{"id":136660,"date":"2026-09-08T12:22:58","date_gmt":"2026-09-08T03:22:58","guid":{"rendered":"https:\/\/www.hiro-clinic.or.jp\/nipt\/?post_type=genetic-diseases&#038;p=136660"},"modified":"2026-09-08T12:22:58","modified_gmt":"2026-09-08T03:22:58","slug":"vlcad-deficiency-acadvl","status":"publish","type":"genetic-diseases","link":"https:\/\/www.hiro-clinic.or.jp\/nipt\/genetic-diseases2\/vlcad-deficiency-acadvl\/","title":{"rendered":"\u6975\u9577\u9396\u30a2\u30b7\u30ebCoA\u8131\u6c34\u7d20\u9175\u7d20\u6b20\u640d\u75c7\uff08VLCAD\u6b20\u640d\u75c7\uff09"},"content":{"rendered":"\n<h2 class=\"wp-block-heading\">\u6982\u8981<\/h2>\n\n\n\n<p>\u6975\u9577\u9396\u30a2\u30b7\u30ebCoA\u8131\u6c34\u7d20\u9175\u7d20\u6b20\u640d\u75c7\uff08VLCAD\u6b20\u640d\u75c7\uff09\u306f\u3001\u4f53\u306e\u306a\u304b\u3067\u300c\u9577\u3044\u8102\u80aa\u9178\u300d\u3092\u30a8\u30cd\u30eb\u30ae\u30fc\u306b\u5909\u3048\u308b\u306f\u305f\u3089\u304d\u304c\u5f31\u304f\u306a\u308b\u75c5\u6c17\u3067\u3059\u3002\u5e38\u67d3\u8272\u4f53\u6f5c\u6027\uff08\u52a3\u6027\uff09\u907a\u4f1d\u306e\u5f62\u5f0f\u3092\u3068\u308a\u3001<strong>ACADVL<\/strong> \u907a\u4f1d\u5b50\u306e\u5909\u5316\u304c\u539f\u56e0\u3068\u306a\u308a\u307e\u3059\u3002<\/p>\n\n\n\n<p>\u98df\u4e8b\u304c\u3068\u308c\u306a\u3044\u3068\u304d\u3084\u3001\u71b1\u304c\u51fa\u305f\u3068\u304d\u3001\u904b\u52d5\u3092\u9577\u304f\u7d9a\u3051\u305f\u3068\u304d\u306a\u3069\u3001\u4f53\u304c\u30a8\u30cd\u30eb\u30ae\u30fc\u3092\u5fc5\u8981\u3068\u3059\u308b\u5834\u9762\u3067\u75c7\u72b6\u304c\u73fe\u308c\u307e\u3059\u3002\u65e5\u672c\u3067\u306f\u65b0\u751f\u5150\u30de\u30b9\u30b9\u30af\u30ea\u30fc\u30cb\u30f3\u30b0\u306e\u5bfe\u8c61\u3067\u3001\u65e9\u304f\u898b\u3064\u3051\u3066\u5099\u3048\u308b\u3053\u3068\u304c\u3067\u304d\u307e\u3059\u3002<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">\u539f\u56e0<\/h2>\n\n\n\n<p><strong>ACADVL<\/strong> \u907a\u4f1d\u5b50\u306f\u3001\u6975\u9577\u9396\u30a2\u30b7\u30ebCoA\u8131\u6c34\u7d20\u9175\u7d20\u3068\u3044\u3046\u9175\u7d20\u306e\u8a2d\u8a08\u56f3\u3067\u3059\u3002\u3053\u306e\u9175\u7d20\u306f\u3001\u30df\u30c8\u30b3\u30f3\u30c9\u30ea\u30a2\u306e\u306a\u304b\u3067\u3001\u9396\u306e\u9577\u3044\u8102\u80aa\u9178\u3092\u5206\u89e3\u3057\u3066\u30a8\u30cd\u30eb\u30ae\u30fc\u306b\u5909\u3048\u308b\u6700\u521d\u306e\u6bb5\u968e\u3092\u62c5\u5f53\u3057\u3066\u3044\u307e\u3059\u3002<\/p>\n\n\n\n<p>\u3053\u306e\u9175\u7d20\u304c\u306f\u305f\u3089\u304b\u306a\u3044\u3068\u3001\u7d76\u98df\u6642\u306a\u3069\u306b\u8102\u80aa\u3092\u30a8\u30cd\u30eb\u30ae\u30fc\u3068\u3057\u3066\u4f7f\u3048\u305a\u3001\u4f4e\u8840\u7cd6\u306b\u304a\u3061\u3044\u308a\u307e\u3059\u3002\u540c\u6642\u306b\u3001\u5206\u89e3\u3055\u308c\u306a\u304b\u3063\u305f\u8102\u80aa\u9178\u304c\u5fc3\u81d3\u3084\u7b4b\u8089\u3001\u809d\u81d3\u306b\u305f\u307e\u3063\u3066\u969c\u5bb3\u3092\u8d77\u3053\u3057\u307e\u3059\u3002<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">\u75c7\u72b6<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>\u65b0\u751f\u5150\u30fb\u4e73\u5150\u671f\u767a\u75c7\u578b\uff08\u91cd\u75c7\uff09<\/strong>\uff1a\u5fc3\u7b4b\u75c7\u3001\u4e0d\u6574\u8108\u3001\u809d\u81d3\u306e\u816b\u5927\u3001\u4f4e\u8840\u7cd6\u3001\u7a81\u7136\u6b7b<\/li>\n\n\n\n<li><strong>\u5c0f\u5150\u671f\u767a\u75c7\u578b\uff08\u4e2d\u9593\u578b\uff09<\/strong>\uff1a\u767a\u71b1\u3084\u7d76\u98df\u3092\u304d\u3063\u304b\u3051\u3068\u3057\u305f\u4f4e\u30b1\u30c8\u30f3\u6027\u4f4e\u8840\u7cd6\u3001\u809d\u81d3\u306e\u816b\u5927<\/li>\n\n\n\n<li><strong>\u6210\u4eba\u671f\u767a\u75c7\u578b\uff08\u8efd\u75c7\uff09<\/strong>\uff1a\u904b\u52d5\u5f8c\u306e\u7b4b\u8089\u75db\u3001\u7b4b\u529b\u4f4e\u4e0b\u3001\u6a2a\u7d0b\u7b4b\u878d\u89e3\u75c7\uff08\u30df\u30aa\u30b0\u30ed\u30d3\u30f3\u5c3f\u30fb\u8336\u8272\u3044\u5c3f\uff09<\/li>\n\n\n\n<li>\u304b\u305c\u3092\u3072\u3044\u305f\u3068\u304d\u3084\u3001\u9577\u6642\u9593\u98df\u4e8b\u3092\u3068\u3089\u306a\u304b\u3063\u305f\u3068\u304d\u306b\u60aa\u5316\u3057\u3084\u3059\u3044<\/li>\n\n\n<\/ul>\n\n\n\n<h2 class=\"wp-block-heading\">\u691c\u67fb\u30fb\u8a3a\u65ad<\/h2>\n\n\n\n<p>\u65b0\u751f\u5150\u30de\u30b9\u30b9\u30af\u30ea\u30fc\u30cb\u30f3\u30b0\u3067\u3001\u8840\u6db2\u4e2d\u306eC14:1\uff08\u30c6\u30c8\u30e9\u30c7\u30bb\u30ce\u30a4\u30eb\u30ab\u30eb\u30cb\u30c1\u30f3\uff09\u304c\u9ad8\u3044\u3053\u3068\u304b\u3089\u7591\u308f\u308c\u307e\u3059\u3002\u78ba\u5b9a\u306b\u306f <strong>ACADVL<\/strong> \u907a\u4f1d\u5b50\u306e\u89e3\u6790\u3084\u3001\u57f9\u990a\u3057\u305f\u76ae\u819a\u306e\u7d30\u80de\u3067\u306e\u9175\u7d20\u6d3b\u6027\u306e\u6e2c\u5b9a\u3092\u884c\u3044\u307e\u3059\u3002<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">\u6cbb\u7642<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>\u7d76\u98df\u3092\u907f\u3051\u308b<\/strong>\uff08\u5e74\u9f62\u306b\u5fdc\u3058\u305f\u9593\u9694\u3067\u5fc5\u305a\u98df\u4e8b\u3092\u3068\u308b\uff09<\/li>\n\n\n\n<li>\u9577\u9396\u8102\u80aa\u3092\u63a7\u3048\u3001<strong>\u4e2d\u9396\u8102\u80aa\u9178\uff08MCT\uff09<\/strong>\u3092\u7528\u3044\u305f\u98df\u4e8b\u7642\u6cd5<\/li>\n\n\n\n<li>\u4f53\u8abf\u304c\u60aa\u3044\u3068\u304d\u306f\u65e9\u3081\u306b\u533b\u7642\u6a5f\u95a2\u3092\u53d7\u8a3a\u3057\u3001\u30d6\u30c9\u30a6\u7cd6\u306e\u70b9\u6ef4\u3092\u884c\u3046\uff08\u30b7\u30c3\u30af\u30c7\u30a4\u5bfe\u5fdc\uff09<\/li>\n\n\n\n<li>\u30ab\u30eb\u30cb\u30c1\u30f3\u306e\u88dc\u5145\u304c\u691c\u8a0e\u3055\u308c\u308b\u3053\u3068\u304c\u3042\u308a\u307e\u3059<\/li>\n\n\n\n<li>\u6fc0\u3057\u3044\u904b\u52d5\u3084\u9577\u6642\u9593\u306e\u904b\u52d5\u3092\u907f\u3051\u308b<\/li>\n\n\n<\/ul>\n\n\n\n<h2 class=\"wp-block-heading\">\u3053\u306e\u75be\u60a3\u306f\u691c\u67fb\u3067\u308f\u304b\u308a\u307e\u3059<\/h2>\n\n\n\n<p>\u3053\u306e\u75be\u60a3\u306f\u3001\u539f\u56e0\u3068\u306a\u308b <strong>ACADVL<\/strong> \u907a\u4f1d\u5b50\u3092\u8abf\u3079\u308b\u3053\u3068\u3067\u3001\u767a\u75c7\u524d\u3084\u51fa\u751f\u524d\u306b\u898b\u3064\u3051\u308b\u3053\u3068\u304c\u3067\u304d\u307e\u3059\u3002\u30d2\u30ed\u30af\u30ea\u30cb\u30c3\u30afNIPT\u3067\u306f\u6b21\u306e2\u3064\u306e\u691c\u67fb\u3092\u3054\u7528\u610f\u3057\u3066\u3044\u307e\u3059\u3002<\/p>\n\n\n\n<h3 class=\"wp-block-heading\">\u30a8\u30af\u30bd\u30fc\u30e0\u89e3\u6790\u3092\u7528\u3044\u305f\u691c\u67fb\uff081,200\u7a2e\u985e\u4ee5\u4e0a\uff09<\/h3>\n\n\n\n<p>\u30a8\u30af\u30bd\u30fc\u30e0\u89e3\u6790\u306f\u3001\u907a\u4f1d\u5b50\u306e\u3046\u3061\u305f\u3093\u3071\u304f\u8cea\u306e\u8a2d\u8a08\u56f3\u306b\u3042\u305f\u308b\u90e8\u5206\uff08\u30a8\u30af\u30bd\u30f3\uff09\u3092\u307e\u3068\u3081\u3066\u8aad\u307f\u53d6\u308b\u691c\u67fb\u3067\u3059\u3002\u3053\u306e\u89e3\u6790\u3092\u884c\u3063\u305f\u3046\u3048\u3067\u3001<strong>ACADVL<\/strong> \u907a\u4f1d\u5b50\u3092\u542b\u3080<strong>1,200\u7a2e\u985e\u4ee5\u4e0a<\/strong>\u306e\u907a\u4f1d\u5b50\u3092\u5bfe\u8c61\u306b\u8abf\u3079\u3001<strong>\u3053\u306e\u75be\u60a3\u306e\u539f\u56e0\u3068\u306a\u308b\u5909\u5316\u3092\u691c\u51fa\u3059\u308b\u3053\u3068\u304c\u53ef\u80fd\u3067\u3059<\/strong>\u3002\u56fd\u5185\u306e\u691c\u67fb\u6240\u3067\u884c\u3044\u3001\u7d50\u679c\u307e\u3067\u306e\u671f\u9593\u306f5\u9031\u9593\u301c7\u9031\u9593\u3067\u3059\u3002<\/p>\n\n\n\n<h3 class=\"wp-block-heading\">\u30ad\u30e3\u30ea\u30a2\u30b9\u30af\u30ea\u30fc\u30cb\u30f3\u30b0\u30c6\u30b9\u30c8\uff08228\u7a2e\u985e\uff09<\/h3>\n\n\n\n<p>\u5e38\u67d3\u8272\u4f53\u6f5c\u6027\uff08\u52a3\u6027\uff09\u907a\u4f1d\u306e\u75be\u60a3\u306f\u3001\u3054\u4e21\u89aa\u304c\u3069\u3061\u3089\u3082\u75c7\u72b6\u306e\u306a\u3044\u300c\u30ad\u30e3\u30ea\u30a2\uff08\u4fdd\u56e0\u8005\uff09\u300d\u3067\u3042\u308b\u3068\u304d\u306b\u3001<strong>4\u5206\u306e1\u306e\u78ba\u7387<\/strong>\u3067\u304a\u5b50\u3055\u307e\u304c\u767a\u75c7\u3057\u307e\u3059\u3002\u30ad\u30e3\u30ea\u30a2\u30b9\u30af\u30ea\u30fc\u30cb\u30f3\u30b0\u30c6\u30b9\u30c8\u3067\u306f\u3001\u3054\u592b\u5a66\u304c\u3053\u306e\u75be\u60a3\u306e\u30ad\u30e3\u30ea\u30a2\u304b\u3069\u3046\u304b\u3092\u982c\u306e\u5185\u5074\u306e\u7c98\u819c\u304b\u3089\u8abf\u3079\u3001<strong>\u304a\u5b50\u3055\u307e\u306b\u53d7\u3051\u7d99\u304c\u308c\u308b\u53ef\u80fd\u6027\u3092\u3001\u598a\u5a20\u524d\u3084\u51fa\u751f\u524d\u306b\u4e88\u6e2c\u3059\u308b\u3053\u3068\u304c\u3067\u304d\u307e\u3059<\/strong>\u3002<\/p>\n\n\n\n<p>\u691c\u67fb\u306e\u5185\u5bb9\u30fb\u8cbb\u7528\u30fb\u7d50\u679c\u307e\u3067\u306e\u671f\u9593\u306f <a href=\"\/nipt\/nipt\/autosomal-recessive-inheritance\/\">\u30ad\u30e3\u30ea\u30a2\u30b9\u30af\u30ea\u30fc\u30cb\u30f3\u30b0\u30c6\u30b9\u30c8228<\/a> \u306e\u30da\u30fc\u30b8\u3092\u3054\u89a7\u304f\u3060\u3055\u3044\u3002<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">\u5c02\u9580\u7684\u306a\u89e3\u8aac<\/h2>\n\n\n\n<p>VLCAD\u6b20\u640d\u75c7\u306f <strong>ACADVL<\/strong>\uff0817p13.1\uff09\u306e\u4e21\u30a2\u30ec\u30eb\u5909\u7570\u306b\u3088\u308a\u751f\u3058\u308b\u3001\u9577\u9396\u8102\u80aa\u9178\u03b2\u9178\u5316\u7570\u5e38\u75c7\u3067\u3042\u308b\u3002VLCAD\u306f\u30df\u30c8\u30b3\u30f3\u30c9\u30ea\u30a2\u5185\u819c\u306b\u5c40\u5728\u3057\u3001\u70ad\u7d20\u657014\u301c20\u306e\u30a2\u30b7\u30ebCoA\u306e\u521d\u767a\u8131\u6c34\u7d20\u53cd\u5fdc\u3092\u89e6\u5a92\u3059\u308b\u3002<\/p>\n\n\n\n<p>\u81e8\u5e8a\u578b\u306f\u6b8b\u5b58\u9175\u7d20\u6d3b\u6027\u3068\u76f8\u95a2\u3057\u3001(1) \u65b0\u751f\u5150\u671f\u767a\u75c7\u306e\u91cd\u75c7\u578b\uff08\u5fc3\u7b4b\u75c7\u30fb\u4e0d\u6574\u8108\u30fb\u7a81\u7136\u6b7b\uff09\u3001(2) \u4e73\u5e7c\u5150\u671f\u767a\u75c7\u306e\u809d\u578b\uff08\u4f4e\u30b1\u30c8\u30f3\u6027\u4f4e\u8840\u7cd6\uff09\u3001(3) \u9045\u767a\u6027\u306e\u30df\u30aa\u30d1\u30c1\u30fc\u578b\uff08\u904b\u52d5\u8a98\u767a\u6027\u6a2a\u7d0b\u7b4b\u878d\u89e3\u75c7\uff09\u306b\u5927\u5225\u3055\u308c\u308b\u3002<\/p>\n\n\n\n<p>\u30a2\u30b7\u30eb\u30ab\u30eb\u30cb\u30c1\u30f3\u5206\u6790\u3067C14:1\u306e\u4e0a\u6607\u304c\u7279\u5fb4\u7684\u3067\u3001C14:1\/C12:1\u6bd4\u304c\u8a3a\u65ad\u306b\u6709\u7528\u3067\u3042\u308b\u3002\u65e5\u672c\u3067\u306f2014\u5e74\u3088\u308a\u65b0\u751f\u5150\u30de\u30b9\u30b9\u30af\u30ea\u30fc\u30cb\u30f3\u30b0\u306e\u4e00\u6b21\u5bfe\u8c61\u75be\u60a3\u306b\u542b\u307e\u308c\u308b\u3002<\/p>\n\n\n\n<p>\u6cbb\u7642\u306e\u67f1\u306f\u7570\u5316\u4ea2\u9032\u306e\u56de\u907f\u3067\u3042\u308a\u3001\u30b7\u30c3\u30af\u30c7\u30a4\u306b\u306f\u65e9\u671f\u306e\u30d6\u30c9\u30a6\u7cd6\u8f38\u6db2\uff08GIR 6\u301c8 mg\/kg\/\u5206\uff09\u3092\u884c\u3046\u3002\u4e2d\u9396\u8102\u80aa\u9178\uff08MCT\uff09\u306fVLCAD\u3092\u4ecb\u3055\u305a\u4ee3\u8b1d\u3055\u308c\u308b\u305f\u3081\u30a8\u30cd\u30eb\u30ae\u30fc\u6e90\u3068\u3057\u3066\u6709\u7528\u3067\u3042\u308b\u3002\u8fd1\u5e74\u3001\u30c8\u30ea\u30d8\u30d7\u30bf\u30ce\u30a4\u30f3\uff08\u5947\u6570\u9396\u8102\u80aa\u9178\uff09\u304c\u9577\u9396\u8102\u80aa\u9178\u9178\u5316\u7570\u5e38\u75c7\u306b\u5bfe\u3057\u3066\u627f\u8a8d\u3055\u308c\u3066\u3044\u308b\u3002<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">\u3088\u304f\u3042\u308b\u8cea\u554f<\/h2>\n\n\n\n<h3 class=\"wp-block-heading\">Q. \u3069\u3093\u306a\u3068\u304d\u306b\u75c7\u72b6\u304c\u51fa\u3084\u3059\u3044\u3067\u3059\u304b\uff1f<\/h3>\n\n\n\n<p>A. \u98df\u4e8b\u3092\u3068\u308c\u306a\u3044\u3068\u304d\u3001\u71b1\u304c\u51fa\u305f\u3068\u304d\u3001\u4e0b\u75e2\u3084\u5614\u5410\u304c\u7d9a\u304f\u3068\u304d\u3001\u9577\u3044\u6642\u9593\u306e\u904b\u52d5\u3092\u3057\u305f\u3068\u304d\u3067\u3059\u3002\u3053\u3046\u3057\u305f\u3068\u304d\u306f\u65e9\u3081\u306b\u53d7\u8a3a\u3057\u3066\u304f\u3060\u3055\u3044\u3002<\/p>\n\n\n\n<h3 class=\"wp-block-heading\">Q. \u98df\u4e8b\u306f\u3069\u3046\u3059\u308c\u3070\u3088\u3044\u3067\u3059\u304b\uff1f<\/h3>\n\n\n\n<p>A. \u9577\u6642\u9593\u306e\u7d76\u98df\u3092\u907f\u3051\u3001\u9577\u9396\u306e\u8102\u80aa\u3092\u63a7\u3048\u307e\u3059\u3002\u4e2d\u9396\u8102\u80aa\u9178\uff08MCT\uff09\u3092\u7528\u3044\u305f\u98df\u4e8b\u304c\u4f7f\u308f\u308c\u307e\u3059\u3002\u7ba1\u7406\u6804\u990a\u58eb\u3068\u4e00\u7dd2\u306b\u8a08\u753b\u3092\u7acb\u3066\u307e\u3059\u3002<\/p>\n\n\n\n<h3 class=\"wp-block-heading\">Q. \u904b\u52d5\u306f\u3057\u3066\u3082\u3088\u3044\u3067\u3059\u304b\uff1f<\/h3>\n\n\n\n<p>A. \u8efd\u3044\u904b\u52d5\u306f\u53ef\u80fd\u3067\u3059\u304c\u3001\u9577\u6642\u9593\u306e\u6fc0\u3057\u3044\u904b\u52d5\u306f\u7b4b\u8089\u304c\u58ca\u308c\u308b\u539f\u56e0\u306b\u306a\u308a\u307e\u3059\u3002\u62c5\u5f53\u306e\u5148\u751f\u3068\u3054\u76f8\u8ac7\u304f\u3060\u3055\u3044\u3002<\/p>\n\n\n\n<h3 class=\"wp-block-heading\">Q. \u65b0\u751f\u5150\u30de\u30b9\u30b9\u30af\u30ea\u30fc\u30cb\u30f3\u30b0\u3067\u898b\u3064\u304b\u308a\u307e\u3057\u305f\u3002\u3059\u3050\u75c7\u72b6\u304c\u51fa\u307e\u3059\u304b\uff1f<\/h3>\n\n\n\n<p>A. \u75c7\u72b6\u304c\u51fa\u308b\u304b\u3069\u3046\u304b\u3001\u3044\u3064\u51fa\u308b\u304b\u306b\u306f\u5e45\u304c\u3042\u308a\u307e\u3059\u3002\u65e9\u304f\u898b\u3064\u304b\u3063\u305f\u3053\u3068\u3067\u3001\u767a\u4f5c\u3092\u9632\u3050\u5099\u3048\u304c\u3067\u304d\u307e\u3059\u3002<\/p>\n\n\n\n<h3 class=\"wp-block-heading\">Q. \u8336\u8272\u3044\u5c3f\u304c\u51fa\u307e\u3057\u305f\u3002<\/h3>\n\n\n\n<p>A. \u7b4b\u8089\u304c\u58ca\u308c\u3066\u3044\u308b\u30b5\u30a4\u30f3\uff08\u6a2a\u7d0b\u7b4b\u878d\u89e3\u75c7\uff09\u306e\u53ef\u80fd\u6027\u304c\u3042\u308a\u307e\u3059\u3002\u3059\u3050\u306b\u53d7\u8a3a\u3057\u3066\u304f\u3060\u3055\u3044\u3002<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">\u53c2\u8003\u6587\u732e<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Aoyama T, Souri M, Ushikubo S, et al. Purification of human very-long-chain acyl-coenzyme A dehydrogenase and characterization of its deficiency in seven patients. <em>J Clin Invest<\/em>. 1995;95(6):2465-2473.<\/li>\n\n\n\n<li>Leslie ND, Saenz-Ayala S. Very Long-Chain Acyl-Coenzyme A Dehydrogenase Deficiency. In: <em>GeneReviews\u00ae<\/em>. University of Washington, Seattle.<\/li>\n\n\n\n<li>Arnold GL, Van Hove J, Freedenberg D, et al. A Delphi clinical practice protocol for the management of very long chain acyl-CoA dehydrogenase deficiency. <em>Mol Genet Metab<\/em>. 2009;96(3):85-90.<\/li>\n\n\n\n<li>Vockley J, Burton B, Berry GT, et al. Results from a 78-week, single-arm, open-label phase 2 study to evaluate UX007 in pediatric and adult patients with severe long-chain fatty acid oxidation disorders. <em>J Inherit Metab Dis<\/em>. 2019;42(1):169-177.<\/li>\n\n\n\n<li>OMIM #201475 VLCAD deficiency<\/li>\n\n\n<\/ul>\n","protected":false},"featured_media":0,"menu_order":0,"template":"page_gd.php","class_list":["post-136660","genetic-diseases","type-genetic-diseases","status-publish","hentry"],"acf":[],"_links":{"self":[{"href":"https:\/\/www.hiro-clinic.or.jp\/nipt\/wp-json\/wp\/v2\/genetic-diseases\/136660","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.hiro-clinic.or.jp\/nipt\/wp-json\/wp\/v2\/genetic-diseases"}],"about":[{"href":"https:\/\/www.hiro-clinic.or.jp\/nipt\/wp-json\/wp\/v2\/types\/genetic-diseases"}],"version-history":[{"count":1,"href":"https:\/\/www.hiro-clinic.or.jp\/nipt\/wp-json\/wp\/v2\/genetic-diseases\/136660\/revisions"}],"predecessor-version":[{"id":137030,"href":"https:\/\/www.hiro-clinic.or.jp\/nipt\/wp-json\/wp\/v2\/genetic-diseases\/136660\/revisions\/137030"}],"wp:attachment":[{"href":"https:\/\/www.hiro-clinic.or.jp\/nipt\/wp-json\/wp\/v2\/media?parent=136660"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}