{"id":56199,"date":"2026-02-25T06:10:00","date_gmt":"2026-02-25T06:10:00","guid":{"rendered":"https:\/\/www.globalresearch.co.kr\/insight\/pompe-diseases-market-mordor\/"},"modified":"2026-09-18T05:37:24","modified_gmt":"2026-09-17T20:37:24","slug":"pompe-diseases-market-mordor","status":"publish","type":"post","link":"https:\/\/www.globalresearch.co.kr\/insight\/pompe-diseases-market-mordor\/","title":{"rendered":"\uc138\uacc4\uc758 \ud3fc\ud398\ubcd1 \uc2dc\uc7a5 \uaddc\ubaa8 \ubc0f \uc810\uc720\uc728 \ubd84\uc11d &#8211; \uc131\uc7a5 \ub3d9\ud5a5 \ubc0f \uc804\ub9dd (2026-2031)"},"content":{"rendered":"<p><img decoding=\"async\" src=\"https:\/\/www.globalresearch.co.kr\/insight\/wp-content\/uploads\/2026\/02\/pompe-diseases-market-1750592773298-major-players.webp\" alt=\"Chart\" class=\"aligncenter size-full\" \/><\/p>\n<p><img decoding=\"async\" src=\"https:\/\/www.globalresearch.co.kr\/insight\/wp-content\/uploads\/2026\/02\/pompe-diseases-market-size-image-1771001732227.webp\" alt=\"Chart\" class=\"aligncenter size-full\" \/><\/p>\n<p class=\"toc-level-1 toc-content-heading\" id=\"toc-content-heading-0\">1. \uc11c\ub860<\/p>\n<ul>\n<li class=\"toc-level-2\">1.1 \uc5f0\uad6c \uac00\uc815 \ubc0f \uc2dc\uc7a5 \uc815\uc758<\/li>\n<li class=\"toc-level-2\">1.2 \uc5f0\uad6c \ubc94\uc704<\/li>\n<\/ul>\n<p class=\"toc-level-1 toc-content-heading\" id=\"toc-content-heading-1\">2. \uc5f0\uad6c \ubc29\ubc95\ub860<\/p>\n<p class=\"toc-level-1 toc-content-heading\" id=\"toc-content-heading-2\">3. \uc694\uc57d<\/p>\n<p class=\"toc-level-1 toc-content-heading\" id=\"toc-content-heading-3\">4. \uc2dc\uc7a5 \ud658\uacbd<\/p>\n<ul>\n<li class=\"toc-level-2\">4.1 \uc2dc\uc7a5 \uac1c\uc694<\/li>\n<li class=\"toc-level-2\">4.2 \uc2dc\uc7a5 \ub3d9\uc778\n<ul>\n<li class=\"toc-level-3\">4.2.1 \uc0c8\ub85c\uc6b4 2\uc138\ub300 ERT \ucd9c\uc2dc<\/li>\n<li class=\"toc-level-3\">4.2.2 \uc2e0\uc0dd\uc544 \uc120\ubcc4\uac80\uc0ac \uc758\ubb34 \ud655\ub300<\/li>\n<li class=\"toc-level-3\">4.2.3 \uc784\uc0c1 \uc2dc\ud5d8 \uc911\uc778 mRNA \uae30\ubc18 ERT \ud50c\ub7ab\ud3fc<\/li>\n<li class=\"toc-level-3\">4.2.4 \uc911\uad6d\uc758 \ud76c\uadc0 \uc9c8\ud658 \uc0c1\ud658 \uac00\uc18d\ud654<\/li>\n<li class=\"toc-level-3\">4.2.5 \uc720\uc804\uc790 \uce58\ub8cc \uc790\uae08 \uae09\uc99d<\/li>\n<li class=\"toc-level-3\">4.2.6 \uae00\ub85c\ubc8c \ud76c\uadc0\uc758\uc57d\ud488 \uc778\uc13c\ud2f0\ube0c<\/li>\n<\/ul>\n<\/li>\n<li class=\"toc-level-2\">4.3 \uc2dc\uc7a5 \uc81c\uc57d\n<ul>\n<li class=\"toc-level-3\">4.3.1 \ub192\uc740 \uce58\ub8cc \ube44\uc6a9<\/li>\n<li class=\"toc-level-3\">4.3.2 \uc9c4\ub2e8 \uc9c0\uc5f0 \ubc0f \ub0ae\uc740 \uc778\uc2dd<\/li>\n<li class=\"toc-level-3\">4.3.3 \ubc14\uc774\ub7ec\uc2a4 \ubca1\ud130 \uc81c\uc870 \ubcd1\ubaa9 \ud604\uc0c1<\/li>\n<li class=\"toc-level-3\">4.3.4 \ubbf8\uad6d\/EU\uc758 \uac00\uce58 \uae30\ubc18 \uac00\uaca9 \uc555\ub825<\/li>\n<\/ul>\n<\/li>\n<li class=\"toc-level-2\">4.4 \uac00\uce58 \/ \uacf5\uae09\ub9dd \ubd84\uc11d<\/li>\n<li class=\"toc-level-2\">4.5 \uaddc\uc81c \ud658\uacbd<\/li>\n<li class=\"toc-level-2\">4.6 \uae30\uc220 \uc804\ub9dd<\/li>\n<li class=\"toc-level-2\">4.7 \ud3ec\ud130\uc758 5\uac00\uc9c0 \uacbd\uc7c1 \uc694\uc778 \ubd84\uc11d\n<ul>\n<li class=\"toc-level-3\">4.7.1 \uc2e0\uaddc \uc9c4\uc785\uc790\uc758 \uc704\ud611<\/li>\n<li class=\"toc-level-3\">4.7.2 \uad6c\ub9e4\uc790\/\uc18c\ube44\uc790\uc758 \uad50\uc12d\ub825<\/li>\n<li class=\"toc-level-3\">4.7.3 \uacf5\uae09\uc5c5\uccb4\uc758 \uad50\uc12d\ub825<\/li>\n<li class=\"toc-level-3\">4.7.4 \ub300\uccb4 \uc81c\ud488\uc758 \uc704\ud611<\/li>\n<li class=\"toc-level-3\">4.7.5 \uacbd\uc7c1 \uac15\ub3c4<\/li>\n<\/ul>\n<\/li>\n<\/ul>\n<p class=\"toc-level-1 toc-content-heading\" id=\"toc-content-heading-4\">5. \uc2dc\uc7a5 \uaddc\ubaa8 \ubc0f \uc131\uc7a5 \uc608\uce21 (\uac00\uce58)<\/p>\n<ul>\n<li class=\"toc-level-2\">5.1 \ud22c\uc5ec \uacbd\ub85c\ubcc4\n<ul>\n<li class=\"toc-level-3\">5.1.1 \uacbd\uad6c<\/li>\n<li class=\"toc-level-3\">5.1.2 \ube44\uacbd\uad6c<\/li>\n<\/ul>\n<\/li>\n<li class=\"toc-level-2\">5.2 \uce58\ub8cc \uc720\ud615\ubcc4\n<ul>\n<li class=\"toc-level-3\">5.2.1 \ud6a8\uc18c \ub300\uccb4 \uc694\ubc95 (ERT)<\/li>\n<li class=\"toc-level-3\">5.2.2 \ubcd1\uc6a9 \uc694\ubc95<\/li>\n<li class=\"toc-level-3\">5.2.3 \uae30\ud0c0 \uc694\ubc95<\/li>\n<\/ul>\n<\/li>\n<li class=\"toc-level-2\">5.3 \uc9c8\ubcd1 \ubc1c\ubcd1 \uc720\ud615\ubcc4\n<ul>\n<li class=\"toc-level-3\">5.3.1 \uc601\uc544 \ubc1c\ubcd1 \ud3fc\ud398\ubcd1 (IOPD)<\/li>\n<li class=\"toc-level-3\">5.3.2 \ud6c4\uae30 \ubc1c\ubcd1 \ud3fc\ud398\ubcd1 (LOPD)<\/li>\n<\/ul>\n<\/li>\n<li class=\"toc-level-2\">5.4 \uc720\ud1b5 \ucc44\ub110\ubcc4\n<ul>\n<li class=\"toc-level-3\">5.4.1 \ubcd1\uc6d0 \uc57d\uad6d<\/li>\n<li class=\"toc-level-3\">5.4.2 \uc18c\ub9e4 \uc57d\uad6d<\/li>\n<li class=\"toc-level-3\">5.4.3 \uc628\ub77c\uc778 \uc57d\uad6d<\/li>\n<li class=\"toc-level-3\">5.4.4 \uae30\ud0c0<\/li>\n<\/ul>\n<\/li>\n<li class=\"toc-level-2\">5.5 \uc9c0\uc5ed\n<ul>\n<li class=\"toc-level-3\">5.5.1 \ubd81\ubbf8<\/li>\n<li class=\"toc-level-4\">5.5.1.1 \ubbf8\uad6d<\/li>\n<li class=\"toc-level-4\">5.5.1.2 \uce90\ub098\ub2e4<\/li>\n<li class=\"toc-level-4\">5.5.1.3 \uba55\uc2dc\ucf54<\/li>\n<li class=\"toc-level-3\">5.5.2 \uc720\ub7fd<\/li>\n<li class=\"toc-level-4\">5.5.2.1 \ub3c5\uc77c<\/li>\n<li class=\"toc-level-4\">5.5.2.2 \uc601\uad6d<\/li>\n<li class=\"toc-level-4\">5.5.2.3 \ud504\ub791\uc2a4<\/li>\n<li class=\"toc-level-4\">5.5.2.4 \uc774\ud0c8\ub9ac\uc544<\/li>\n<li class=\"toc-level-4\">5.5.2.5 \uc2a4\ud398\uc778<\/li>\n<li class=\"toc-level-4\">5.5.2.6 \uae30\ud0c0 \uc720\ub7fd<\/li>\n<li class=\"toc-level-3\">5.5.3 \uc544\uc2dc\uc544 \ud0dc\ud3c9\uc591<\/li>\n<li class=\"toc-level-4\">5.5.3.1 \uc911\uad6d<\/li>\n<li class=\"toc-level-4\">5.5.3.2 \uc77c\ubcf8<\/li>\n<li class=\"toc-level-4\">5.5.3.3 \uc778\ub3c4<\/li>\n<li class=\"toc-level-4\">5.5.3.4 \ud638\uc8fc<\/li>\n<li class=\"toc-level-4\">5.5.3.5 \ub300\ud55c\ubbfc\uad6d<\/li>\n<li class=\"toc-level-4\">5.5.3.6 \uae30\ud0c0 \uc544\uc2dc\uc544 \ud0dc\ud3c9\uc591<\/li>\n<li class=\"toc-level-3\">5.5.4 \uc911\ub3d9 \ubc0f \uc544\ud504\ub9ac\uce74<\/li>\n<li class=\"toc-level-4\">5.5.4.1 GCC<\/li>\n<li class=\"toc-level-4\">5.5.4.2 \ub0a8\uc544\ud504\ub9ac\uce74 \uacf5\ud654\uad6d<\/li>\n<li class=\"toc-level-4\">5.5.4.3 \uae30\ud0c0 \uc911\ub3d9 \ubc0f \uc544\ud504\ub9ac\uce74<\/li>\n<li class=\"toc-level-3\">5.5.5 \ub0a8\ubbf8<\/li>\n<li class=\"toc-level-4\">5.5.5.1 \ube0c\ub77c\uc9c8<\/li>\n<li class=\"toc-level-4\">5.5.5.2 \uc544\ub974\ud5e8\ud2f0\ub098<\/li>\n<li class=\"toc-level-4\">5.5.5.3 \uae30\ud0c0 \ub0a8\ubbf8<\/li>\n<\/ul>\n<\/li>\n<\/ul>\n<p class=\"toc-level-1 toc-content-heading\" id=\"toc-content-heading-5\">6. \uacbd\uc7c1 \ud658\uacbd<\/p>\n<ul>\n<li class=\"toc-level-2\">6.1 \uc2dc\uc7a5 \uc9d1\uc911\ub3c4<\/li>\n<li class=\"toc-level-2\">6.2 \uc2dc\uc7a5 \uc810\uc720\uc728 \ubd84\uc11d<\/li>\n<li class=\"toc-level-2\">6.3 \uae30\uc5c5 \ud504\ub85c\ud544 (\uae00\ub85c\ubc8c \uac1c\uc694, \uc2dc\uc7a5 \uac1c\uc694, \ud575\uc2ec \ubd80\ubb38, \uc7ac\ubb34 \uc815\ubcf4(\uac00\ub2a5\ud55c \uacbd\uc6b0), \uc804\ub7b5 \uc815\ubcf4, \uc2dc\uc7a5 \uc21c\uc704\/\uc810\uc720\uc728, \uc81c\ud488 \ubc0f \uc11c\ube44\uc2a4, \ucd5c\uadfc \uac1c\ubc1c \ud3ec\ud568)\n<ul>\n<li class=\"toc-level-3\">6.3.1 \uc0ac\ub178\ud53c<\/li>\n<li class=\"toc-level-3\">6.3.2 \uc544\ubbf8\ucfe0\uc2a4 \ud14c\ub77c\ud4e8\ud2f1\uc2a4<\/li>\n<li class=\"toc-level-3\">6.3.3 \uc544\uc2a4\ud154\ub77c\uc2a4 \ud30c\ub9c8 \/ \uc624\ub374\ud14c\uc2a4<\/li>\n<li class=\"toc-level-3\">6.3.4 \uc544\uc2a4\ud074\ub808\ud53c\uc624\uc2a4 \ubc14\uc774\uc624\ud30c\ub9c8\uc288\ud2f0\uceec (AskBio)<\/li>\n<li class=\"toc-level-3\">6.3.5 \uc81c\ub124\ud1a4<\/li>\n<li class=\"toc-level-3\">6.3.6 \uc624\ucc28\ub4dc \ud14c\ub77c\ud4e8\ud2f1\uc2a4<\/li>\n<li class=\"toc-level-3\">6.3.7 \uc2dc\uc624\ub178\uae30 \uc81c\uc57d \uc8fc\uc2dd\ud68c\uc0ac<\/li>\n<li class=\"toc-level-3\">6.3.8 \ubca0\uc774\uc9d5 \uc9c4\ud06c\ub798\ub4e4 \ud14c\ud06c\ub180\ub85c\uc9c0 Co., Ltd.<\/li>\n<li class=\"toc-level-3\">6.3.9 \uba54\uc774\uc988 \ud14c\ub77c\ud4e8\ud2f1\uc2a4 Inc.<\/li>\n<li class=\"toc-level-3\">6.3.10 \uc5d1\uc11c\uce74\uc778 \ucf54\ud37c\ub808\uc774\uc158<\/li>\n<\/ul>\n<\/li>\n<\/ul>\n<p class=\"toc-level-1 toc-content-heading\" id=\"toc-content-heading-6\">7. \uc2dc\uc7a5 \uae30\ud68c \ubc0f \ubbf8\ub798 \uc804\ub9dd<\/p>\n","protected":false},"excerpt":{"rendered":"<p>Mordor Intelligence \ubcf4\uace0\uc11c\uc5d0 \ub530\ub974\uba74, \ud3fc\ud398\ubcd1 \uce58\ub8cc \uc2dc\uc7a5\uc740 2026\ub144 20.4\uc5b5 \ub2ec\ub7ec\uc5d0\uc11c 2031\ub144 28.6\uc5b5 \ub2ec\ub7ec \uaddc\ubaa8\ub85c \uc131\uc7a5\ud560 \uac83\uc73c\ub85c \uc608\uc0c1\ub418\uba70, \uc608\uce21 \uae30\uac04(2026-2031) \ub3d9\uc548 \uc5f0\ud3c9\uade0 6.98%\uc758 \uacac\uace0\ud55c \uc131\uc7a5\ub960\uc744 \ubcf4\uc77c \uc804\ub9dd\uc785\ub2c8\ub2e4. \uc774 \uc2dc\uc7a5\uc740 \uae30\uc874\uc758 \ub2e8\uc77c \ud6a8\uc18c \ub300\uccb4 \uc694\ubc95(ERT)\uc5d0\uc11c \ucc28\uc138\ub300 ERT, \ubcd1\uc6a9 \uc694\ubc95, \uadf8\ub9ac\uace0 \uc720\uc804\uc790 \ubc0f mRNA \uae30\ubc18 \ud50c\ub7ab\ud3fc\uc73c\ub85c \uce58\ub8cc \ud328\ub7ec\ub2e4\uc784\uc774 \uc804\ud658\ub418\uace0 \uc788\uc73c\uba70, \uc774\ub294 \uc2e0\uaddc \ucd9c\uc2dc \uc81c\ud488\uc758 \ub450 \uc790\ub9bf\uc218 \ub9e4\ucd9c \uc131\uc7a5\uc744 \uacac\uc778\ud558\uace0 \uc788\uc2b5\ub2c8\ub2e4. \ud2b9\ud788 \uc544\uc2dc\uc544-\ud0dc\ud3c9\uc591 \uc9c0\uc5ed\uc5d0\uc11c \uc2e0\uc0dd\uc544 \uc120\ubcc4 \uac80\uc0ac \uc758\ubb34\ud654\uac00 \uac00\uc18d\ud654\ub418\uba74\uc11c \uc9c8\ubcd1 \uc720\ubcd1\ub960\uc774 \uc774\uc804 \ucd94\uc815\uce58\ubcf4\ub2e4 \ud6e8\uc52c \ub192\ub2e4\ub294 \uc0ac\uc2e4\uc774 \ubc1d\ud600\uc838, \uc870\uae30 \uac1c\uc785 \ubc0f \ud3c9\uc0dd \uce58\ub8cc\uc758 \uc0c1\uc5c5\uc801 \ud0c0\ub2f9\uc131\uc774 \uac15\ud654\ub418\uace0 \uc788\uc2b5\ub2c8\ub2e4.<\/p>\n<p>\uac00\uce58 \uc0ac\uc2ac \uc804\ubc18\uc5d0 \uac78\uccd0 \ud22c\uc790\uac00 \uc2ec\ud654\ub418\uace0 \uc788\uc73c\uba70, \uc2a4\ud3f0\uc11c\ub4e4\uc740 \ubc14\uc774\ub7ec\uc2a4 \ubca1\ud130 \ubc0f 2\uc138\ub300 \uc7ac\uc870\ud569 \ud6a8\uc18c\uc758 \uc81c\uc870 \uc5ed\ub7c9\uc744 \ud655\ubcf4\ud558\uae30 \uc704\ud574 \uc218\uc5b5 \ub2ec\ub7ec\ub97c \ud22c\uc790\ud558\uc5ec \uacf5\uae09 \ud0c4\ub825\uc131\uc744 \ubcf4\uc7a5\ud558\uace0 \ucd9c\uc2dc \uc77c\uc815\uc744 \uc704\ud5d8\uc5d0\uc11c \ubc97\uc5b4\ub098\uac8c \ud558\uace0 \uc788\uc2b5\ub2c8\ub2e4. \ub3d9\uc2dc\uc5d0 \ubbf8\uad6d\uacfc \uc720\ub7fd\uc758 \uc9c0\ubd88\uc790\ub4e4\uc740 \uac00\uce58 \uae30\ubc18 \uc0c1\ud658 \uae30\uc900\uc744 \uac15\ud654\ud558\uace0 \uc788\uc5b4, \uac1c\ubc1c\uc790\ub4e4\uc740 \ub192\uc740 \ucd08\uae30 \uac00\uaca9\uc774 \uc785\uc99d \uac00\ub2a5\ud55c \uae30\ub2a5\uc801 \uac1c\uc120 \ubc0f \uc7a5\uae30\uc801\uc778 \ube44\uc6a9 \uc0c1\uc1c4\ub85c \uc774\uc5b4\uc9c4\ub2e4\ub294 \uc2e4\uc81c \uc99d\uac70\ub97c \uc81c\uc2dc\ud558\ub3c4\ub85d \uc694\uad6c\ubc1b\uace0 \uc788\uc2b5\ub2c8\ub2e4. Sanofi\uac00 \uae30\uc874 \uc2dc\uc7a5 \uc9c0\uc704\ub97c \ubc29\uc5b4\ud558\ub294 \uac00\uc6b4\ub370, Amicus Therapeutics, Shionogi \ubc0f \uc5ec\ub7ec \uc720\uc804\uc790 \uce58\ub8cc \uc804\ubb38 \uae30\uc5c5\ub4e4\uc774 \uc601\uc544 \ubc1c\ubcd1 \ubc0f \ud6c4\uae30 \ubc1c\ubcd1 \uc9c8\ud658 \ubd80\ubb38 \uc804\ubc18\uc5d0 \uac78\uccd0 \uc0c8\ub85c\uc6b4 \uae30\ud68c\ub97c \ud3ec\ucc29\ud558\uae30 \uc704\ud574 \uc790\ubcf8\uc744 \ud22c\uc785\ud558\uba74\uc11c \uacbd\uc7c1 \uac15\ub3c4\uac00 \ub192\uc544\uc9c0\uace0 \uc788\uc2b5\ub2c8\ub2e4.<\/p>\n<p>\uc8fc\uc694 \uc2dc\uc7a5 \ud1b5\uacc4 (2025\ub144 \uae30\uc900):<br \/>\n*   \ud22c\uc5ec \uacbd\ub85c\ubcc4: \uc8fc\uc0ac\uc81c(Parenteral)\uac00 90.88%\uc758 \uc2dc\uc7a5 \uc810\uc720\uc728\uc744 \ucc28\uc9c0\ud588\uc73c\uba70, \uacbd\uad6c\uc81c(Oral)\ub294 2031\ub144\uae4c\uc9c0 \uc5f0\ud3c9\uade0 8.25%\uc758 \uc131\uc7a5\ub960\uc744 \uae30\ub85d\ud560 \uac83\uc73c\ub85c \uc608\uc0c1\ub429\ub2c8\ub2e4.<br \/>\n*   \uce58\ub8cc \uc720\ud615\ubcc4: \ud6a8\uc18c \ub300\uccb4 \uc694\ubc95(ERT)\uc774 89.75%\uc758 \uc2dc\uc7a5 \uc810\uc720\uc728\uc744 \uc720\uc9c0\ud588\uc73c\uba70, \ubcd1\uc6a9 \uc694\ubc95(Combination Therapy)\uc740 2031\ub144\uae4c\uc9c0 \uc5f0\ud3c9\uade0 8.55%\uc758 \uc131\uc7a5\ub960\uc744 \ubcf4\uc77c \uac83\uc73c\ub85c \uc804\ub9dd\ub429\ub2c8\ub2e4.<br \/>\n*   \uc9c8\ubcd1 \ubc1c\ubcd1 \uc720\ud615\ubcc4: \ud6c4\uae30 \ubc1c\ubcd1 \ud3fc\ud398\ubcd1(LOPD)\uc774 68.22%\uc758 \uc2dc\uc7a5 \uc810\uc720\uc728\uc744 \ucc28\uc9c0\ud588\uc73c\uba70, \uc601\uc544 \ubc1c\ubcd1 \ud3fc\ud398\ubcd1(IOPD) \uce58\ub8cc\uc81c\ub294 \uc5f0\ud3c9\uade0 9.12%\uc758 \uc131\uc7a5\ub960\ub85c \ubc1c\uc804\ud558\uace0 \uc788\uc2b5\ub2c8\ub2e4.<br \/>\n*   \uc720\ud1b5 \ucc44\ub110\ubcc4: \ubcd1\uc6d0 \uc57d\uad6d(Hospital Pharmacies)\uc774 56.10%\uc758 \ub9e4\ucd9c \uc810\uc720\uc728\uc744 \uae30\ub85d\ud588\uc73c\ub098, \uc628\ub77c\uc778 \uc57d\uad6d(Online Pharmacies)\uc774 2031\ub144\uae4c\uc9c0 \uc5f0\ud3c9\uade0 8.78%\ub85c \uac00\uc7a5 \ube60\ub978 \uc131\uc7a5\uc744 \ubcf4\uc77c \uac83\uc73c\ub85c \uc608\uc0c1\ub429\ub2c8\ub2e4.<br \/>\n*   \uc9c0\uc5ed\ubcc4: \ubd81\ubbf8\uac00 \ud3fc\ud398\ubcd1 \uc2dc\uc7a5 \uc810\uc720\uc728\uc758 46.20%\ub97c \ucc28\uc9c0\ud588\uc73c\uba70, \uc544\uc2dc\uc544-\ud0dc\ud3c9\uc591 \uc9c0\uc5ed\uc740 2031\ub144\uae4c\uc9c0 \uc5f0\ud3c9\uade0 9.02%\ub85c \uac00\uc7a5 \ube60\ub974\uac8c \uc131\uc7a5\ud558\uace0 \uc788\uc2b5\ub2c8\ub2e4.<\/p>\n<p>\uae00\ub85c\ubc8c \ud3fc\ud398\ubcd1 \uc2dc\uc7a5 \ub3d9\ud5a5 \ubc0f \ud1b5\ucc30\ub825<\/p>\n<p>\uc2dc\uc7a5 \ub3d9\uc778:<br \/>\n*   \uc0c8\ub85c\uc6b4 2\uc138\ub300 ERT\uc758 \ucd9c\uc2dc: \ubbf8\uae00\ub8e8\uc2a4\ud0c0\ud2b8(miglustat)\uc640 \ubcd1\uc6a9\ub418\ub294 \uc2dc\ud30c\uae00\ub8e8\ucf54\uc2dc\ub2e4\uc81c \uc54c\ud30c(cipaglucosidase alfa)\uc640 \uac19\uc740 2\uc138\ub300 \ud6a8\uc18c\ub294 \ub9cc\ub178\uc2a4-6-\uc778\uc0b0(mannose-6-phosphate) \ud0dc\uae45 \ubc0f \uc138\ud3ec \ud761\uc218\ub97c \uac1c\uc120\ud558\uc5ec \uace8\uaca9\uadfc\uc758 \uae00\ub9ac\ucf54\uac90 \uc81c\uac70\ub97c \ud5a5\uc0c1\uc2dc\ud0a4\uace0, \uae30\uc874 \uc54c\uae00\ub8e8\ucf54\uc2dc\ub2e4\uc81c \uc54c\ud30c(alglucosidase alfa) \ub300\ube44 \uc6b0\uc218\ud55c \ud658\uc790 \ubcf4\uace0 \uacb0\uacfc\ub97c \ubcf4\uc5ec\uc90d\ub2c8\ub2e4. \uc720\ub7fd\uc5d0\uc11c \ud6c4\uae30 \ubc1c\ubcd1 \ud3fc\ud398\ubcd1(LOPD) \uc131\uc778 \ud658\uc790\ub97c \uc704\ud55c \uc120\ud638 \uc694\ubc95\uc73c\ub85c \uc9c0\uc815\ub41c \uac83\uc740 \ubbf8\uad6d \uc678 \uc9c0\uc5ed\uc5d0\uc11c\uc758 \ud655\uc0b0 \uac00\ub2a5\uc131\uc744 \uc2dc\uc0ac\ud558\uba70, Amicus Therapeutics\ub294 2028\ub144\uae4c\uc9c0 \ub204\uc801 \ub9e4\ucd9c 10\uc5b5 \ub2ec\ub7ec \uc774\uc0c1\uc744 \uc608\uc0c1\ud558\uace0 \uc788\uc2b5\ub2c8\ub2e4. \ud76c\uadc0\uc758\uc57d\ud488(orphan-drug) \uaddc\uc81c \ud504\ub808\uc784\uc6cc\ud06c\uc758 \uc720\uc5f0\uc131\uc740 \uac80\ud1a0 \uae30\uac04\uc744 \ub2e8\ucd95\ud558\uace0 \ub3c5\uc810\uad8c\uc744 \uc5f0\uc7a5\ud558\uc5ec, \ub2e4\ub978 \uae30\uc5c5\ub4e4\uc774 \ud5a5\uc0c1\ub41c ERT \uac1c\ubc1c\uc5d0 \uc6b0\uc120\uc21c\uc704\ub97c \ub450\ub3c4\ub85d \uc7a5\ub824\ud569\ub2c8\ub2e4.<br \/>\n*   \uc2e0\uc0dd\uc544 \uc120\ubcc4 \uac80\uc0ac \uc758\ubb34\ud654 \ud655\ub300: \uc911\uad6d, \uc77c\ubcf8 \ubc0f \uc5ec\ub7ec \ubbf8\uad6d \uc8fc\uc5d0\uc11c \uc2dc\ud589\ub418\ub294 \uc720\uc804\uccb4 \uae30\ubc18 \uc120\ubcc4 \uac80\uc0ac \ud504\ub85c\uadf8\ub7a8\uc740 \ud6a8\uc18c \ud65c\uc131 \uac80\uc0ac\ubcf4\ub2e4 \ud6e8\uc52c \ub192\uc740 \ube44\uc728\ub85c \ud3fc\ud398\ubcd1\uc744 \uac10\uc9c0\ud558\uba70, \uc911\uad6d\uc758 \uc2dc\ubc94 \ud504\ub85c\uadf8\ub7a8\uc5d0\uc11c\ub294 \uc804\uccb4 \ub9ac\uc18c\uc880 \uc9c8\ud658 \uc720\ubcd1\ub960\uc774 \uc2e0\uc0dd\uc544 1,512\uba85\ub2f9 1\uba85\uc73c\ub85c \ubcf4\uace0\ub418\uc5c8\uc2b5\ub2c8\ub2e4. \uc870\uae30 \uc9c4\ub2e8\uc740 \ud2b9\ud788 \uae09\uaca9\ud55c \uc2ec\uc7a5 \ubc0f \ud638\ud761\uae30 \uae30\ub2a5 \uc800\ud558\ub97c \uc608\ubc29\ud560 \uc218 \uc788\ub294 \uc601\uc544 \ubc1c\ubcd1 \uc0ac\ub840\uc5d0\uc11c \uc99d\uc0c1 \uc804 \uce58\ub8cc\ub97c \ucd09\uc9c4\ud569\ub2c8\ub2e4. \uc815\ubd80\ub294 \ud3fc\ud398\ubcd1 \ud328\ub110\uc744 \uad6d\uac00 \uc120\ubcc4 \uac80\uc0ac \ubaa9\ub85d\uc5d0 \ud3ec\ud568\uc2dc\ucf1c \ubcf4\ud5d8 \uc801\uc6a9\uc744 \ud655\ub300\ud558\uace0 \uc9c0\uc18d\uc801\uc778 \ucc44\ud0dd\uc744 \uc720\ub3c4\ud558\uc5ec \uc7a5\uae30\uc801\uc778 \ucc98\ubc29\ub7c9 \uc99d\uac00\ub97c \ub4b7\ubc1b\uce68\ud558\uace0 \uc788\uc2b5\ub2c8\ub2e4.<br \/>\n*   \uc784\uc0c1 \uc2dc\ud5d8 \uc911\uc778 mRNA \uae30\ubc18 ERT \ud50c\ub7ab\ud3fc: \ud504\ub85c\ud53c\uc628\uc0b0\ud608\uc99d(propionic acidemia)\uc5d0\uc11c mRNA-3927\uc758 \uac1c\ub150 \uc99d\uba85 \ub370\uc774\ud130\ub294 \ub300\uc0ac \uc704\uae30\ub97c 70% \uac10\uc18c\uc2dc\ud0a4\ub294 \uac83\uc744 \ubcf4\uc5ec\uc8fc\uba70, \ub0b4\uc778\uc131 \ud6a8\uc18c \uc0dd\uc0b0\uc744 \uc704\ud55c \uba54\uc2e0\uc800 RNA\uc758 \uc9c0\uc9c8 \ub098\ub178\uc785\uc790 \uc804\ub2ec \ubc29\uc2dd\uc758 \uc720\ud6a8\uc131\uc744 \uc785\uc99d\ud588\uc2b5\ub2c8\ub2e4. \ud3fc\ud398\ubcd1\uc5d0 \ub300\ud55c \ubc88\uc5ed \ud504\ub85c\uadf8\ub7a8\uc740 \uae30\ub2a5\uc131 \uc0b0\uc131 \uc54c\ud30c-\uae00\ub8e8\ucf54\uc2dc\ub2e4\uc81c(acid alpha-glucosidase)\ub97c \ubd84\ube44\ud558\ub3c4\ub85d \uac04\uc138\ud3ec\ub97c \ud45c\uc801\uc73c\ub85c \ud558\uba70, \uc7a0\uc7ac\uc801\uc73c\ub85c \uc6d4 1\ud68c \ud22c\uc5ec \ub610\ub294 \uc790\uac00 \ud22c\uc5ec\ub97c \uac00\ub2a5\ud558\uac8c \ud569\ub2c8\ub2e4. \uc81c\uc870 \ud655\uc7a5\uc131\uc740 COVID-19 \ubc31\uc2e0 \ud50c\ub7ab\ud3fc\uacfc \uc720\uc0ac\ud558\uc5ec, \ubcf5\uc7a1\ud55c \uc7ac\uc870\ud569 \ud6a8\uc18c\uc5d0 \ube44\ud574 \uc81c\uc870 \uc6d0\uac00 \uc808\uac10 \uac00\ub2a5\uc131\uc744 \uc2dc\uc0ac\ud569\ub2c8\ub2e4.<br \/>\n*   \uc720\uc804\uc790 \uce58\ub8cc\uc81c \uc790\uae08 \uc870\ub2ec \uae09\uc99d: 2024-2025\ub144 \ub3d9\uc548 \ud3fc\ud398\ubcd1 AAV \ud504\ub85c\uc81d\ud2b8\uc5d0 15\uc5b5 \ub2ec\ub7ec \uc774\uc0c1\uc758 \ubca4\ucc98 \ubc0f \uc804\ub7b5\uc801 \uc790\ubcf8\uc774 \uc720\uc785\ub418\uc5c8\uc73c\uba70, \uac1c\ubc1c\uc790\ub4e4\uc740 SPK-3006, AT845 \ubc0f \uae30\ud0c0 \ud6c4\ubcf4 \ubb3c\uc9c8\uc744 \ub4f1\ub85d \uc5f0\uad6c \ub2e8\uacc4\ub85c \uc9c4\uc785\uc2dc\ud0a4\uace0 \uc788\uc2b5\ub2c8\ub2e4. Astellas\uc758 \ub178\uc2a4\uce90\ub864\ub77c\uc774\ub098\uc5d0 \uc704\uce58\ud55c 1\uc5b5 \ub2ec\ub7ec \uaddc\ubaa8\uc758 \ubca1\ud130 \uc2dc\uc124 \ud22c\uc790\ub294 \uc784\uc0c1 \ubc0f \ucd08\uae30 \uc0c1\uc5c5\uc801 \uacf5\uae09\uc744 \ud655\ubcf4\ud558\uae30 \uc704\ud55c \uac83\uc785\ub2c8\ub2e4.<\/p>\n<p>\uc2dc\uc7a5 \uc81c\uc57d:<br \/>\n*   \ub192\uc740 \uce58\ub8cc \ube44\uc6a9: \ub3c5\uc77c\uc5d0\uc11c \ud3fc\ud398\ubcd1 \ud658\uc790 1\uc778\ub2f9 \uc5f0\uac04 \uc9c0\ucd9c\uc561\uc740 483,907\uc720\ub85c\ub85c, \uc774\ub294 \uac00\uc7a5 \ube44\uc6a9\uc774 \ub9ce\uc774 \ub4dc\ub294 \ub9ac\uc18c\uc880 \ucd95\uc801 \uc9c8\ud658 \uc911 \ud558\ub098\uc774\uba70, \uc774 \uc911 98.5%\uac00 \uc57d\ubb3c \uad6c\ub9e4 \ube44\uc6a9\uc785\ub2c8\ub2e4. \ud76c\uadc0\uc758\uc57d\ud488 \uc815\ucc45\uc774 \uace0\uac00 \uc815\ucc45\uc744 \uc9c0\uc9c0\ud558\uc9c0\ub9cc, \ud604\uc7ac \uc9c0\ubd88\uc790\ub4e4\uc740 \uc644\uc804\ud55c \ubcf4\ud5d8 \uc801\uc6a9\uc744 \uc2b9\uc778\ud558\uae30 \uc804\uc5d0 \ud655\uc2e4\ud55c \uce58\ub8cc \uacb0\uacfc \ub370\uc774\ud130\ub97c \uc694\uad6c\ud558\uace0 \uc788\uc5b4, \ud2b9\ud788 \ubcd1\uc6a9 \uc694\ubc95 \ubc0f \uc720\uc804\uc790 \uce58\ub8cc\uc81c \uc2e0\uaddc \uc9c4\uc785\uc790\ub4e4\uc758 \ud611\uc0c1 \uae30\uac04\uc744 \uc5f0\uc7a5\ud558\uace0 \uad11\ubc94\uc704\ud55c \ucd9c\uc2dc\ub97c \uc9c0\uc5f0\uc2dc\ud0a4\uace0 \uc788\uc2b5\ub2c8\ub2e4.<br \/>\n*   \uc9c0\uc5f0\ub41c \uc9c4\ub2e8 \ubc0f \ub0ae\uc740 \uc778\uc2dd: \ud6c4\uae30 \ubc1c\ubcd1 \uc0ac\ub840\ub294 \uc885\uc885 \uadfc\uc774\uc601\uc591\uc99d(muscular dystrophy)\uacfc \uc720\uc0ac\ud558\uc5ec \uc9c4\ub2e8\uae4c\uc9c0 \uc218\ub144\uc774 \uac78\ub9ac\uace0 \uc7a0\uc7ac\uc801\uc778 \uce58\ub8cc \uae30\ud68c\ub97c \ub193\uce58\uac8c \ub429\ub2c8\ub2e4. \uc804\uc790 \uac74\uac15 \uae30\ub85d \ub370\uc774\ud130 \uc138\ud2b8\uc5d0 \uc801\uc6a9\ub41c \uc778\uacf5\uc9c0\ub2a5 \uc54c\uace0\ub9ac\uc998\uc740 \uc758\uc2ec\uc2a4\ub7ec\uc6b4 \ud45c\ud604\ud615\uc744 \uc2dd\ubcc4\ud558\ub294 \ub370 \uc720\ub9dd\ud558\uc9c0\ub9cc, \ud559\uc220 \uc13c\ud130 \uc678\ubd80\uc5d0\uc11c\uc758 \ucc44\ud0dd\uc740 \uc81c\ud55c\uc801\uc774\uc5b4\uc11c \ub18d\ucd0c \ubc0f \uc800\uc18c\ub4dd \uc9c0\uc5ed\uc5d0\uc11c\uc758 \uc9c4\ub2e8 \ubd80\uc871\uc774 \uc9c0\uc18d\ub418\uace0 \uc788\uc2b5\ub2c8\ub2e4.<br \/>\n*   \ubc14\uc774\ub7ec\uc2a4 \ubca1\ud130 \uc81c\uc870 \ubcd1\ubaa9 \ud604\uc0c1: \uc720\uc804\uc790 \uce58\ub8cc\uc81c\uc758 \ud575\uc2ec \uad6c\uc131 \uc694\uc18c\uc778 \ubc14\uc774\ub7ec\uc2a4 \ubca1\ud130\uc758 \uc81c\uc870\ub294 \ubcf5\uc7a1\ud558\uace0 \uc2dc\uac04\uc774 \ub9ce\uc774 \uc18c\uc694\ub418\uba70, \uc774\ub294 \uac1c\ubc1c\ub41c \uc2dc\uc7a5\uc5d0\uc11c \uacf5\uae09 \ubcd1\ubaa9 \ud604\uc0c1\uc744 \ucd08\ub798\ud558\uc5ec \uc2e0\uaddc \uce58\ub8cc\uc81c\uc758 \ucd9c\uc2dc \ubc0f \ud655\uc0b0\uc744 \uc9c0\uc5f0\uc2dc\ud0ac \uc218 \uc788\uc2b5\ub2c8\ub2e4.<br \/>\n*   \uac00\uce58 \uae30\ubc18 \uac00\uaca9 \ucc45\uc815 \uc555\ub825: \ubd81\ubbf8\uc640 \uc720\ub7fd\uc758 \uc9c0\ubd88\uc790\ub4e4\uc740 \uce58\ub8cc\uc81c\uc758 \uac00\uce58\uc5d0 \ub530\ub77c \uac00\uaca9\uc744 \ucc45\uc815\ud558\ub824\ub294 \uc555\ub825\uc744 \uac00\ud558\uace0 \uc788\uc73c\uba70, \uc774\ub294 \uac1c\ubc1c\uc790\ub4e4\uc774 \uce58\ub8cc \ud6a8\uacfc\uc5d0 \ub300\ud55c \uac15\ub825\ud55c \uc99d\uac70\ub97c \uc81c\uc2dc\ud558\ub3c4\ub85d \uc694\uad6c\ud558\uc5ec \uc2e0\uaddc \uc81c\ud488\uc758 \uc2dc\uc7a5 \uc9c4\uc785\uc744 \uc5b4\ub835\uac8c \ub9cc\ub4e4 \uc218 \uc788\uc2b5\ub2c8\ub2e4.<\/p>\n<p>\uc138\uadf8\uba3c\ud2b8 \ubd84\uc11d<\/p>\n<p>*   \ud22c\uc5ec \uacbd\ub85c\ubcc4: \uc8fc\uc0ac\uc81c\ub294 2025\ub144 \ud3fc\ud398\ubcd1 \uc2dc\uc7a5\uc758 90.88%\ub97c \ucc28\uc9c0\ud558\uba70 17.4\uc5b5 \ub2ec\ub7ec \uaddc\ubaa8\ub97c \ud615\uc131\ud588\uace0, \uc5f0\ud3c9\uade0 6.25%\uc758 \uc131\uc7a5\ub960\uc744 \ubcf4\uc600\uc2b5\ub2c8\ub2e4. \uc774\ub294 \uac1c\uc120\ub41c \ud6a8\uc18c \uc81c\uc81c\uc758 \uc784\uc0c1 \ubc1c\uc804, \uc218\uc2ed \ub144\uac04\uc758 \uc784\uc0c1\uc758 \uc775\uc219\ud568, \uc131\uc219\ud55c \uc804\ubb38 \uc57d\uad6d \ubb3c\ub958, \uadf8\ub9ac\uace0 \ud638\ud761\uae30 \ubc0f \ubcf4\ud589 \uae30\ub2a5 \uc548\uc815\ud654\uc5d0 \ub300\ud55c \uc785\uc99d\ub41c \uc784\uc0c1\uc801 \uc774\uc810\uc5d0 \uae30\uc778\ud569\ub2c8\ub2e4. \uc544\ubc1c\uae00\ub8e8\ucf54\uc2dc\ub2e4\uc81c \uc54c\ud30c(avalglucosidase alfa)\ub294 \uadfc\uc721 \ud761\uc218\ub97c \ud5a5\uc0c1\uc2dc\ud0a4\uace0 \uc8fc\uc785 \uac04\uaca9\uc744 \ub298\ub9ac\ub294 \ube44\uc2a4-\uc778\uc0b0\ud654 \ub9cc\ub178\uc2a4-6-\uc778\uc0b0 \ud0dc\uadf8\ub97c \ud3ec\ud568\ud558\ub294 \uc81c\ud615 \ud601\uc2e0\uc744 \ubcf4\uc5ec\uc90d\ub2c8\ub2e4. \ubc18\uba74, \uacbd\uad6c \ud22c\uc5ec\ub294 \ucd08\uae30 \ub2e8\uacc4\uc774\uc9c0\ub9cc \uc804\ub7b5\uc801\uc73c\ub85c \uc911\uc694\ud55c \uc811\uadfc \ubc29\uc2dd\uc785\ub2c8\ub2e4. Maze Therapeutics\uc758 \uacbd\uad6c\uc6a9 \uae00\ub9ac\ucf54\uac90 \ud569\uc131\ud6a8\uc18c \uc5b5\uc81c\uc81c\uc778 MZE-001\uc740 Sanofi\uc758 7.5\uc5b5 \ub2ec\ub7ec \uc778\uc218 \ubc0f Shionogi\uc758 1.5\uc5b5 \ub2ec\ub7ec \ub77c\uc774\uc120\uc2a4 \uacc4\uc57d\uc758 \uae30\ubc18\uc774 \ub418\uc5c8\uc73c\uba70, \uc774\ub294 \ud3b8\ub9ac\ud55c \uacbd\uad6c \uc57d\ubb3c\uc774 \uaca9\uc8fc \uc8fc\uc785\uc744 \ubcf4\uc644\ud558\uac70\ub098 \ub300\uccb4\ud560 \uc218 \uc788\ub2e4\ub294 \ud655\uc2e0\uc744 \ubc18\uc601\ud569\ub2c8\ub2e4.<br \/>\n*   \uce58\ub8cc \uc720\ud615\ubcc4: \ud6a8\uc18c \ub300\uccb4 \uc694\ubc95(ERT)\uc740 2025\ub144 \ub9e4\ucd9c\uc758 89.75%\ub97c \ucc3d\ucd9c\ud558\uba70 17.1\uc5b5 \ub2ec\ub7ec \uaddc\ubaa8\ub97c \uae30\ub85d\ud588\uc9c0\ub9cc, \ubcd1\uc6a9 \uc694\ubc95\uc774 \ud655\ub300\ub428\uc5d0 \ub530\ub77c \uadf8 \uc810\uc720\uc728\uc740 \uc18c\ud3ed \uac10\uc18c\ud560 \uac83\uc73c\ub85c \uc608\uc0c1\ub429\ub2c8\ub2e4. \uc54c\uae00\ub8e8\ucf54\uc2dc\ub2e4\uc81c \uc54c\ud30c(alglucosidase alfa)\uc640 \uc544\ubc1c\uae00\ub8e8\ucf54\uc2dc\ub2e4\uc81c \uc54c\ud30c(avalglucosidase alfa)\ub294 \uad11\ubc94\uc704\ud55c \uc7a5\uae30 \uc548\uc804\uc131 \ub370\uc774\ud130\uc640 \ubcf4\ud5d8 \uc801\uc6a9\uc758 \uc775\uc219\ud568\uc73c\ub85c \uc778\ud574 \uc784\uc0c1\uc758\ub4e4\uc774 \uacc4\uc18d \uc758\uc874\ud558\uace0 \uc788\uc2b5\ub2c8\ub2e4. \uadf8\ub7ec\ub098 \uc2dc\ud30c\uae00\ub8e8\ucf54\uc2dc\ub2e4\uc81c \uc54c\ud30c\uc640 \ubbf8\uae00\ub8e8\uc2a4\ud0c0\ud2b8 \ubcd1\uc6a9 \uc694\ubc95\uc758 FDA \uc2b9\uc778\uc740 \ud328\ub7ec\ub2e4\uc784 \uc804\ud658\uc744 \uc608\uace0\ud569\ub2c8\ub2e4. Amicus\ub294 2025\ub144 1\ubd84\uae30\uc5d0 \uc804\ub144 \ub300\ube44 90%\uc758 \ub9e4\ucd9c \uc131\uc7a5\uc744 \ubcf4\uace0\ud588\uc73c\uba70, \uc774\ub294 6\ubd84 \ubcf4\ud589 \uac80\uc0ac \uc810\uc218 \uac1c\uc120 \ud6c4 \uce58\ub8cc \uacbd\ud5d8\uc774 \uc788\ub294 \ud658\uc790\ub4e4\uc774 \ubcd1\uc6a9 \uc694\ubc95\uc73c\ub85c \uc804\ud658\ud588\uae30 \ub54c\ubb38\uc785\ub2c8\ub2e4. \uc720\uc804\uc790 \ubc0f mRNA \uce58\ub8cc\uc81c\ub294 &#8220;\uae30\ud0c0&#8221; \ubc94\uc8fc\uc5d0 \uc18d\ud558\uba70 ERT\uc758 \uc9c0\ubc30 \uae30\uac04\uc744 \ub2e8\ucd95\uc2dc\ud0ac \uc218 \uc788\uc2b5\ub2c8\ub2e4. \ub2e8\uc77c \ud22c\uc5ec AAV \ubca1\ud130 \ub610\ub294 \uc6d4 1\ud68c mRNA \uc8fc\uc0ac\ub294 \ud2b9\ud788 \uc80a\uc740 \ucf54\ud638\ud2b8\uc758 \uce58\ub8cc \uc21c\uc751\ub3c4\ub97c \uc7ac\ud3b8\ud560 \uc218 \uc788\uc9c0\ub9cc, \ub300\uaddc\ubaa8 \uc0c1\uc5c5\ud654 \uc804\uc5d0 \uc81c\uc870 \ubc0f \uba74\uc5ed\uc6d0\uc131 \ubb38\uc81c\ub97c \ud574\uacb0\ud574\uc57c \ud569\ub2c8\ub2e4.<br \/>\n*   \uc9c8\ubcd1 \ubc1c\ubcd1 \uc720\ud615\ubcc4: \ud6c4\uae30 \ubc1c\ubcd1 \ud3fc\ud398\ubcd1(LOPD)\uc740 \ub354 \ub192\uc740 \uc720\ubcd1\ub960\uacfc \ub354 \uae34 \uce58\ub8cc \uae30\uac04\uc744 \ubc18\uc601\ud558\uc5ec 2025\ub144 \ub9e4\ucd9c\uc758 68.22%\ub97c \ucc28\uc9c0\ud588\uc2b5\ub2c8\ub2e4. \uc131\uc7a5\uc740 \uc9c0\uc18d\ub418\uaca0\uc9c0\ub9cc, \uc608\uce21 \ubaa8\ub378\uc740 \uc601\uc544 \ubc1c\ubcd1 \ud3fc\ud398\ubcd1(IOPD)\uc5d0 2031\ub144\uae4c\uc9c0 \uc5f0\ud3c9\uade0 9.12%\uc758 \uc131\uc7a5\ub960\uc744 \ud560\ub2f9\ud558\uc5ec, \uc0b0\uc804 \ubc0f \uc2e0\uc0dd\uc544 \uac1c\uc785\uc774 \ud655\ub300\ub428\uc5d0 \ub530\ub77c \ub9e4\ucd9c \uc810\uc720\uc728\uc774 \uc99d\uac00\ud560 \uac83\uc73c\ub85c \uc608\uc0c1\ud569\ub2c8\ub2e4. PEARL \uc784\uc0c1 \uc2dc\ud5d8\uc758 \uc790\uad81 \ub0b4 ERT\ub294 \uba74\uc5ed \uad00\uc6a9\uc744 \uc720\ub3c4\ud558\uc5ec \ud56d\uccb4 \ub9e4\uac1c \uc57d\ubb3c \ud6a8\ub2a5 \uac10\uc18c\ub97c \uc7a0\uc7ac\uc801\uc73c\ub85c \uc81c\uac70\ud558\ub294 \uac83\uc744 \ubaa9\ud45c\ub85c \ud569\ub2c8\ub2e4. \uc131\uacf5\ud560 \uacbd\uc6b0, \uc601\uc544 \uc0dd\uc874\uc728\uacfc \uc2e0\uacbd \uc6b4\ub3d9 \uacb0\uacfc\uac00 \uadf9\uc801\uc73c\ub85c \uac1c\uc120\ub418\uc5b4 \uac00\uc774\ub4dc\ub77c\uc778 \ubcc0\uacbd \ubc0f \uce58\ub8cc \ub300\uc0c1 \ud658\uc790 \ud480\uc774 \ub354\uc6b1 \ud655\ub300\ub420 \uc218 \uc788\uc2b5\ub2c8\ub2e4. LOPD\uc758 \uacbd\uc6b0, \uccab \uc99d\uc0c1 \ubc1c\ud604 \ud6c4 2\ub144 \uc774\ub0b4\uc5d0 \uce58\ub8cc\ub97c \uc2dc\uc791\ud558\uba74 \ub098\uc911\uc5d0 \uc2dc\uc791\ud558\ub294 \uacbd\uc6b0\ubcf4\ub2e4 \uc778\uacf5\ud638\ud761\uae30 \uc5c6\uc774 \uc0dd\uc874\ud558\ub294 \uae30\uac04\uc774 5\ub144 \uc774\uc0c1 \uc5f0\uc7a5\ub41c\ub2e4\ub294 \ub370\uc774\ud130\uac00 \ud655\uc778\ub418\uc5c8\uc2b5\ub2c8\ub2e4. \uacb0\uacfc\uc801\uc73c\ub85c, \uc9c0\ubd88\uc790 \uc815\ucc45\uc740 \uc124\uba85\ud560 \uc218 \uc5c6\ub294 \uadfc\uc704\ubd80 \uadfc\uc721 \uc57d\ud654\ub97c \uac00\uc9c4 \uc131\uc778\uc5d0 \ub300\ud55c \uc720\uc804\uc790 \uac80\uc0ac\ub97c \uc810\uc810 \ub354 \ub9ce\uc774 \ubcf4\uc0c1\ud558\uace0 \uc788\uc73c\uba70, \uc774\ub294 \ub354 \ub192\uc740 \uc9c4\ub2e8\uc728\uacfc \uc804\ubb38 \uc13c\ud130\ub85c\uc758 \uafb8\uc900\ud55c \ud658\uc790 \uc720\uc785\uc73c\ub85c \uc774\uc5b4\uc9c0\uace0 \uc788\uc2b5\ub2c8\ub2e4.<br \/>\n*   \uc720\ud1b5 \ucc44\ub110\ubcc4: \ubcd1\uc6d0 \uc57d\uad6d\uc740 \uc8fc\uc785 \uc13c\ud130 \uc778\ud504\ub77c, \ub2e4\ud559\uc81c \uc9c4\ub8cc\ud300, \uadf8\ub9ac\uace0 \uc881\uc740 \uc548\uc815\uc131 \ubc94\uc704\uc758 \uc0dd\ubb3c\ud559\uc801 \uc81c\uc81c\uc5d0 \ud544\uc218\uc801\uc778 \uc5c4\uaca9\ud55c \ud488\uc9c8 \uad00\ub9ac \ud504\ub85c\ud1a0\ucf5c\uc5d0 \ud798\uc785\uc5b4 2025\ub144 \ub9e4\ucd9c\uc758 56.10%\ub97c \ucc28\uc9c0\ud588\uc2b5\ub2c8\ub2e4. \uc628\ub77c\uc778 \uc57d\uad6d\uc774 \ub514\uc9c0\ud138\uc5d0 \ub2a5\uc219\ud55c \ud658\uc790 \uc138\uadf8\uba3c\ud2b8\ub97c \ud3ec\ucc29\ud568\uc5d0 \ub530\ub77c \uadf8 \uc810\uc720\uc728\uc740 \uc18c\ud3ed \uac10\uc18c\ud560 \uac83\uc73c\ub85c \uc608\uc0c1\ub418\uc9c0\ub9cc, \ubcd1\uc6d0\uc73c\ub85c\ubd80\ud130\uc758 \uc808\ub300 \ub9e4\ucd9c\uc740 \uc804\uccb4 \ud3fc\ud398\ubcd1 \uc2dc\uc7a5 \ud655\ub300\uc640 \ud568\uaed8 \uc99d\uac00\ud560 \uac83\uc785\ub2c8\ub2e4. Accredo\uc640 \uac19\uc740 \ub300\uaddc\ubaa8 \ub124\ud2b8\uc6cc\ud06c\ub294 \uc608\uce21 \ucf5c\ub4dc \uccb4\uc778 \ubd84\uc11d\uc744 \ud65c\uc6a9\ud558\uc5ec 99%\uc758 \uc870\uc81c \uc815\ud655\ub3c4\ub97c \ub2ec\uc131\ud568\uc73c\ub85c\uc368 \uc784\uc0c1\uc758\uc758 \uc2e0\ub8b0\ub97c \uac15\ud654\ud569\ub2c8\ub2e4. \uc804\uc790 \uc57d\uad6d \ud50c\ub7ab\ud3fc\uc740 \uc6d0\uaca9 \uc758\ub8cc \uc0c1\ub2f4, \uc6d0\uaca9 \ud3d0\ud65c\ub7c9 \uce21\uc815 \ubc0f \uc8fc\uc785 \uc6a9\ud488\uc758 \uac00\uc815 \ubc30\uc1a1\uc744 \ubb36\ub294 \ud558\uc774\ube0c\ub9ac\ub4dc \ucf00\uc5b4 \ubaa8\ub378\uc5d0 \ud798\uc785\uc5b4 \uc5f0\ud3c9\uade0 8.78%\uc758 \uc131\uc7a5\ub960\uc744 \uae30\ub85d\ud560 \uac83\uc73c\ub85c \uc608\uc0c1\ub429\ub2c8\ub2e4. \uc18c\ub9e4 \ub300\uae30\uc5c5\ub4e4\uc740 \uc804\uc6a9 \ud76c\uadc0 \uc9c8\ud658 \ud5c8\ube0c\uc5d0 \ud22c\uc790\ud558\uc5ec \uc811\uadfc \uc9c0\uc810\uc744 \ub2e4\uc591\ud654\ud558\uace0 3\ucc28 \uc9c4\ub8cc \uc13c\ud130\uc5d0\uc11c \uba40\ub9ac \ub5a8\uc5b4\uc9c4 \ud658\uc790\ub4e4\uc758 \uc774\ub3d9 \ubd80\ub2f4\uc744 \uc644\ud654\ud558\uace0 \uc788\uc2b5\ub2c8\ub2e4. \uadf8\ub7fc\uc5d0\ub3c4 \ubd88\uad6c\ud558\uace0 \uc81c\ud55c\ub41c \uc720\ud1b5 \uacc4\uc57d\uc740 \ubb3c\ub7c9\uc744 10\uac1c \ubbf8\ub9cc\uc758 \uacf5\uc778 \uc57d\uad6d\uc73c\ub85c \uc81c\ud55c\ud558\uc5ec \uc5c4\uaca9\ud55c \uac10\ub3c5\uc744 \uc720\uc9c0\ud558\uace0 \uc704\uc870 \uc704\ud5d8\uc744 \ucd5c\uc18c\ud654\ud569\ub2c8\ub2e4.<\/p>\n<p>\uc9c0\uc5ed \ubd84\uc11d<\/p>\n<p>*   \ubd81\ubbf8: 2025\ub144 \ub9e4\ucd9c\uc758 46.20%\ub97c \ucc28\uc9c0\ud558\uba70 8.8\uc5b5 \ub2ec\ub7ec \uaddc\ubaa8\ub97c \uae30\ub85d\ud588\uc2b5\ub2c8\ub2e4. \uc774\ub294 \uac15\ub825\ud55c \ubcf4\ud5d8 \uc801\uc6a9 \ubc94\uc704, \ubbf8\uad6d \uc804\uc5ed\uc758 \ud65c\ubc1c\ud55c \uc2e0\uc0dd\uc544 \uc120\ubcc4 \uac80\uc0ac, \uadf8\ub9ac\uace0 \uc2e0\uacbd\uadfc \uc804\ubb38\uc758\ub4e4 \uc0ac\uc774\uc758 \ucd08\uae30 \ucc44\ud0dd \ud589\ub3d9\uc744 \ubc18\uc601\ud569\ub2c8\ub2e4. \ub610\ud55c, \uc774 \uc9c0\uc5ed\uc740 \uc2ec\uce35\uc801\uc778 \uc790\ubcf8 \uc2dc\uc7a5\uacfc \uc219\ub828\ub41c \uacc4\uc57d \uc81c\uc870 \uc0dd\ud0dc\uacc4\uc758 \uc9c0\uc6d0\uc744 \ubc1b\uc544 \uc9c4\ud589 \uc911\uc778 \ub300\ubd80\ubd84\uc758 3\uc0c1 \uc720\uc804\uc790 \uce58\ub8cc\uc81c \uc784\uc0c1 \uc2dc\ud5d8\uc744 \uc720\uce58\ud558\uace0 \uc788\uc2b5\ub2c8\ub2e4. \uc778\ud50c\ub808\uc774\uc158 \uac10\ucd95\ubc95(Inflation Reduction Act)\uc758 \uc57d\uac00 \ud611\uc0c1\uacfc \uac19\uc740 \uc815\ucc45 \ubcc0\ud654\ub294 \ubd88\ud655\uc2e4\uc131\uc744 \uc57c\uae30\ud558\uc9c0\ub9cc, \uc0dd\uba85\uc744 \uc704\ud611\ud558\ub294 \ud76c\uadc0 \uc9c8\ud658\uc5d0 \ub300\ud55c \ub192\uc740 \uc784\uc0c1\uc801 \uac00\uce58 \ud3c9\uac00\ub97c \ud6fc\uc190\ud560 \uac00\ub2a5\uc131\uc740 \ub0ae\uc2b5\ub2c8\ub2e4.<br \/>\n*   \uc720\ub7fd: \ub450 \ubc88\uc9f8\ub85c \ud070 \uc9c0\uc5ed \ube14\ub85d\uc744 \uad6c\uc131\ud558\uba70, \uc720\ub7fd \uc758\uc57d\ud488\uccad(European Medicines Agency)\uc744 \ud1b5\ud55c \uc870\ud654\ub85c\uc6b4 \uaddc\uc81c \uacbd\ub85c\ub97c \ud2b9\uc9d5\uc73c\ub85c \ud558\uc9c0\ub9cc \ud68c\uc6d0\uad6d \uc218\uc900\uc5d0\uc11c\ub294 \uc0c1\ud658 \uacb0\uc815\uc774 \ubd84\uc5f4\ub418\uc5b4 \uc788\uc2b5\ub2c8\ub2e4. \uc11c\uc720\ub7fd \uad6d\uac00\ub4e4\uc740 \uc77c\ubc18\uc801\uc73c\ub85c \uc2dc\ud310 \ud5c8\uac00 \ud6c4 3\uac1c\uc6d4 \uc774\ub0b4\uc5d0 \ud3fc\ud398\ubcd1 ERT\uc5d0 \uc790\uae08\uc744 \uc9c0\uc6d0\ud558\ub294 \ubc18\uba74, \ub9ce\uc740 \uc911\ubd80 \ubc0f \ub3d9\uc720\ub7fd \uc2dc\uc7a5\uc740 2\ub144 \uc774\uc0c1 \uc9c0\uc5f0\ub418\uc5b4 \uc9c0\uc5ed \ub0b4 \uc811\uadfc\uc131 \uaca9\ucc28\ub97c \ub9cc\ub4ed\ub2c8\ub2e4. Roche\uc758 \ub3c5\uc77c \ub0b4 9\ucc9c\ub9cc \uc720\ub85c \uaddc\ubaa8\uc758 \uc720\uc804\uc790 \uce58\ub8cc\uc81c \uc2dc\uc124 \ubc0f UCB\uc758 \ubca8\uae30\uc5d0 \ub0b4 2\uc5b5 \uc720\ub85c \uaddc\ubaa8\uc758 \ud6a8\uc18c \uc2dc\uc124\uacfc \uac19\uc740 \uc0b0\uc5c5\uc801 \uc57d\uc18d\uc740 \uac74\uac15 \uae30\uc220 \ud3c9\uac00(health-technology-assessment) \uc2ec\uc0ac\uac00 \uac15\ud654\ub428\uc5d0\ub3c4 \ubd88\uad6c\ud558\uace0 \uc720\ub7fd\uc774 \uc5ec\uc804\ud788 \uc218\uc775\uc131 \uc788\ub294 \ucd9c\uc2dc \uc9c0\uc5ed\uc73c\ub85c \ub0a8\uc744 \uac83\uc774\ub77c\ub294 \ud655\uc2e0\uc744 \ubc18\uc601\ud569\ub2c8\ub2e4.<br \/>\n*   \uc544\uc2dc\uc544-\ud0dc\ud3c9\uc591: \uc5f0\ud3c9\uade0 9.02%\ub85c \uac00\uc7a5 \ube60\ub978 \uc131\uc7a5 \uada4\ub3c4\ub97c \uae30\ub85d\ud558\uace0 \uc788\uc2b5\ub2c8\ub2e4. \uc911\uad6d\uc758 \uc758\ubb34\uc801\uc778 \uc720\uc804\uccb4 \uc2e0\uc0dd\uc544 \uc120\ubcc4 \uac80\uc0ac\ub294 \uc9c4\ub2e8\ub41c \uc778\uad6c\ub97c \uc989\uc2dc \ud655\ub300\ud558\uace0 ERT \ubcf4\uc870\uae08 \uc9c0\uae09\uc744 \uc704\ud55c \uc815\ucc45 \uc6c0\uc9c1\uc784\uc744 \ucd09\ubc1c\ud558\ub294 \ubc1c\uc0dd\ub960 \ub370\uc774\ud130\ub97c \uc0b0\ucd9c\ud588\uc2b5\ub2c8\ub2e4. \uc77c\ubcf8\uc758 \uc758\uc57d\ud488\uc758\ub8cc\uae30\uae30\uc885\ud569\uae30\uad6c(PMDA)\ub294 \ud76c\uadc0\uc758\uc57d\ud488\uc5d0 \ub300\ud574 9\uac1c\uc6d4\uc758 \uc2e0\uc18d \uac80\ud1a0 \uc8fc\uae30\uc640 7\ub144\uac04\uc758 \ub3c5\uc810\uad8c\uc744 \uc720\uc9c0\ud558\uc5ec, \uc2a4\ud3f0\uc11c\ub4e4\uc774 \uae00\ub85c\ubc8c \uc11c\ub958\ub97c \uc870\uae30\uc5d0 \uc81c\ucd9c\ud558\ub3c4\ub85d \uc7a5\ub824\ud569\ub2c8\ub2e4. \ud55c\ud3b8, \ud638\uc8fc\uc640 \ud55c\uad6d\uc740 \uc544\ubc1c\uae00\ub8e8\ucf54\uc2dc\ub2e4\uc81c \uc54c\ud30c\ub97c \uad6d\uac00 \ubcf4\ud5d8 \ubaa9\ub85d\uc5d0 \ucd94\uac00\ud558\uc5ec \ucc28\uc138\ub300 \uce58\ub8cc\uc81c\uc758 \uad11\ubc94\uc704\ud55c \uc9c0\uc5ed \ud1b5\ud569\uc744 \uc2dc\uc0ac\ud558\uace0 \uc788\uc2b5\ub2c8\ub2e4. \ud604\uc9c0 \ucda9\uc804-\ub9c8\uac10(fill-finish) \uc5ed\ub7c9\uc744 \ud3ec\ud568\ud55c \uacf5\uae09\ub9dd \ud22c\uc790\ub294 \uc218\uc785 \uad00\ub828 \uc9c0\uc5f0\uc744 \uc644\ud654\ud558\uace0 \ucc29\ub959 \ube44\uc6a9\uc744 \ub0ae\ucdb0 \uc9c0\uc18d \uac00\ub2a5\ud55c \ubb3c\ub7c9 \uc99d\uac00\ub97c \ucd09\uc9c4\ud569\ub2c8\ub2e4.<\/p>\n<p>\uacbd\uc7c1 \ud658\uacbd<\/p>\n<p>\ud3fc\ud398\ubcd1 \uc2dc\uc7a5\uc758 \uacbd\uc7c1 \uc9d1\uc911\ub3c4\ub294 \uc5ec\uc804\ud788 \ub192\uc73c\uba70, Sanofi, Amicus Therapeutics \ubc0f \uc18c\uc218\uc758 \ub2e4\ub978 \uae30\uc5c5\ub4e4\uc774 \uc804 \uc138\uacc4 \uc2dc\uc7a5 \ub9e4\ucd9c\uc758 \ub300\ubd80\ubd84\uc744 \ucc28\uc9c0\ud558\uace0 \uc788\uc2b5\ub2c8\ub2e4. Sanofi\ub294 1\uc138\ub300 \ubc0f 2\uc138\ub300 ERT \ud504\ub79c\ucc28\uc774\uc988\ub97c \ud1b5\ud574 \uc548\uc815\uc801\uc778 \uc5f0\uac04 \ud604\uae08 \ud750\ub984\uc744 \ucc3d\ucd9c\ud558\uba70 \uc5ed\uc0ac\uc801\uc778 \ub9ac\ub354\uc2ed\uc744 \uc720\uc9c0\ud558\uace0 \uc788\uc2b5\ub2c8\ub2e4. Amicus\ub294 \ucd5c\ucd08\ub85c \uc2b9\uc778\ub41c \ubcd1\uc6a9 \uc694\ubc95\uc744 \ucd9c\uc2dc\ud558\uc5ec \uc2dc\uc7a5 \uc810\uc720\uc728\uc744 \ube60\ub974\uac8c \ud655\ub300\ud588\uc73c\uba70, \uac15\ub825\ud55c \uc2dc\ud310 \ud6c4 \ub370\uc774\ud130 \ud328\ud0a4\uc9c0\uc5d0 \ud798\uc785\uc5b4 2025\ub144 \ub9d0\uae4c\uc9c0 \ucd5c\uc18c 20\uac1c\uad6d\uc5d0\uc11c \uae00\ub85c\ubc8c \ucd9c\uc2dc\ub97c \ubaa9\ud45c\ub85c \ud558\uace0 \uc788\uc2b5\ub2c8\ub2e4.<\/p>\n<p>\uc804\ub7b5\uc801 \uac70\ub798\ub294 \ucc28\ubcc4\ud654\ub41c \uba54\ucee4\ub2c8\uc998\uc5d0 \ub300\ud55c \ub192\uc740 \uac00\uce58\ub97c \ubcf4\uc5ec\uc90d\ub2c8\ub2e4. Sanofi\ub294 Maze Therapeutics\uc758 \uacbd\uad6c\uc6a9 \uae00\ub9ac\ucf54\uac90 \ud569\uc131\ud6a8\uc18c \ud504\ub85c\uadf8\ub7a8\uc5d0 7.5\uc5b5 \ub2ec\ub7ec\ub97c \uc9c0\ubd88\ud558\uba70 \uacbd\uc7c1\uc0ac\ub4e4\uc744 \uc81c\uce58\uace0 \uc778\uc218\ud588\uc73c\uba70, Shionogi\ub294 \ub3d9\uc2dc\uc5d0 1.5\uc5b5 \ub2ec\ub7ec\uc758 \uc120\ubd88\uae08\uacfc \ub9c8\uc77c\uc2a4\ud1a4\uc744 \uc9c0\ubd88\ud558\uace0 \uc804 \uc138\uacc4 \ud310\uad8c\uc744 \ud655\ubcf4\ud558\uc5ec, \ub3d9\uc77c\ud55c \uc790\uc0b0 \ud074\ub798\uc2a4\uc5d0 \ub300\ud55c \ubcd1\ub82c \uac1c\ubc1c \uacbd\ub85c\ub97c \ud6a8\uacfc\uc801\uc73c\ub85c \uc120\uc810\ud588\uc2b5\ub2c8\ub2e4. \uc774\ub7ec\ud55c \uac70\ub798\ub294 \uc131\uc219\ud55c ERT \ud3ec\ud2b8\ud3f4\ub9ac\uc624\uc5d0 \ub300\ud55c \uc555\ub825\uc744 \uc0c1\uc1c4\ud560 \uc218 \uc788\ub294 \uc720\ub9dd\ud55c \ud30c\uc774\ud504\ub77c\uc778 \uc790\uc0b0\uc5d0 \ub300\ud55c \uce58\uc5f4\ud55c \uacbd\uc7c1\uc744 \ubcf4\uc5ec\uc90d\ub2c8\ub2e4.<\/p>\n<p>\uc720\ud1b5 \ub610\ud55c \uc9d1\uc911\ub418\uc5b4 \uc788\uc2b5\ub2c8\ub2e4. 10\uac1c \ubbf8\ub9cc\uc758 \uc804\ubb38 \uc57d\uad6d\uc774 \uc8fc\uc785\ud615 \ud3fc\ud398\ubcd1 \uce58\ub8cc\uc81c\uc758 \uc57d 80%\ub97c \uad00\ub9ac\ud558\uba70, \uc774\ub294 \ucc98\ubc29\uc9d1 \ud611\uc0c1 \ubc0f \ud658\uc790 \uc9c0\uc6d0 \uacc4\uc57d\uc5d0\uc11c \uc774\ub4e4\uc5d0\uac8c \uc601\ud5a5\ub825\uc744 \ubd80\uc5ec\ud569\ub2c8\ub2e4. \uadf8\ub7ec\ub098 \uc774\ub7ec\ud55c \uc9d1\uc911\uc740 \ud601\uc2e0\uc758 \ud655\uc0b0\uc744 \uac00\uc18d\ud654\ud569\ub2c8\ub2e4. \uc77c\ub2e8 \uc2e0\uc81c\ud488\uc774 \uc81c\ud55c\ub41c \uc720\ud1b5 \uacc4\uc57d\uc744 \ud655\ubcf4\ud558\uba74, \uc804\uad6d \ubcd1\uc6d0 \ub124\ud2b8\uc6cc\ud06c\ub294 \uba87 \uc8fc \ub0b4\uc5d0 \ud658\uc790\ub97c \uc804\ud658\ud560 \uc218 \uc788\uc5b4 \uc77c\ubc18\uc801\uc778 \ucd9c\uc2dc \ud6c4 \ucc44\ud0dd \uace1\uc120\uc744 \ub2e8\ucd95\uc2dc\ud0b5\ub2c8\ub2e4.<\/p>\n<p>\uc8fc\uc694 \uc2dc\uc7a5 \ucc38\uc5ec\uc790:<br \/>\n*   Amicus Therapeutics<br \/>\n*   Sanofi<br \/>\n*   Exerkine Corporation<br \/>\n*   Genethon<br \/>\n*   Maze Therapeutics, Inc.<\/p>\n<p>\ucd5c\uadfc \uc0b0\uc5c5 \ub3d9\ud5a5:<br \/>\n*   2025\ub144 5\uc6d4: Amicus Therapeutics\ub294 2025\ub144 1\ubd84\uae30 Pombiliti + Opfolda \ub9e4\ucd9c\uc774 1\uc5b5 2,520\ub9cc \ub2ec\ub7ec\ub85c \uc804\ub144 \ub300\ube44 90% \uc131\uc7a5\ud588\ub2e4\uace0 \ubcf4\uace0\ud588\uc73c\uba70, 2025\ub144 \ud558\ubc18\uae30 GAAP \uc218\uc775\uc131\uc744 \ub2ec\uc131\ud558\uace0 2025\ub144 \ub3d9\uc548 \ucd5c\ub300 10\uac1c\uad6d\uc5d0\uc11c \uc2e0\uaddc \ucd9c\uc2dc\ub97c\uacc4\ud68d\ud558\uace0 \uc788\ub2e4\uace0 \ubc1c\ud45c\ud588\uc2b5\ub2c8\ub2e4.<\/p>\n<p>*   2024\ub144 11\uc6d4: \uc720\ub7fd \uc758\uc57d\ud488\uccad(EMA)\uc740 \ud76c\uadc0 \uc9c8\ud658 \uce58\ub8cc\ub97c \uc704\ud55c \uc0c8\ub85c\uc6b4 \uc720\uc804\uc790 \uce58\ub8cc\uc81c\uc5d0 \ub300\ud55c \uc2e0\uc18d \uc2ec\uc0ac \uc808\ucc28\ub97c \uc2b9\uc778\ud558\uc5ec, \ud601\uc2e0\uc801\uc778 \uce58\ub8cc\ubc95\uc758 \uc2dc\uc7a5 \ucd9c\uc2dc\ub97c \uac00\uc18d\ud654\ud560 \uac83\uc73c\ub85c \uc608\uc0c1\ub429\ub2c8\ub2e4.<br \/>\n*   2024\ub144 9\uc6d4: \uc5ec\ub7ec \ubc14\uc774\uc624 \uc81c\uc57d \ud68c\uc0ac\ub4e4\uc774 \ud3fc\ud398\ubcd1\uacfc \uac19\uc740 \ud76c\uadc0 \uc720\uc804 \uc9c8\ud658\uc5d0 \ub300\ud55c \uc720\uc804\uc790 \ud3b8\uc9d1 \uae30\uc220\uc744 \ud65c\uc6a9\ud55c \uc804\uc784\uc0c1 \uc5f0\uad6c\uc5d0\uc11c \uae0d\uc815\uc801\uc778 \uacb0\uacfc\ub97c \ubc1c\ud45c\ud558\uba70, \ucc28\uc138\ub300 \uce58\ub8cc\ubc95 \uac1c\ubc1c\uc5d0 \ub300\ud55c \uae30\ub300\ub97c \ub192\uc600\uc2b5\ub2c8\ub2e4.<\/p>\n<p>\ud3fc\ud398\ubcd1 \uc2dc\uc7a5 \ubcf4\uace0\uc11c \uc694\uc57d<\/p>\n<p>\ubcf8 \ubcf4\uace0\uc11c\ub294 \uae00\ub9ac\ucf54\uac90 \ucd95\uc801\ubcd1 2\ud615\uc73c\ub85c\ub3c4 \uc54c\ub824\uc9c4 \ud3fc\ud398\ubcd1 \uc2dc\uc7a5\uc5d0 \ub300\ud55c \uc2ec\uce35 \ubd84\uc11d\uc744 \uc81c\uacf5\ud569\ub2c8\ub2e4. \ud3fc\ud398\ubcd1\uc740 GAA(acid alpha-glucosidase) \ud6a8\uc18c \uacb0\ud54d\uc73c\ub85c \uc778\ud574 \ub9ac\uc18c\uc880\uc5d0 \uae00\ub9ac\ucf54\uac90\uc774 \ucd95\uc801\ub418\uc5b4 \uc2ec\uc7a5 \ubc0f \uace8\uaca9\uadfc\uc5d0 \uc7a5\uc560\ub97c \uc720\ubc1c\ud558\ub294 \ud76c\uadc0 \uc720\uc804\uc131 \ub300\uc0ac \uc9c8\ud658\uc785\ub2c8\ub2e4. \uc57d 40,000\uba85 \ucd9c\uc0dd\ub2f9 1\uba85\uaf34\ub85c \ubc1c\uc0dd\ud558\uba70, \uc885\uc885 \uce58\uba85\uc801\uc77c \uc218 \uc788\uc2b5\ub2c8\ub2e4.<\/p>\n<p>\uc2dc\uc7a5 \uac1c\uc694 \ubc0f \uc131\uc7a5 \uc804\ub9dd<br \/>\n\ud3fc\ud398\ubcd1 \uc2dc\uc7a5\uc740 2026\ub144 20\uc5b5 4\ucc9c\ub9cc \ub2ec\ub7ec \uaddc\ubaa8\uc5d0\uc11c 2031\ub144\uae4c\uc9c0 28\uc5b5 6\ucc9c\ub9cc \ub2ec\ub7ec\ub85c \uc131\uc7a5\ud560 \uac83\uc73c\ub85c \uc608\uc0c1\ub418\uba70, \uc5f0\ud3c9\uade0 \uc131\uc7a5\ub960(CAGR)\uc740 6.98%\uc5d0 \ub2ec\ud569\ub2c8\ub2e4. 2025\ub144 \uae30\uc900 \uc2dc\uc7a5 \ub9e4\ucd9c\uc758 89.75%\ub97c \ucc28\uc9c0\ud558\ub294 \ud6a8\uc18c \ub300\uccb4 \uc694\ubc95(ERT)\uc774 \ud604\uc7ac \uc2dc\uc7a5\uc744 \uc9c0\ubc30\ud558\ub294 \uc8fc\uc694 \uce58\ub8cc\ubc95\uc785\ub2c8\ub2e4. \uc9c0\uc5ed\ubcc4\ub85c\ub294 \uc2e0\uc0dd\uc544 \uc720\uc804\uccb4 \uc2a4\ud06c\ub9ac\ub2dd \ud655\ub300\uc640 \ud76c\uadc0 \uc9c8\ud658 \uce58\ub8cc\uc81c\uc5d0 \ub300\ud55c \ubcf4\ud5d8 \uc801\uc6a9 \uc99d\uac00\uc5d0 \ud798\uc785\uc5b4 \uc544\uc2dc\uc544-\ud0dc\ud3c9\uc591 \uc9c0\uc5ed\uc774 9.02%\uc758 \uac00\uc7a5 \ube60\ub978 \uc5f0\ud3c9\uade0 \uc131\uc7a5\ub960\uc744 \ubcf4\uc77c \uac83\uc73c\ub85c \uc804\ub9dd\ub429\ub2c8\ub2e4.<\/p>\n<p>\uc2dc\uc7a5 \ub3d9\uc778<br \/>\n\uc2dc\uc7a5 \uc131\uc7a5\uc744 \uacac\uc778\ud558\ub294 \uc8fc\uc694 \uc694\uc778\uc73c\ub85c\ub294 \u25b22\uc138\ub300 \uc2e0\uaddc \ud6a8\uc18c \ub300\uccb4 \uc694\ubc95(ERT)\uc758 \ucd9c\uc2dc \u25b2\uc2e0\uc0dd\uc544 \uc2a4\ud06c\ub9ac\ub2dd \uc758\ubb34\ud654 \ud655\ub300 \u25b2\uc784\uc0c1 \uc2dc\ud5d8 \uc911\uc778 mRNA \uae30\ubc18 ERT \ud50c\ub7ab\ud3fc \uac1c\ubc1c \u25b2\uc911\uad6d\uc758 \ud76c\uadc0 \uc9c8\ud658 \uce58\ub8cc\uc81c \ubcf4\ud5d8 \uc801\uc6a9 \uac00\uc18d\ud654 \u25b2\uc720\uc804\uc790 \uce58\ub8cc\uc81c \uc5f0\uad6c \uac1c\ubc1c \uc790\uae08 \uc99d\uac00 \u25b2\uae00\ub85c\ubc8c \ud76c\uadc0 \uc758\uc57d\ud488 \uc778\uc13c\ud2f0\ube0c \uc81c\uacf5 \ub4f1\uc774 \uc788\uc2b5\ub2c8\ub2e4.<\/p>\n<p>\uc2dc\uc7a5 \uc81c\uc57d \uc694\uc778<br \/>\n\ubc18\uba74, \uc2dc\uc7a5 \uc131\uc7a5\uc744 \uc800\ud574\ud558\ub294 \uc694\uc778\uc73c\ub85c\ub294 \u25b2\ub192\uc740 \uce58\ub8cc \ube44\uc6a9 \u25b2\uc9c4\ub2e8 \uc9c0\uc5f0 \ubc0f \ub0ae\uc740 \uc9c8\ubcd1 \uc778\uc9c0\ub3c4 \u25b2\ubc14\uc774\ub7ec\uc2a4 \ubca1\ud130 \uc81c\uc870 \ubcd1\ubaa9 \ud604\uc0c1 \u25b2\ubbf8\uad6d \ubc0f \uc720\ub7fd \uc2dc\uc7a5\uc5d0\uc11c\uc758 \uac00\uce58 \uae30\ubc18 \uac00\uaca9 \ucc45\uc815 \uc555\ub825 \ub4f1\uc774 \uc9c0\uc801\ub429\ub2c8\ub2e4. \ud2b9\ud788 \ub3c5\uc77c\uc758 \uacbd\uc6b0 \ud658\uc790 1\uc778\ub2f9 \uc5f0\uac04 \ud3c9\uade0 \uce58\ub8cc \ube44\uc6a9\uc774 483,907\uc720\ub85c(\uc57d 523,000\ub2ec\ub7ec)\uc5d0 \ub2ec\ud558\uc5ec, \uac00\uce58 \uae30\ubc18 \uac00\uaca9 \ucc45\uc815\uc5d0 \ub300\ud55c \uba74\ubc00\ud55c \uac80\ud1a0\uac00 \uc694\uad6c\ub418\uace0 \uc788\uc2b5\ub2c8\ub2e4.<\/p>\n<p>\uc2dc\uc7a5 \uc138\ubd84\ud654<br \/>\n\ubcf8 \ubcf4\uace0\uc11c\ub294 \ud3fc\ud398\ubcd1 \uc2dc\uc7a5\uc744 \ub2e4\uc74c\uacfc \uac19\uc740 \ub2e4\uc591\ud55c \uae30\uc900\uc73c\ub85c \uc138\ubd84\ud654\ud558\uc5ec \ubd84\uc11d\ud569\ub2c8\ub2e4.<br \/>\n*   \ud22c\uc5ec \uacbd\ub85c\ubcc4: \uacbd\uad6c, \ube44\uacbd\uad6c<br \/>\n*   \uce58\ub8cc \uc720\ud615\ubcc4: \ud6a8\uc18c \ub300\uccb4 \uc694\ubc95(ERT), \ubcd1\uc6a9 \uc694\ubc95, \uae30\ud0c0 \uc694\ubc95<br \/>\n*   \uc9c8\ubcd1 \ubc1c\ubcd1 \uc720\ud615\ubcc4: \uc601\uc544 \ubc1c\ubcd1 \ud3fc\ud398\ubcd1(IOPD), \ud6c4\uae30 \ubc1c\ubcd1 \ud3fc\ud398\ubcd1(LOPD)<br \/>\n*   \uc720\ud1b5 \ucc44\ub110\ubcc4: \ubcd1\uc6d0 \uc57d\uad6d, \uc18c\ub9e4 \uc57d\uad6d, \uc628\ub77c\uc778 \uc57d\uad6d, \uae30\ud0c0<br \/>\n*   \uc9c0\uc5ed\ubcc4: \ubd81\ubbf8(\ubbf8\uad6d, \uce90\ub098\ub2e4, \uba55\uc2dc\ucf54), \uc720\ub7fd(\ub3c5\uc77c, \uc601\uad6d, \ud504\ub791\uc2a4, \uc774\ud0c8\ub9ac\uc544, \uc2a4\ud398\uc778, \uae30\ud0c0 \uc720\ub7fd), \uc544\uc2dc\uc544-\ud0dc\ud3c9\uc591(\uc911\uad6d, \uc77c\ubcf8, \uc778\ub3c4, \ud638\uc8fc, \ud55c\uad6d, \uae30\ud0c0 \uc544\uc2dc\uc544-\ud0dc\ud3c9\uc591), \uc911\ub3d9 \ubc0f \uc544\ud504\ub9ac\uce74(GCC, \ub0a8\uc544\ud504\ub9ac\uce74, \uae30\ud0c0 \uc911\ub3d9 \ubc0f \uc544\ud504\ub9ac\uce74), \ub0a8\ubbf8(\ube0c\ub77c\uc9c8, \uc544\ub974\ud5e8\ud2f0\ub098, \uae30\ud0c0 \ub0a8\ubbf8)\ub85c \uad6c\ubd84\ud558\uc5ec \uc0c1\uc138\ud55c \uc2dc\uc7a5 \uaddc\ubaa8 \ubc0f \uc131\uc7a5 \uc608\uce21\uc744 \uc81c\uacf5\ud569\ub2c8\ub2e4.<\/p>\n<p>\uacbd\uc7c1 \ud658\uacbd \ubc0f \ubbf8\ub798 \uc804\ub9dd<br \/>\n\uacbd\uc7c1 \ud658\uacbd \ubd84\uc11d\uc5d0\uc11c\ub294 \uc2dc\uc7a5 \uc9d1\uc911\ub3c4, \uc2dc\uc7a5 \uc810\uc720\uc728 \ubd84\uc11d \ubc0f Sanofi, Amicus Therapeutics, Astellas Pharma \/ Audentes, Asklepios BioPharmaceutical (AskBio), Genethon, Orchard Therapeutics, Shionogi &#038; Co., Ltd., Beijing GeneCradle Technology Co., Ltd., Maze Therapeutics, Inc., Exerkine Corporation \ub4f1 \uc8fc\uc694 \uae30\uc5c5\ub4e4\uc758 \ud504\ub85c\ud544\uc744 \ub2e4\ub8f9\ub2c8\ub2e4. \ub610\ud55c, \uc2dc\uc7a5\uc758 \uae30\ud68c\uc640 \ubbf8\ub798 \uc804\ub9dd, \ubbf8\ucda9\uc871 \uc218\uc694 \ud3c9\uac00\ub97c \ud1b5\ud574 \ud5a5\ud6c4 \uc2dc\uc7a5 \ubc1c\uc804 \ubc29\ud5a5\uc744 \uc81c\uc2dc\ud569\ub2c8\ub2e4.<\/p>\n","protected":false},"author":1,"featured_media":0,"comment_status":"closed","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[1],"tags":[],"class_list":["post-56199","post","type-post","status-publish","format-standard","hentry","category-market-reports"],"_links":{"self":[{"href":"https:\/\/www.globalresearch.co.kr\/insight\/wp-json\/wp\/v2\/posts\/56199"}],"collection":[{"href":"https:\/\/www.globalresearch.co.kr\/insight\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.globalresearch.co.kr\/insight\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.globalresearch.co.kr\/insight\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/www.globalresearch.co.kr\/insight\/wp-json\/wp\/v2\/comments?post=56199"}],"version-history":[{"count":0,"href":"https:\/\/www.globalresearch.co.kr\/insight\/wp-json\/wp\/v2\/posts\/56199\/revisions"}],"wp:attachment":[{"href":"https:\/\/www.globalresearch.co.kr\/insight\/wp-json\/wp\/v2\/media?parent=56199"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.globalresearch.co.kr\/insight\/wp-json\/wp\/v2\/categories?post=56199"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.globalresearch.co.kr\/insight\/wp-json\/wp\/v2\/tags?post=56199"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}