{"id":3482,"date":"2025-06-14T15:25:00","date_gmt":"2025-06-14T13:25:00","guid":{"rendered":"https:\/\/remeds.org\/?p=3482"},"modified":"2025-11-14T09:19:28","modified_gmt":"2025-11-14T08:19:28","slug":"survey-results-the-global-mecp2-duplication-community-speaks","status":"publish","type":"post","link":"https:\/\/remeds.org\/ja\/2025\/06\/14\/survey-results-the-global-mecp2-duplication-community-speaks\/","title":{"rendered":"\u8abf\u67fb\u7d50\u679c:\u4e16\u754c\u306eMECP2\u91cd\u8907\u75c7\u5019\u7fa4\u30b3\u30df\u30e5\u30cb\u30c6\u30a3\u304c\u8a9e\u308b"},"content":{"rendered":"

MECP2\u91cd\u8907\u75c7\u5019\u7fa4\u306e\u7814\u7a76\u306f\u3001\u304b\u3064\u3066\u306a\u3044\u4e00\u6b69\u3092\u8e0f\u307f\u51fa\u3057\u3001\u81e8\u5e8a\u8a66\u9a13\u306e\u6bb5\u968e\u306b\u5165\u308a\u307e\u3057\u305f\u3002\u3053\u308c\u306f\u3001\u5b50\u4f9b\u305f\u3061\u306e\u3088\u308a\u826f\u3044\u672a\u6765\u306e\u305f\u3081\u306b\u9577\u5e74\u61f8\u547d\u306b\u53d6\u308a\u7d44\u3093\u3067\u304d\u305f\u591a\u304f\u306e\u5bb6\u65cf\u306b\u3068\u3063\u3066\u3001\u5e0c\u671b\u306e\u5149\u3068\u306a\u3063\u3066\u3044\u307e\u3059\u3002<\/p>\n\n\n\n

\u7c73\u56fd\u3067\u306fATTUNE(Ionis\u793e\u306eION440)\u3001\u4e2d\u56fd\u3067\u306fHERO(HuidaGene\u793e\u306eHG204)\u3068\u3044\u3063\u305f\u521d\u671f\u6bb5\u968e\u306e\u81e8\u5e8a\u8a66\u9a13\u304c\u958b\u59cb\u3055\u308c\u3001\u79c1\u305f\u3061\u306f\u6cbb\u7642\u6cd5\u306e\u63a2\u7d22\u306b\u304a\u3051\u308b\u6b74\u53f2\u7684\u306a\u77ac\u9593\u3092\u8fce\u3048\u3066\u307e\u3059\u3002\u3053\u308c\u3089\u306e\u7b2c1\/2\u76f8\u81e8\u5e8a\u8a66\u9a13\u306f\u3001\u6cbb\u9a13\u85ac\u306e\u5b89\u5168\u6027\u3068\u6709\u52b9\u6027\u306e\u53ef\u80fd\u6027\u3092\u8a55\u4fa1\u3059\u308b\u91cd\u8981\u306a\u5f79\u5272\u3092\u679c\u305f\u3057\u307e\u3059\u304c\u3001\u3042\u304f\u307e\u3067\u59cb\u307e\u308a\u306b\u904e\u304e\u307e\u305b\u3093\u3002<\/p>\n\n\n\n

\u4eca\u5f8c\u306e\u5c55\u958b\u3001\u7279\u306b\u3088\u308a\u5927\u898f\u6a21\u3067\u5f8c\u671f\u306e\u81e8\u5e8a\u8a66\u9a13\u306b\u5099\u3048\u308b\u305f\u3081\u30012024\u5e743\u6708\u306b\u4e16\u754c\u898f\u6a21\u306e\u8abf\u67fb\u3092\u958b\u59cb\u3057\u307e\u3057\u305f\u3002\u3053\u306e\u8abf\u67fb\u306e\u76ee\u7684\u306f\u3001\u4e16\u754c\u4e2d\u306e\u5bb6\u65cf\u306e\u95a2\u5fc3\u3068\u6e96\u5099\u72b6\u6cc1\u3092\u628a\u63e1\u3057\u3001\u73fe\u5728\u306e\u8a66\u9a13\u3067\u6709\u671b\u306a\u7d50\u679c\u304c\u5f97\u3089\u308c\u305f\u5834\u5408\u306b\u3001\u6b21\u306e\u8a66\u9a13\u30c7\u30b6\u30a4\u30f3\u3001\u8a08\u753b\u3001\u5b9f\u65bd\u62e0\u70b9\u306e\u9078\u5b9a\u3092\u652f\u63f4\u3059\u308b\u3053\u3068\u3067\u3059\u3002\u8abf\u67fb\u306b\u306f\u3001\u4e16\u754c\u5404\u56fd\u306e94\u5bb6\u65cf\u304b\u3089\u56de\u7b54\u3092\u304a\u5bc4\u305b\u3044\u305f\u3060\u304d\u307e\u3057\u305f\u3002<\/p>\n\n\n\n

\u6642\u9593\u3092\u5272\u3044\u3066\u53c2\u52a0\u3057\u3066\u304f\u308c\u305f\u3059\u3079\u3066\u306e\u5bb6\u65cf\u306b\u5fc3\u304b\u3089\u611f\u8b1d\u3057\u305f\u3044\u3002 <\/strong>\u3059\u3079\u3066\u306e\u56de\u7b54\u306f\u3001\u79c1\u305f\u3061\u304c\u5730\u57df\u793e\u4f1a\u304b\u3089\u8cb4\u91cd\u306a\u60c5\u5831\u3092\u53ce\u96c6\u3057\u3001\u6700\u7d42\u7684\u306b\u5730\u57df\u793e\u4f1a\u3092\u3088\u308a\u3088\u304f\u30b5\u30dd\u30fc\u30c8\u3059\u308b\u306e\u306b\u5f79\u7acb\u3061\u307e\u3059\u3002<\/p>\n\n\n\n

\u4ee5\u4e0b\u306b\u8abf\u67fb\u7d50\u679c\u3092\u307e\u3068\u3081\u305f\u306e\u3067\u3001\u305c\u3072\u3054\u89a7\u304f\u3060\u3055\u3044\u3002<\/p>\n\n\n\n

<\/a><\/p>\n\n\n\n

\"\"<\/figure>\n\n\n\n

\u4e16\u754c\u5404\u56fd\u304b\u3089\u5408\u8a0894\u5bb6\u65cf\u304c\u8abf\u67fb\u306b\u53c2\u52a0\u3057\u3001\u81e8\u5e8a\u8a66\u9a13\u3078\u306e\u53c2\u52a0\u306b\u5bfe\u3059\u308b\u4e16\u754c\u7684\u306a\u95a2\u5fc3\u306e\u9ad8\u3055\u304c\u793a\u3055\u308c\u307e\u3057\u305f\u3002\u56de\u7b54\u306e\u591a\u304f\u306f\u3001\u30e8\u30fc\u30ed\u30c3\u30d1\u3068\u5317\u7c73\u304b\u3089\u3067\u3057\u305f\u3002\u5168\u4f53\u306e91\uff05\u304c\u7537\u5150\u306e\u4fdd\u8b77\u8005\u307e\u305f\u306f\u4ecb\u8b77\u8005\u3067\u30019\uff05\u304c\u5973\u5150\u306e\u4fdd\u8b77\u8005\u307e\u305f\u306f\u4ecb\u8b77\u8005\u3067\u3057\u305f\u3002\u8abf\u67fb\u306b\u53c2\u52a0\u3057\u305f\u5b50\u4f9b\u305f\u3061\u306e\u5e74\u9f62\u306f0\u6b73\u304b\u308934\u6b73\u307e\u3067\u5e45\u5e83\u304f\u542b\u307e\u308c\u3066\u3044\u307e\u3057\u305f\u3002<\/p>\n\n\n\n

\"\"
\u81e8\u5e8a\u8a66\u9a13\u3078\u306e\u53c2\u52a0\u610f\u6b32\uff1a <\/strong>\u5404\u53c2\u52a0\u8005\u306f1\u3064\u306e\u56de\u7b54\u306e\u307f\u9078\u629e\u53ef\u80fd\u3002\u30d1\u30fc\u30bb\u30f3\u30c8\u306f94\u540d\u306e\u56de\u7b54\u8005\u3092\u30d9\u30fc\u30b9\u306b\u3057\u3066\u3044\u307e\u3059\u3002<\/em><\/figcaption><\/figure>\n\n\n\n

\u6027\u5225\u3001\u5e74\u9f62\u3001\u5730\u7406\u7684\u306a\u5206\u5e03\u306b\u304b\u304b\u308f\u3089\u305a\u3001\u56de\u7b54\u8005\u306e\u5927\u591a\u6570(77%)\u306f\u3001\u81ea\u5206\u306e\u5b50\u4f9b\u3092\u81e8\u5e8a\u8a66\u9a13\u306b\u767b\u9332\u3059\u308b\u3053\u3068\u306b\u5f37\u3044\u95a2\u5fc3\u3092\u793a\u3057\u307e\u3057\u305f\u3002\u4e00\u65b9\u3067\u300119%\u306f\u672a\u5b9a\u30014%\u306f\u53c2\u52a0\u3092\u5e0c\u671b\u3057\u3066\u3044\u307e\u305b\u3093\u3067\u3057\u305f\u3002<\/p>\n\n\n\n

\"\"
\u81e8\u5e8a\u8a66\u9a13\u53c2\u52a0\u306b\u5f71\u97ff\u3092\u4e0e\u3048\u308b\u4e3b\u306a\u8981\u56e0\uff1a <\/strong>\u5404\u56de\u7b54\u8005\u306f\u8907\u6570\u306e\u8981\u56e0\u3092\u9078\u629e\u53ef\u80fd\u3067\u3059\u3002\u5186\u306e\u5927\u304d\u3055\u3068\u30d1\u30fc\u30bb\u30f3\u30c6\u30fc\u30b8\u306f\u3001\u5404\u9078\u629e\u80a2\u3092\u9078\u3093\u3060\u56de\u7b54\u8005\uff08\u5408\u8a0894\u540d\uff09\u306b\u5360\u3081\u308b\u5272\u5408\u3092\u793a\u3057\u3066\u3044\u307e\u3059\u3002<\/em><\/figcaption><\/figure>\n\n\n\n

\u53c2\u52a0\u610f\u6b32\u306b\u5f71\u97ff\u3092\u4e0e\u3048\u308b\u8981\u56e0\u3092\u3088\u308a\u3088\u304f\u7406\u89e3\u3059\u308b\u305f\u3081\u3001\u5bb6\u65cf\u306b\u5bfe\u3057\u3066\u5b50\u4f9b\u3092\u81e8\u5e8a\u8a66\u9a13\u306b\u53c2\u52a0\u3055\u305b\u308b\u304b\u3069\u3046\u304b\u5224\u65ad\u3059\u308b\u969b\u306b\u3001\u91cd\u8981\u8996\u3059\u308b\u30dd\u30a4\u30f3\u30c8\u304c\u4f55\u304b\u3092\u5c0b\u306d\u307e\u3057\u305f\u3002\u6700\u3082\u591a\u304f\u9078\u3070\u308c\u305f\u306e\u306f\u3001\u6cbb\u7642\u3084\u624b\u7d9a\u304d\u306b\u4f34\u3046\u30ea\u30b9\u30af\u3067\u3057\u305f\u300294\u540d\u4e2d86\u540d\uff0891\uff05\uff09\u304c\u6319\u3052\u307e\u3057\u305f\u3002\u6b21\u3044\u3067\u3001\u6cbb\u7642\u306e\u6f5c\u5728\u7684\u306a\u5229\u76ca\u304c94\u540d\u4e2d77\u540d\uff0882\uff05\uff09\u3067\u7d9a\u304d\u307e\u3057\u305f\u3002\u8a66\u9a13\u306e\u5834\u6240\uff0860\u4ef6\u300164\uff05\uff09\u3001\u4ea4\u901a\u8cbb\u3084\u5bbf\u6cca\u8cbb\u306e\u652f\u63f4\uff0847\u4ef6\u300150\uff05\uff09\u3001\u8a66\u9a13\u671f\u9593\uff0837\u4ef6\u300139\uff05\uff09\u3092\u9078\u3076\u4eba\u306f\u3001\u305d\u308c\u307b\u3069\u591a\u304f\u306f\u3042\u308a\u307e\u305b\u3093\u3067\u3057\u305f\u3002<\/p>\n\n\n\n

\u307e\u3068\u3081<\/strong><\/p>\n\n\n\n

\u3053\u306e\u8abf\u67fb\u7d50\u679c\u306f\u3001MECP2\u91cd\u8907\u75c7\u5019\u7fa4\u306b\u7f79\u60a3\u3057\u305f\u5bb6\u65cf\u306e\u8996\u70b9\u3068\u512a\u5148\u4e8b\u9805\u306b\u95a2\u3059\u308b\u8cb4\u91cd\u306a\u6d1e\u5bdf\u3092\u63d0\u4f9b\u3059\u308b\u3082\u306e\u3068\u306a\u308a\u307e\u3057\u305f\u3002\u5e74\u9f62\u3001\u6027\u5225\u3001\u5730\u7406\u7684\u306a\u9055\u3044\u306b\u3082\u304b\u304b\u308f\u3089\u305a\u3001\u5bb6\u65cf\u306e\u5927\u591a\u6570\u304c\u81e8\u5e8a\u8a66\u9a13\u3078\u306e\u53c2\u52a0\u306b\u8208\u5473\u3092\u793a\u3057\u307e\u3057\u305f\u3002<\/p>\n\n\n\n

\u30ea\u30b9\u30af\u3068\u6f5c\u5728\u7684\u306a\u5229\u76ca\u304c\u610f\u601d\u6c7a\u5b9a\u306e\u969b\u306e\u6700\u3082\u91cd\u8981\u306a\u8981\u7d20\u3067\u3042\u308b\u4e00\u65b9\u3067\u3001\u8a66\u9a13\u5834\u6240\u3001\u79fb\u52d5\u624b\u6bb5\u3001\u8a66\u9a13\u671f\u9593\u306a\u3069\u306e\u8981\u7d20\u3082\u3001\u91cd\u8981\u5ea6\u306f\u3084\u3084\u4f4e\u3044\u3082\u306e\u306e\u3001\u7121\u8996\u3067\u304d\u306a\u3044\u8003\u616e\u4e8b\u9805\u3067\u3042\u308b\u3053\u3068\u304c\u308f\u304b\u308a\u307e\u3057\u305f\u3002<\/p>\n\n\n\n

ATTUNE\u8a66\u9a13\u3068HERO\u8a66\u9a13\u306f\u73fe\u5728\u3001\u30d2\u30c8\u3067\u306e\u521d\u56de\u30c7\u30fc\u30bf\u3092\u53ce\u96c6\u4e2d\u3067\u3042\u3059\u304c\u3001\u3053\u308c\u3089\u306e\u7591\u554f\u3084\u61f8\u5ff5\u306e\u3044\u304f\u3064\u304b\u304c\u3001\u6642\u9593\u3092\u304b\u3051\u3066\u89e3\u6d88\u3055\u308c\u308b\u3053\u3068\u3092\u671f\u5f85\u3057\u3066\u3044\u307e\u3059\u3002<\/p>\n\n\n\n

\u305d\u306e\u9593\u306b\u3001\u79c1\u305f\u3061\u306e\u30b3\u30df\u30e5\u30cb\u30c6\u30a3\u306f\u6b21\u306e\u30b9\u30c6\u30c3\u30d7\u306b\u5099\u3048\u308b\u5fc5\u8981\u304c\u3042\u308a\u307e\u3059\u3002\u521d\u671f\u306e\u81e8\u5e8a\u8a66\u9a13\u3067\u6709\u671b\u306a\u7d50\u679c\u304c\u51fa\u308c\u3070\u3001\u5f8c\u671f\u306e\u8a66\u9a13\u3084\u4e16\u754c\u7684\u306a\u6cbb\u7642\u30a2\u30af\u30bb\u30b9\u306e\u305f\u3081\u306b\u3001\u3088\u308a\u591a\u304f\u306e\u53c2\u52a0\u304c\u4e0d\u53ef\u6b20\u3068\u306a\u308a\u307e\u3059\u3002\u3053\u306e\u8abf\u67fb\u306f\u3001\u5bb6\u65cf\u306e\u3044\u308b\u5834\u6240\u3001\u5fc5\u8981\u306a\u652f\u63f4\u3001\u53c2\u52a0\u610f\u6b32\u304c\u3069\u306e\u7a0b\u5ea6\u3042\u308b\u304b\u3092\u628a\u63e1\u3057\u3001\u305d\u306e\u6e96\u5099\u3092\u652f\u63f4\u3059\u308b\u305f\u3081\u306b\u5b9f\u65bd\u3055\u308c\u307e\u3057\u305f\u3002<\/p>\n\n\n\n

\u3053\u306e\u8abf\u67fb\u306f\u5358\u306a\u308b\u30c7\u30fc\u30bf\u3067\u306f\u306a\u304f\u3001\u9032\u6b69\u306b\u5411\u3051\u305f\u6b21\u306e\u30b9\u30c6\u30c3\u30d7\u3092\u8e0f\u307f\u51fa\u3059\u6e96\u5099\u304c\u3067\u304d\u3066\u3044\u308b\u3001\u732e\u8eab\u7684\u3067\u30b0\u30ed\u30fc\u30d0\u30eb\u306b\u3064\u306a\u304c\u3063\u305f\u30b3\u30df\u30e5\u30cb\u30c6\u30a3\u306e\u610f\u601d\u3092\u53cd\u6620\u3057\u305f\u3082\u306e\u3067\u3059\u3002\u307e\u3060\u53c2\u52a0\u3055\u308c\u3066\u3044\u306a\u3044\u3054\u5bb6\u65cf\u306e\u305f\u3081\u306b\u3001\u30a2\u30f3\u30b1\u30fc\u30c8\u306f\u307e\u3060\u53d7\u4ed8\u4e2d\u3067\u3059\u3002\u58f0\u3092\u5c4a\u3051\u3001\u81e8\u5e8a\u7814\u7a76\u306e\u672a\u6765\u3092\u5171\u306b\u7bc9\u304f\u306e\u306b\u9045\u3059\u304e\u308b\u3053\u3068\u306f\u3042\u308a\u307e\u305b\u3093\u3002<\/p>\n\n\n\n

\u30a2\u30f3\u30b1\u30fc\u30c8\u306b\u7b54\u3048\u308b<\/a><\/p>","protected":false},"excerpt":{"rendered":"

Research on MECP2 Duplication Syndrome has taken an unprecedented step: entering the world of clinical trials. This is a spark of hope for many families who have worked tirelessly for years to provide a better future for their children. With the launch of early-stage clinical trials such as ATTUNE (ION440 by Ionis) in the U.S. and HERO (HG204 by HuidaGene) in China, we are witnessing a historic moment in the search for a treatment. These phase 1\/2 trials will help determine whether these investigational drugs are safe and potentially effective \u2013 but they are only the beginning. To prepare for what comes next \u2013 including larger, later-phase trials \u2013 we […]<\/p>\n","protected":false},"author":114,"featured_media":3557,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_jf_save_progress":"","footnotes":""},"categories":[23],"tags":[],"class_list":["post-3482","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-survey"],"_links":{"self":[{"href":"https:\/\/remeds.org\/ja\/wp-json\/wp\/v2\/posts\/3482","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/remeds.org\/ja\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/remeds.org\/ja\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/remeds.org\/ja\/wp-json\/wp\/v2\/users\/114"}],"replies":[{"embeddable":true,"href":"https:\/\/remeds.org\/ja\/wp-json\/wp\/v2\/comments?post=3482"}],"version-history":[{"count":5,"href":"https:\/\/remeds.org\/ja\/wp-json\/wp\/v2\/posts\/3482\/revisions"}],"predecessor-version":[{"id":3496,"href":"https:\/\/remeds.org\/ja\/wp-json\/wp\/v2\/posts\/3482\/revisions\/3496"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/remeds.org\/ja\/wp-json\/wp\/v2\/media\/3557"}],"wp:attachment":[{"href":"https:\/\/remeds.org\/ja\/wp-json\/wp\/v2\/media?parent=3482"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/remeds.org\/ja\/wp-json\/wp\/v2\/categories?post=3482"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/remeds.org\/ja\/wp-json\/wp\/v2\/tags?post=3482"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}