{"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":"2026-06-10T16:24:00","modified_gmt":"2026-06-10T16:24:00","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":"

Explore over 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, discover the significant advancements and ongoing efforts to improve the lives of those affected by LGMD.<\/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>

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

The FDA has accepted the new drug application (NDA) from BridgeBio (MLBio Solutions)! This is an amazing step toward potential approval of the first-ever treatment<\/strong> for people living with LGMD 2I\/R9! A PDUFA (Prescription Drug User Fee Act) target action date of November 27, 2026 has been set, the date by which the FDA will complete its review.<\/span><\/p>\n<\/div>

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

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