5458\u6709\u673a\u4f1a\u4e0e\u76d1\u7ba1\u5b98\u5458\u76f4\u63a5\u4ea4\u6d41\u7ecf\u9a8c\uff0c\u5e76\u5f3a\u8c03\u5bf9\u65b0\u7597\u6cd5\u548c\u7597\u6cd5\u7684\u8feb\u5207\u9700\u6c42\u3002<\/p>\n<\/div><\/div><\/div><\/div><\/div>

2021<\/h2><\/div><\/div><\/div>
<\/div><\/div><\/div><\/div>
<\/div><\/div><\/div>
\"2021lgmdnews\"<\/span><\/div>

\u7b2c\u4e00\u671f LGMD \u65b0\u95fb<\/em> \u6697\u76d2<\/strong><\/a> \u4e8e 2021 \u5e74\u51fa\u7248\uff08\u7b2c 1 \u5377\/\u7b2c 1 \u671f\uff09\u3002<\/p>\n<\/div>

<\/div><\/div>

\u7b2c\u4e00\u4e2a \"\u865a\u62df \"\u56fd\u9645 LGMD \u4f1a\u8bae<\/strong> \u5c3d\u7ba1\u80a2\u8170\u808c\u8425\u517b\u4e0d\u826f\u75c7\uff08LGMD\uff09\u5728\u5168\u7403\u8303\u56f4\u5185\u7684\u6d41\u884c\u5e26\u6765\u4e86\u5404\u79cd\u6311\u6218\uff0c\u4f46\u8be5\u4f1a\u8bae\u7684\u53ec\u5f00\u4f7f\u6765\u81ea\u4e16\u754c\u5404\u5730\u7684\u4e0e\u4f1a\u8005\u80fd\u591f\u5c31\u8be5\u75be\u75c5\u7684\u6700\u65b0\u7814\u7a76\u3001\u6cbb\u7597\u548c\u652f\u6301\u8fdb\u884c\u4ea4\u6d41\u548c\u8ba8\u8bba\u3002<\/p>\n<\/div>

<\/div><\/div>
\"2021diagnosticcodes\"<\/span><\/div>

\u4e13\u7528\u7684 ICD-10 \u533b\u7597\u4ee3\u7801\u5df2\u83b7\u6279\u51c6<\/strong> \u5b83\u53ef\u7528\u4e8e\u51e0\u79cd\u7279\u5b9a\u4e9a\u578b\u7684\u80a2\u8170\u808c\u8425\u517b\u4e0d\u826f\u75c7\uff08LGMD\uff09\u4ee5\u53ca\u975e\u7279\u5b9a\u4e9a\u578b\u7684\u80a2\u8170\u808c\u8425\u517b\u4e0d\u826f\u75c7\uff0c\u4ece\u800c\u6709\u52a9\u4e8e\u533b\u7597\u7cfb\u7edf\u66f4\u597d\u5730\u8bca\u65ad\u3001\u6cbb\u7597\u548c\u8ddf\u8e2a\u8be5\u75be\u75c5\u3002<\/p>\n<\/div>

<\/div><\/div>

\u7684 2 \u671f\u4e34\u5e8a\u8bd5\u9a8c\u4e2d\uff0c\u4e3a\u7b2c\u4e00\u4f4d\u60a3\u8005\u6ce8\u5c04\u4e86\u5242\u91cf\u3002 BBP-418\uff08\u6838\u7cd6\u9187\uff09<\/strong> \u8be5\u9879\u76ee\u7531 BridgeBio Pharma \u53ca\u5176\u5b50\u516c\u53f8 ML Bio Solutions \u8d1f\u8d23\u5b9e\u65bd\uff0c\u7528\u4e8e\u6cbb\u7597 2I\/R9 \u578b\u80a2\u4f53\u8170\u808c\u840e\u7f29\u75c7\uff08LGMD2I\/R9\uff09\u3002<\/p>\n<\/div><\/div><\/div><\/div><\/div>

2022<\/h2><\/div><\/div><\/div>
<\/div><\/div><\/div><\/div>
<\/div><\/div><\/div>
\"Screenshot<\/span><\/div>

2022 \u5e74 LGMD \u5168\u7403\u5ba3\u4f20\u5cf0\u4f1a<\/strong> \u4f1a\u8bae\u6c47\u96c6\u4e86\u6765\u81ea\u4e16\u754c\u5404\u5730\u7684\u5021\u5bfc\u8005\u3001\u60a3\u8005\u3001\u7814\u7a76\u4eba\u5458\u548c\u653f\u7b56\u5236\u5b9a\u8005\uff0c\u5171\u540c\u534f\u4f5c\uff0c\u63a8\u8fdb\u63d0\u9ad8\u8ba4\u8bc6\u3001\u652f\u6301\u7814\u7a76\u548c\u6539\u5584\u80a2\u8170\u808c\u8425\u517b\u4e0d\u826f\u75c7\uff08LGMD\uff09\u60a3\u8005\u751f\u6d3b\u7684\u5de5\u4f5c\u3002<\/p>\n<\/div>

<\/div><\/div>
\"2022girdie\"<\/span><\/div>

\u5409\u5c14\u8fea<\/strong> LGMD \u5927\u4f7f \"\u6829\u6829\u5982\u751f\"\uff0c\u5e76\u5728\u7231\u8377\u534e\u5dde\u4e3e\u884c\u7684\u97e6\u5c14\u65af\u901a\u808c\u5f20\u529b\u969c\u788d\u60a3\u8005\u548c\u5bb6\u5ead\u4f1a\u8bae\u4e0a\u9996\u6b21\u4eae\u76f8\u3002<\/p>\n<\/div>

<\/div><\/div>
\"2022externallyled\"<\/span><\/div>

\u7b2c\u4e00\u4e2a \u9488\u5bf9\u80a2\u8170\u808c\u8425\u517b\u4e0d\u826f\u75c7\uff08LGMDs\uff09\u7684\u5916\u90e8\u4e3b\u5bfc\u7684\u4ee5\u60a3\u8005\u4e3a\u4e2d\u5fc3\u7684\u836f\u7269\u5f00\u53d1\u4f1a\u8bae\uff08EL-PFDD\uff09<\/strong> \u4e0e\u7f8e\u56fd\u98df\u54c1\u53ca\u836f\u7269\u7ba1\u7406\u5c40\u548c\u5176\u4ed6\u5229\u76ca\u76f8\u5173\u8005\u4e00\u8d77\u4e3e\u884c\uff0c\u4e3a\u60a3\u8005\u548c\u62a4\u7406\u4eba\u5458\u63d0\u4f9b\u4e86\u4e00\u4e2a\u91cd\u8981\u7684\u5e73\u53f0\uff0c\u5206\u4eab\u4ed6\u4eec\u7684\u7ecf\u9a8c\u548c\u89c2\u70b9\uff0c\u4e3a\u836f\u7269\u5f00\u53d1\u548c\u76d1\u7ba1\u51b3\u7b56\u63d0\u4f9b\u4fe1\u606f\u3002<\/p>\n<\/div>

<\/div><\/div>

\u75be\u75c5\u9884\u9632\u63a7\u5236\u4e2d\u5fc3\u5236\u5b9a\u4e86\u9488\u5bf9\u8f7b\u578b\u5730\u4e2d\u6d77\u816e\u817a\u708e\u7684 ICD-10 \u7f16\u7801<\/strong>\u8fd9\u6837\u5c31\u80fd\u5728\u533b\u7597\u4fdd\u5065\u7cfb\u7edf\u4e2d\u66f4\u7cbe\u786e\u5730\u8bca\u65ad\u3001\u8bb0\u5f55\u548c\u8ddf\u8e2a\u80a2\u8170\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684\u4e9a\u578b\u3002<\/p>\n<\/div>

<\/div><\/div>
\"2023<\/span><\/div>

\u975e\u8425\u5229\u7ec4\u7ec7 \u8482\u5ef7\u56e2\u961f<\/strong><\/a> \u6210\u7acb\uff0c\u4e3a LGMD2J\/R10 \u793e\u533a\u63d0\u4f9b\u652f\u6301\u3002<\/p>\n<\/div>

\u8482\u5ef7\u56e2\u961f\u4e3b\u529e\u4e86 \u79d1\u5b66\u5bb6\u5ead\u7814\u7a76-\u5bb6\u5ead\u4f1a\u8bae\u3002<\/strong><\/p>\n<\/div>

<\/div><\/div>
\"2023<\/span><\/div>

\"(\u300a\u4e16\u754c\u4eba\u6743\u5ba3\u8a00\u300b) \u4e39\u5c3c\u5c14-\u5f17\u683c\u68ee LGMD \u57fa\u91d1\u4f1a<\/strong><\/a> \u8be5\u7ec4\u7ec7\u6210\u7acb\u4e8e\u6fb3\u5927\u5229\u4e9a\uff0c\u81f4\u529b\u4e8e\u652f\u6301\u7814\u7a76\u3001\u63d0\u9ad8\u8ba4\u8bc6\uff0c\u5e76\u4e3a\u53d7\u8170\u808c\u8425\u517b\u4e0d\u826f\u75c7\u5f71\u54cd\u7684\u4e2a\u4eba\u548c\u5bb6\u5ead\u63d0\u4f9b\u8d44\u6e90\u3002<\/p>\n<\/div>

<\/div><\/div>

\u7b2c\u4e00\u540d\u60a3\u8005\u7684\u5242\u91cf\u662f\u5728 \u9488\u5bf9 LGMD-R9 \u7684\u57fa\u56e0\u7597\u6cd5\u5728\u6b27\u6d32\u8fdb\u884c 1\/2 \u671f\u4e34\u5e8a\u8bd5\u9a8c<\/strong>\u8be5\u7814\u7a76\u7531 Atamyo Therapeutics \u516c\u53f8\u8fdb\u884c\uff0c\u6807\u5fd7\u7740\u8be5\u516c\u53f8\u5728\u5f00\u53d1\u80a2\u8170\u808c\u8425\u517b\u4e0d\u826f\u75c7\u8fd9\u4e00\u4e9a\u578b\u7684\u6cbb\u7597\u65b9\u6cd5\u65b9\u9762\u8fc8\u51fa\u4e86\u91cd\u8981\u4e00\u6b65\u3002<\/p>\n<\/div>

<\/div><\/div>
\"2022<\/span><\/div>

CureLGMD2i \u4e3e\u529e\u4e86\u7b2c\u4e00\u5c4a \"\u8fde\u63a5\u6cbb\u7597\"<\/strong> \u5728 LGMD \u5ba3\u4f20\u65e5\u4e3e\u529e\u6d3b\u52a8\uff0c\u5c06\u60a3\u8005\u3001\u5bb6\u5c5e\u3001\u7814\u7a76\u4eba\u5458\u548c\u5021\u5bfc\u8005\u805a\u96c6\u5728\u4e00\u8d77\uff0c\u4ee5\u63d0\u9ad8\u8ba4\u8bc6\u3001\u5206\u4eab\u77e5\u8bc6\u5e76\u652f\u6301\u5bfb\u627e 2i \u578b\u80a2\u4f53\u8170\u808c\u840e\u7f29\u75c7\u7684\u6cbb\u7597\u65b9\u6cd5\u3002<\/p>\n<\/div>

<\/div><\/div>

\"(\u300a\u4e16\u754c\u4eba\u6743\u5ba3\u8a00\u300b) \u4e34\u5e8a\u7ed3\u679c\u7814\u7a76\/\u94c1\u75e2\u75be\u7684\u81ea\u7136\u53f2<\/strong> \u5e86\u795d\u60a3\u8005\u53c2\u4e0e\u548c\u4e34\u5e8a\u6570\u636e\u6536\u96c6\u7684 10 \u5e74\uff0c\u7a81\u51fa\u4e86\u5341\u5e74\u6765\u5bf9\u4e86\u89e3\u548c\u6cbb\u7597\u8fd9\u79cd\u7279\u6b8a\u5f62\u5f0f\u7684\u80a2\u8170\u808c\u8425\u517b\u4e0d\u826f\u75c7\u505a\u51fa\u7684\u5b9d\u8d35\u8d21\u732e\u3002<\/p>\n<\/div>

<\/div><\/div>
\"2022<\/span><\/div>

LGMD \u5ba3\u4f20\u6346\u7ed1\u5305<\/strong><\/a> \u7531 LGMD \u610f\u8bc6\u57fa\u91d1\u4f1a\u5728\u7f8e\u56fd 6 \u5bb6\u8bca\u6240\u90e8\u7f72\u3002<\/p>\n<\/div><\/div><\/div><\/div><\/div>

2023<\/h2><\/div><\/div><\/div>
<\/div><\/div><\/div><\/div>
<\/div><\/div><\/div>
\"2023voicepatient\"<\/span><\/div>

2023 \u5e74 LGMD \"\u60a3\u8005\u4e4b\u58f0 \"\u62a5\u544a<\/strong> \u8be5\u62a5\u544a\u5f3a\u8c03\u4e86\u516d\u79cd\u7279\u5b9a\u4e9a\u578b\u80a2\u8170\u808c\u8425\u517b\u4e0d\u826f\u75c7\u60a3\u8005\u5c1a\u672a\u6ee1\u8db3\u7684\u9700\u6c42\u548c\u9762\u4e34\u7684\u6311\u6218\uff0c\u5e76\u4e3a\u6307\u5bfc\u672a\u6765\u7684\u7814\u7a76\u548c\u6cbb\u7597\u5de5\u4f5c\u63d0\u4f9b\u4e86\u5b9d\u8d35\u7684\u89c1\u89e3\u3002<\/p>\n<\/div>

<\/div><\/div>

\u542f\u52a8\u4e86\u4e00\u9879\u7814\u7a76\uff0c\u4ee5\u8bc4\u4f30 BBP-418\uff08\u6838\u7cd6\u9187\uff09<\/strong> \u7531 ML BioSolutions \u516c\u53f8\u8fdb\u884c\u3002<\/p>\n<\/div>

<\/div><\/div>

\u7b2c\u4e00\u4f4d\u60a3\u8005\u662f\u5728\u4e00\u9879\u540d\u4e3a AB-1003 \u57fa\u56e0\u7597\u6cd5<\/strong> \u8fd9\u662f\u9488\u5bf9\u8fd9\u79cd LGMD \u4e9a\u578b\u5f00\u53d1\u65b0\u7597\u6cd5\u7684\u4e00\u4e2a\u91cd\u8981\u91cc\u7a0b\u7891\u3002<\/p>\n<\/div>

<\/div><\/div>
\"2023intlconference\"<\/span><\/div>

\u7b2c\u4e00\u4e2a \"\u6df7\u5408\u578b \"\u56fd\u9645 LGMD \u4f1a\u8bae<\/strong> \u4f1a\u8bae\u5728\u534e\u76db\u987f\u7279\u533a\u4e3e\u884c\uff0c\u7ed3\u5408\u4e86\u73b0\u573a\u548c\u865a\u62df\u53c2\u4e0e\uff0c\u4ee5\u6ee1\u8db3\u5168\u7403\u89c2\u4f17\u7684\u9700\u6c42\uff0c\u5e76\u4fc3\u8fdb\u7814\u7a76\u4eba\u5458\u3001\u4e34\u5e8a\u533b\u751f\u3001\u60a3\u8005\u548c\u5021\u5bfc\u8005\u4e4b\u95f4\u7684\u5408\u4f5c\uff0c\u5171\u540c\u6297\u51fb\u80a2\u8170\u808c\u8425\u517b\u4e0d\u826f\u75c7\u3002<\/p>\n<\/div>

<\/div><\/div>
\"2023<\/span><\/div>

\u6cbb\u7597\u9499\u86cb\u767d\u9176\u8054\u76df 3<\/a> \u63a8\u51fa\u65b0\u7684 LGMD2A\/Calpainopathy \u767b\u8bb0\u5904 <\/strong><\/p>\n<\/div>

<\/div><\/div>
\"2023<\/span><\/div>

\u975e\u8425\u5229\u7ec4\u7ec7 \u8fea\u7fc1\u57fa\u91d1\u4f1a<\/strong><\/a> \u6210\u7acb\uff0c\u81f4\u529b\u4e8e\u652f\u6301\u7814\u7a76\u3001\u63d0\u9ad8\u8ba4\u8bc6\uff0c\u5e76\u4e3a\u53d7\u8170\u808c\u840e\u7f29\u75c7\u5f71\u54cd\u7684\u4e2a\u4eba\u548c\u5bb6\u5ead\u63d0\u4f9b\u8d44\u6e90\u3002<\/p>\n<\/div>

<\/div><\/div>

Sarepta Therapeutics \u5728 SRP 6004 \u6982\u5ff5\u9a8c\u8bc1\u4e34\u5e8a\u8bd5\u9a8c\u4e2d\u542f\u52a8\u4e86\u9996\u4e2a\u5168\u8eab\/\u7cfb\u7edf\u6cbb\u7597\uff0c\u8be5\u8bd5\u9a8c\u662f \u5bfc\u822a\u4eea<\/strong><\/a> \u8fd9\u662f\u6cbb\u7597\u8fd9\u79cd\u7279\u6b8a\u5f62\u5f0f\u7684\u80a2\u8170\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684\u91cd\u5927\u8fdb\u5c55\u3002<\/p>\n<\/div><\/div><\/div><\/div><\/div>

2024<\/h2><\/div><\/div><\/div>
<\/div><\/div><\/div><\/div>
<\/div><\/div><\/div>
\"2024<\/span><\/div>

2024 \u5e74\uff0c\u7b5b\u67e5\u5f00\u59cb\u5728 EMERGENE<\/strong> Sarepta Therapeutics \u516c\u53f8\u5f00\u5c55\u4e86 SRP-9003 \u6cbb\u7597 2E\/R4 \u578b\u80a2\u8170\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684 3 \u671f\u4e34\u5e8a\u7814\u7a76\uff0c\u8fd9\u6807\u5fd7\u7740\u8be5 LGMD \u4e9a\u578b\u7684\u6f5c\u5728\u65b0\u6cbb\u7597\u65b9\u6848\u8fc8\u51fa\u4e86\u5173\u952e\u4e00\u6b65\u3002<\/p>\n<\/div>

<\/div><\/div>
\"2024drugworkshop\"<\/span><\/div>

LGMD \u79d1\u5b66\u836f\u7269\u5f00\u53d1\u7814\u8ba8\u4f1a<\/strong> \u5728\u9a6c\u91cc\u5170\u5dde\u8d1d\u585e\u65af\u8fbe\u4e3e\u884c\uff0c\u7814\u7a76\u4eba\u5458\u3001\u4e34\u5e8a\u533b\u751f\u548c\u884c\u4e1a\u9886\u8896\u9f50\u805a\u4e00\u5802\uff0c\u8ba8\u8bba\u5e76\u63a8\u52a8\u80a2\u8170\u808c\u8425\u517b\u4e0d\u826f\u75c7\u65b0\u6cbb\u7597\u7b56\u7565\u7684\u5f00\u53d1\u3002<\/p>\n<\/div>

<\/div><\/div>
\"2024<\/span><\/div>

\u7528\u4e8e\u6d4b\u8bd5\u7684\u4e34\u5e8a\u8bd5\u9a8c\u7533\u8bf7 ATA-200 \u57fa\u56e0\u7597\u6cd5\u7814\u7a76<\/strong> Atamyo Therapeutics \u516c\u53f8\u5728\u6cd5\u56fd\u548c\u610f\u5927\u5229\u6279\u51c6\u4e86\u7528\u4e8e 2C\/R5 \u578b\u80a2\u4f53\u8170\u808c\u840e\u7f29\u75c7\u60a3\u513f\u7684\u8be5\u836f\u7269\uff0c\u4e3a\u8be5 LGMD \u4e9a\u578b\u7684\u6f5c\u5728\u6cbb\u7597\u65b9\u6cd5\u7684\u91cd\u8981\u7814\u7a76\u94fa\u5e73\u4e86\u9053\u8def\u3002<\/p>\n<\/div>

<\/div><\/div>
\"2024<\/span><\/div>

\u7b2c\u4e00\u4e2a LGMDR9 (LGMD2I) \u6b27\u6d32\u60a3\u8005\u65e5<\/strong> \u5728\u8377\u5170\u963f\u59c6\u65af\u7279\u4e39\u4e3e\u884c\uff0c\u4e3a\u60a3\u8005\u3001\u5bb6\u5c5e\u3001\u7814\u7a76\u4eba\u5458\u548c\u533b\u62a4\u4e13\u4e1a\u4eba\u5458\u63d0\u4f9b\u4e86\u4e00\u4e2a\u4ea4\u6d41\u5e73\u53f0\uff0c\u5206\u4eab\u7ecf\u9a8c\uff0c\u4e86\u89e3 2I \u578b\u80a2\u4f53\u8170\u808c\u840e\u7f29\u75c7\u6cbb\u7597\u548c\u7ba1\u7406\u65b9\u9762\u7684\u6700\u65b0\u8fdb\u5c55\u3002<\/p>\n<\/div>

<\/div><\/div>

\"(\u300a\u4e16\u754c\u4eba\u6743\u5ba3\u8a00\u300b) LGMD2A\/R1 \u79d1\u5b66\u4f1a\u8bae<\/strong> \u5728\u5f17\u5409\u5c3c\u4e9a\u5dde\u963f\u7075\u987f\u4e3e\u884c\uff0c\u4e13\u5bb6\u548c\u7814\u7a76\u4eba\u5458\u6c47\u805a\u4e00\u5802\uff0c\u5171\u540c\u8ba8\u8bba\u5728\u4e86\u89e3\u548c\u6cbb\u7597 2A\/R1 \u578b\u80a2\u8170\u808c\u8425\u517b\u4e0d\u826f\u75c7\u65b9\u9762\u7684\u6700\u65b0\u53d1\u73b0\u548c\u8fdb\u5c55\u3002<\/p>\n<\/div>

<\/div><\/div>

\"(\u300a\u4e16\u754c\u4eba\u6743\u5ba3\u8a00\u300b) Dysferlin \u79d1\u5b66\u4f1a\u8bae<\/strong> \u7814\u7a76\u4eba\u5458\u3001\u4e34\u5e8a\u533b\u751f\u548c\u884c\u4e1a\u4e13\u5bb6\u9f50\u805a\u5fb7\u514b\u8428\u65af\u5dde\u4f11\u65af\u987f\uff0c\u5206\u4eab\u7814\u7a76\u548c\u6cbb\u7597\u80a2\u8170\u808c\u8425\u517b\u4e0d\u826f\u75c7\uff08dysferlinopathy\uff09\u7684\u89c1\u89e3\u548c\u8fdb\u5c55\u3002<\/p>\n<\/div>

<\/div><\/div>
\"2024\"<\/span><\/div>

\"(\u300a\u4e16\u754c\u4eba\u6743\u5ba3\u8a00\u300b) \u7b2c 10 \u5c4a LGMD \u5ba3\u4f20\u65e5<\/strong><\/a> \u5168\u4e16\u754c\u90fd\u5728\u5e86\u795d\u3002<\/p>\n<\/div><\/div><\/div><\/div><\/div>

2025<\/h2><\/div><\/div><\/div>
<\/div><\/div><\/div><\/div>
<\/div><\/div><\/div>
\"CureSCG<\/a><\/span><\/div>

Cure SCG<\/a> (Cure Sarcoglycanopathy)<\/strong> was established in the United States. Their mission is to find a cure for LGMD Sarcoglycanopathy, inclusive of its LGMD2C\/R5, LGMD2D\/R3, LGMD2E\/\u200bR4, and LGMD2F\/R6 variants.<\/strong><\/p>\n<\/div>

<\/div><\/div>

Coalition of LGMD Advocacy Organizations hosted a reception for researchers and clinicians at the 2025 MDA Clinical and Scientific Conference<\/a> in Dallas, TX<\/p>\n<\/div>

<\/div><\/div>
\"In<\/a><\/span><\/div>

The documentary\u00a0\u201cIn Search of Strength \u2013 The Unfinished Fight Against\u00a0Muscular\u00a0Dystrophy\u201d<\/strong><\/a>\u00a0was released.<\/p>\n<\/div>

<\/div><\/div>
\"Curelgmd2t\"<\/span><\/div>

Cure LGMD2T<\/strong>\u00a0was established in the United States. Their mission is dedicated to raising awareness of\u00a0Limb-Girdle\u00a0Muscular\u00a0Dystrophy\u00a02T<\/strong>\u00a0and providing critical funding for research into effective treatments.<\/p>\n<\/div>

<\/div><\/div>
\"Askbio<\/span><\/div>

AskBio Advances Gene Therapy Clinical Trial\u00a0for\u00a0Limb-Girdle\u00a0Muscular\u00a0Dystrophy\u00a0Type 2I\/R9<\/strong>\u00a0with Dosing of First Participant in Second Cohort<\/p>\n<\/div>

<\/div><\/div>
\"2024<\/span><\/div>

Atamyo Therapeutics Announces First Patients Dosed with ATA-200 Gene Therapy in\u00a0LGMD-2C\/R5<\/strong>\u00a0Clinical Trial<\/p>\n<\/div>

<\/div><\/div>

U.S. FDA Grants Platform Technology Designation to the Viral Vector Used in SRP-9003, Sarepta\u2019s Investigational Gene Therapy for the Treatment of\u00a0Limb\u00a0Girdle\u00a0Muscular\u00a0Dystrophy\u00a0Type 2E\/R4<\/strong><\/p>\n<\/div><\/div><\/div><\/div><\/div>

2026<\/h2><\/div><\/div><\/div>
<\/div><\/div><\/div><\/div>
<\/div><\/div><\/div>
\"CureSCG<\/a><\/span><\/div>

Atamyo Therapeutics<\/a> Presents Positive Safety and Efficacy Data from a US Phase 1b\/2 Trial of its Gene Therapy (ATA-200) for Linb-Girdle Muscular Dystrophy R5 (LGMD-R5)<\/b><\/p>\n<\/div>

<\/div><\/div>
\"CureSCG<\/a><\/span><\/div>

BrideBio<\/a> Submits NDA to FDA for BBP-418 for Individuals Living with LGMD2I\/R9<\/p>\n<\/div>

<\/div><\/div>
\"2026<\/a><\/span><\/div>

LGMD Policy Action Day<\/strong> brought our community directly to Capitol Hill \u2014 with a full schedule of meetings across both the House and Senate to address the realities of ultra-rare drug development. LGMD Policy Action Days bring in scientific experts who understand the health policy ecosystem as it intersects with science. It is a perspective that policymakers want to hear, and the stories of those with lived experience help underscore the need as we share how ultra-rare drug development must evolve to get treatments to our communities.<\/p>\n<\/div>

<\/div><\/div>
<\/span><\/div>

The Speak Foundation Launches Patient-Led National LGMD Centers of Excellence<\/strong>, Uniting Leading Academic Institutions to Accelerate Clinical Trials and New Therapies.<\/p>\n<\/div><\/div><\/div><\/div><\/div><\/p>","protected":false},"excerpt":{"rendered":"

\u63a2\u7d22\u5341\u5e74\u6765\u9632\u6cbb\u80a2\u8170\u808c\u8425\u517b\u4e0d\u826f\u75c7 (LGMD) \u7684\u91cd\u8981\u91cc\u7a0b\u7891\u3002\u4ece\u5f00\u521b\u6027\u7684\u57fa\u56e0\u6d4b\u5e8f\u548c\u5ba3\u4f20\u6d3b\u52a8\uff0c\u5230\u56fd\u9645\u4f1a\u8bae\u548c\u4e34\u5e8a\u8bd5\u9a8c\uff0c\u4e86\u89e3\u4e3a\u6539\u5584 LGMD \u60a3\u8005\u7684\u751f\u6d3b\u800c\u53d6\u5f97\u7684\u91cd\u5927\u8fdb\u5c55\u548c\u6b63\u5728\u8fdb\u884c\u7684\u52aa\u529b\u3002<\/p>","protected":false},"author":1,"featured_media":9006,"parent":43,"menu_order":0,"comment_status":"closed","ping_status":"closed","template":"100-width.php","meta":{"_acf_changed":false,"footnotes":""},"class_list":["post-8368","page","type-page","status-publish","has-post-thumbnail","hentry"],"acf":[],"yoast_head":"\nLGMD Milestones - Over a Decade of Difference - LGMD Awareness Foundation<\/title>\n<meta name=\"description\" content=\"Explore a decade of impactful milestones in the fight against Limb-Girdle Muscular Dystrophy (LGMD), from groundbreaking genetic sequencing and awareness campaigns to international conferences and clinical trials.\" \/>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/www.lgmd-info.org\/zh\/knowledge-base\/lgmd-milestones-a-decade-of-difference\/\" \/>\n<meta property=\"og:locale\" content=\"zh_CN\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"LGMD Milestones - 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