{"id":54111,"date":"2026-02-23T20:54:02","date_gmt":"2026-02-23T20:54:02","guid":{"rendered":"https:\/\/www.globalresearch.co.kr\/insight\/spinal-muscular-atrophy-treatment-mordor\/"},"modified":"2026-10-02T16:43:38","modified_gmt":"2026-10-02T07:43:38","slug":"spinal-muscular-atrophy-treatment-mordor","status":"publish","type":"post","link":"https:\/\/www.globalresearch.co.kr\/insight\/spinal-muscular-atrophy-treatment-mordor\/","title":{"rendered":"\uc138\uacc4\uc758 \ucc99\uc218\uc131 \uadfc\uc704\ucd95\uc99d \uce58\ub8cc \uc2dc\uc7a5 \uaddc\ubaa8 \ubc0f \uc810\uc720\uc728 \ubd84\uc11d &#8211; \uc131\uc7a5 \ub3d9\ud5a5 \ubc0f \uc608\uce21 (2026-2031\ub144)"},"content":{"rendered":"<p><img decoding=\"async\" src=\"https:\/\/www.globalresearch.co.kr\/insight\/wp-content\/uploads\/2026\/02\/spinal-muscular-atrophy-treatment_1645508424348_players_graph_sma.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\/spinal-muscular-atrophy-treatment-size-image-1770826848906.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 \uc2e0\uc0dd\uc544 \uc120\ubcc4\uac80\uc0ac \uc758\ubb34\ud654\ub85c \uce58\ub8cc \uac00\ub2a5\ud55c \ud658\uc790\uad70 \ud655\ub300<\/li>\n<li class=\"toc-level-3\">4.2.2 \uace0\uc18c\ub4dd \uc2dc\uc7a5\uc5d0\uc11c SMN \ud45c\uc801 \uc720\uc804\uc790 \uce58\ub8cc\uc81c\uc758 \ube60\ub978 \ucc44\ud0dd<\/li>\n<li class=\"toc-level-3\">4.2.3 \uace0\uac00 \ud76c\uadc0\uc9c8\ud658 \uc57d\ubb3c\uc5d0 \ub300\ud55c \uae00\ub85c\ubc8c \uc9c0\ubd88\uc790\uc758 \uc218\uc6a9<\/li>\n<li class=\"toc-level-3\">4.2.4 \ucd08\uae30 \ub2e8\uacc4 \uc784\uc0c1\uc2dc\ud5d8\uc744 \uc704\ud55c \uc639\ud638 \ub2e8\uccb4 \uc790\uae08 \uc9c0\uc6d0 \uc99d\uac00<\/li>\n<li class=\"toc-level-3\">4.2.5 SMN \ubc0f \ubbf8\uc624\uc2a4\ud0c0\ud2f4\uc744 \ud45c\uc801\ud558\ub294 \ud30c\uc774\ud504\ub77c\uc778 \ubcd1\uc6a9 \uc694\ubc95<\/li>\n<li class=\"toc-level-3\">4.2.6 \uc7ac\ud0dd \ub514\uc9c0\ud138 \ubcf5\uc57d \uc21c\uc751\ub3c4 \ud50c\ub7ab\ud3fc\uc774 \ub9cc\uc131 \ud22c\uc5ec\ub97c \ucd09\uc9c4<\/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 \uc218\ubc31\ub9cc \ub2ec\ub7ec\uc758 \uac00\uaca9\ud45c\uc640 \ubd88\ud655\uc2e4\ud55c \uc7a5\uae30 \uc0c1\ud658<\/li>\n<li class=\"toc-level-3\">4.3.2 \uc218\uc561 \uc13c\ud130\uc758 \ucc99\uc218\uac15 \ub0b4 \ud22c\uc5ec \ubcf5\uc7a1\uc131 \ubc0f \uc6a9\ub7c9 \uc81c\uc57d<\/li>\n<li class=\"toc-level-3\">4.3.3 \uc2e0\ud765 \uacbd\uc81c\uad6d\uc758 \uc18c\uc544 \uc2e0\uacbd\uadfc \uc804\ubb38\uc758 \ubd80\uc871<\/li>\n<li class=\"toc-level-3\">4.3.4 AAV \uacf5\uae09\uc744 \uc704\ud55c \ubc14\uc774\ub7ec\uc2a4 \ubca1\ud130 \uc81c\uc870 \ubcd1\ubaa9 \ud604\uc0c1<\/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 \uc9c8\ubcd1 \uc720\ud615\ubcc4\n<ul>\n<li class=\"toc-level-3\">5.1.1 \uc720\ud615 0<\/li>\n<li class=\"toc-level-3\">5.1.2 \uc720\ud615 1<\/li>\n<li class=\"toc-level-3\">5.1.3 \uc720\ud615 2<\/li>\n<li class=\"toc-level-3\">5.1.4 \uc720\ud615 3<\/li>\n<li class=\"toc-level-3\">5.1.5 \uc720\ud615 4<\/li>\n<\/ul>\n<\/li>\n<li class=\"toc-level-2\">5.2 \uce58\ub8cc \uc591\uc2dd\ubcc4\n<ul>\n<li class=\"toc-level-3\">5.2.1 \uc720\uc804\uc790 \ub300\uccb4 \uc694\ubc95<\/li>\n<li class=\"toc-level-3\">5.2.2 SMN2 \uc2a4\ud50c\ub77c\uc774\uc2f1 \uc870\uc808\uc81c<\/li>\n<li class=\"toc-level-3\">5.2.3 \uc548\ud2f0\uc13c\uc2a4 \uc62c\ub9ac\uace0\ub274\ud074\ub808\uc624\ud0c0\uc774\ub4dc \uc694\ubc95<\/li>\n<li class=\"toc-level-3\">5.2.4 \uadfc\uc721 \ud45c\uc801 \/ \ubbf8\uc624\uc2a4\ud0c0\ud2f4 \uc5b5\uc81c\uc81c<\/li>\n<li class=\"toc-level-3\">5.2.5 \uc9c0\uc9c0 \ubc0f \ubcf4\uc870 \uc694\ubc95<\/li>\n<\/ul>\n<\/li>\n<li class=\"toc-level-2\">5.3 \ud22c\uc5ec \uacbd\ub85c\ubcc4\n<ul>\n<li class=\"toc-level-3\">5.3.1 \uacbd\uad6c<\/li>\n<li class=\"toc-level-3\">5.3.2 \uc218\ub9c9\uac15\ub0b4<\/li>\n<li class=\"toc-level-3\">5.3.3 \uc815\ub9e5\ub0b4<\/li>\n<\/ul>\n<\/li>\n<li class=\"toc-level-2\">5.4 \uc5f0\ub839 \uadf8\ub8f9\ubcc4\n<ul>\n<li class=\"toc-level-3\">5.4.1 \uc2e0\uc0dd\uc544 (0\u20136\uac1c\uc6d4)<\/li>\n<li class=\"toc-level-3\">5.4.2 \uc18c\uc544 (6\uac1c\uc6d4\u201312\uc138)<\/li>\n<li class=\"toc-level-3\">5.4.3 \uccad\uc18c\ub144 (12\u201318\uc138)<\/li>\n<li class=\"toc-level-3\">5.4.4 \uc131\uc778 (18\uc138 \uc774\uc0c1)<\/li>\n<\/ul>\n<\/li>\n<li class=\"toc-level-2\">5.5 \uc720\ud1b5 \ucc44\ub110\ubcc4\n<ul>\n<li class=\"toc-level-3\">5.5.1 \ubcd1\uc6d0 \uc57d\uad6d<\/li>\n<li class=\"toc-level-3\">5.5.2 \uc804\ubb38 \ud074\ub9ac\ub2c9<\/li>\n<li class=\"toc-level-3\">5.5.3 \uc628\ub77c\uc778 \ubc0f \uc18c\ub9e4 \uc57d\uad6d<\/li>\n<\/ul>\n<\/li>\n<li class=\"toc-level-2\">5.6 \uc9c0\uc5ed\ubcc4\n<ul>\n<li class=\"toc-level-3\">5.6.1 \ubd81\ubbf8<\/li>\n<li class=\"toc-level-4\">5.6.1.1 \ubbf8\uad6d<\/li>\n<li class=\"toc-level-4\">5.6.1.2 \uce90\ub098\ub2e4<\/li>\n<li class=\"toc-level-4\">5.6.1.3 \uba55\uc2dc\ucf54<\/li>\n<li class=\"toc-level-3\">5.6.2 \uc720\ub7fd<\/li>\n<li class=\"toc-level-4\">5.6.2.1 \ub3c5\uc77c<\/li>\n<li class=\"toc-level-4\">5.6.2.2 \uc601\uad6d<\/li>\n<li class=\"toc-level-4\">5.6.2.3 \ud504\ub791\uc2a4<\/li>\n<li class=\"toc-level-4\">5.6.2.4 \uc774\ud0c8\ub9ac\uc544<\/li>\n<li class=\"toc-level-4\">5.6.2.5 \uc2a4\ud398\uc778<\/li>\n<li class=\"toc-level-4\">5.6.2.6 \uc720\ub7fd \uae30\ud0c0 \uc9c0\uc5ed<\/li>\n<li class=\"toc-level-3\">5.6.3 \uc544\uc2dc\uc544 \ud0dc\ud3c9\uc591<\/li>\n<li class=\"toc-level-4\">5.6.3.1 \uc911\uad6d<\/li>\n<li class=\"toc-level-4\">5.6.3.2 \uc77c\ubcf8<\/li>\n<li class=\"toc-level-4\">5.6.3.3 \uc778\ub3c4<\/li>\n<li class=\"toc-level-4\">5.6.3.4 \ud638\uc8fc<\/li>\n<li class=\"toc-level-4\">5.6.3.5 \ub300\ud55c\ubbfc\uad6d<\/li>\n<li class=\"toc-level-4\">5.6.3.6 \uc544\uc2dc\uc544 \ud0dc\ud3c9\uc591 \uae30\ud0c0 \uc9c0\uc5ed<\/li>\n<li class=\"toc-level-3\">5.6.4 \uc911\ub3d9 \ubc0f \uc544\ud504\ub9ac\uce74<\/li>\n<li class=\"toc-level-4\">5.6.4.1 GCC<\/li>\n<li class=\"toc-level-4\">5.6.4.2 \ub0a8\uc544\ud504\ub9ac\uce74<\/li>\n<li class=\"toc-level-4\">5.6.4.3 \uc911\ub3d9 \ubc0f \uc544\ud504\ub9ac\uce74 \uae30\ud0c0 \uc9c0\uc5ed<\/li>\n<li class=\"toc-level-3\">5.6.5 \ub0a8\ubbf8<\/li>\n<li class=\"toc-level-4\">5.6.5.1 \ube0c\ub77c\uc9c8<\/li>\n<li class=\"toc-level-4\">5.6.5.2 \uc544\ub974\ud5e8\ud2f0\ub098<\/li>\n<li class=\"toc-level-4\">5.6.5.3 \ub0a8\ubbf8 \uae30\ud0c0 \uc9c0\uc5ed<\/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(\uc0ac\uc6a9 \uac00\ub2a5\ud55c \uacbd\uc6b0), \uc804\ub7b5 \uc815\ubcf4, \uc8fc\uc694 \uae30\uc5c5\uc758 \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 Biogen Inc.<\/li>\n<li class=\"toc-level-3\">6.3.2 Roche Holding AG (Genentech)<\/li>\n<li class=\"toc-level-3\">6.3.3 Novartis International AG<\/li>\n<li class=\"toc-level-3\">6.3.4 Ionis Pharmaceuticals Inc.<\/li>\n<li class=\"toc-level-3\">6.3.5 PTC Therapeutics Inc.<\/li>\n<li class=\"toc-level-3\">6.3.6 Scholar Rock Inc.<\/li>\n<li class=\"toc-level-3\">6.3.7 Cytokinetics Inc.<\/li>\n<li class=\"toc-level-3\">6.3.8 Sarepta Therapeutics Inc.<\/li>\n<li class=\"toc-level-3\">6.3.9 NMD Pharma A\/S<\/li>\n<li class=\"toc-level-3\">6.3.10 Astellas Pharma Inc.<\/li>\n<li class=\"toc-level-3\">6.3.11 Pfizer Inc.<\/li>\n<li class=\"toc-level-3\">6.3.12 Chugai Pharmaceutical Co., Ltd.<\/li>\n<li class=\"toc-level-3\">6.3.13 Sanofi S.A. (Genzyme)<\/li>\n<li class=\"toc-level-3\">6.3.14 Catalyst Pharmaceuticals<\/li>\n<li class=\"toc-level-3\">6.3.15 Regenxbio Inc.<\/li>\n<li class=\"toc-level-3\">6.3.16 Biohaven Pharmaceutical Holding Co.<\/li>\n<li class=\"toc-level-3\">6.3.17 Audentes Therapeutics (Astellas Gene Therapies)<\/li>\n<li class=\"toc-level-3\">6.3.18 BridgeBio Pharma Inc.<\/li>\n<li class=\"toc-level-3\">6.3.19 Shionogi &amp; Co., Ltd.<\/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>\ucc99\uc218\uc131 \uadfc\uc704\ucd95\uc99d \uce58\ub8cc \uc2dc\uc7a5 \uaddc\ubaa8 \ubc0f \uc810\uc720\uc728 \ubd84\uc11d: \uc131\uc7a5 \ub3d9\ud5a5 \ubc0f \uc608\uce21 (2026-2031)<\/p>\n<p># 1. \uc2dc\uc7a5 \uac1c\uc694 \ubc0f \uc8fc\uc694 \uc9c0\ud45c<\/p>\n<p>\ucc99\uc218\uc131 \uadfc\uc704\ucd95\uc99d(Spinal Muscular Atrophy, SMA) \uce58\ub8cc \uc2dc\uc7a5\uc740 2026\ub144 60\uc5b5 6\ucc9c\ub9cc \ub2ec\ub7ec\uc5d0\uc11c 2031\ub144 134\uc5b5 8\ucc9c\ub9cc \ub2ec\ub7ec \uaddc\ubaa8\ub85c \uc131\uc7a5\ud560 \uac83\uc73c\ub85c \uc608\uc0c1\ub418\uba70, 2026\ub144\ubd80\ud130 2031\ub144\uae4c\uc9c0 \uc5f0\ud3c9\uade0 \uc131\uc7a5\ub960(CAGR)\uc740 17.33%\uc5d0 \ub2ec\ud560 \uc804\ub9dd\uc785\ub2c8\ub2e4. \uc774\ub294 \ud76c\uadc0 \uc9c8\ud658 \ubd84\uc57c\uc5d0\uc11c \uae30\ub85d\ub41c \uac00\uc7a5 \ub192\uc740 \uc131\uc7a5 \uada4\uc801 \uc911 \ud558\ub098\ub85c, \uc131\uc219\ud55c \uc2e0\uc0dd\uc544 \uc120\ubcc4 \uc778\ud504\ub77c, \uc720\uc804\uc790 \uce58\ub8cc\uc81c\uc758 \ube60\ub978 \ucc44\ud0dd, \uadf8\ub9ac\uace0 \uc6b0\ud638\uc801\uc778 \uc0c1\ud658 \uc815\ucc45\uc774 \ubcf5\ud569\uc801\uc73c\ub85c \uc791\uc6a9\ud55c \uacb0\uacfc\uc785\ub2c8\ub2e4. \uc544\uc2dc\uc544 \ud0dc\ud3c9\uc591 \uc9c0\uc5ed\uc774 \uac00\uc7a5 \ube60\ub974\uac8c \uc131\uc7a5\ud558\ub294 \uc2dc\uc7a5\uc73c\ub85c \uc608\uce21\ub418\uba70, \ubd81\ubbf8 \uc9c0\uc5ed\uc740 \uac00\uc7a5 \ud070 \uc2dc\uc7a5 \uc810\uc720\uc728\uc744 \uc720\uc9c0\ud558\uace0 \uc788\uc2b5\ub2c8\ub2e4. \uc2dc\uc7a5 \uc9d1\uc911\ub3c4\ub294 \uc911\uac04 \uc218\uc900\uc785\ub2c8\ub2e4.<\/p>\n<p># 2. \uc2dc\uc7a5 \uc138\ubd84\ud654<\/p>\n<p>\ubcf8 \ubcf4\uace0\uc11c\ub294 \ucc99\uc218\uc131 \uadfc\uc704\ucd95\uc99d \uce58\ub8cc \uc2dc\uc7a5\uc744 \ub2e4\uc74c\uacfc \uac19\uc774 \uc138\ubd84\ud654\ud558\uc5ec \ubd84\uc11d\ud569\ub2c8\ub2e4.<br \/>\n*   \uc720\ud615\ubcc4: Type I, Type II, Type III, Type IV<br \/>\n*   \uc2dc\uc220\ubcc4: \uc720\uc804\uc790 \ub300\uccb4 \uc694\ubc95(Gene Replacement Therapy), \uc57d\ubb3c \uc694\ubc95(Drug Therapy)<br \/>\n*   \ud22c\uc5ec \uacbd\ub85c\ubcc4: \uacbd\uad6c(Oral), \uc218\ub9c9\uac15\ub0b4(Intrathecal)<br \/>\n*   \uc9c0\uc5ed\ubcc4: \ubd81\ubbf8, \uc720\ub7fd, \uc544\uc2dc\uc544 \ud0dc\ud3c9\uc591, \uc911\ub3d9 \ubc0f \uc544\ud504\ub9ac\uce74, \ub0a8\ubbf8<\/p>\n<p># 3. \uc2dc\uc7a5 \ub3d9\uc778 \ubc0f \uc81c\uc57d \uc694\uc778<\/p>\n<p>3.1. \uc2dc\uc7a5 \ub3d9\uc778<\/p>\n<p>*   \uc2e0\uc0dd\uc544 \uc120\ubcc4 \uac80\uc0ac \uc758\ubb34\ud654\ub85c \uce58\ub8cc \uac00\ub2a5\ud55c \ud658\uc790 \ud480 \ud655\ub300: 2024\ub144 1\uc6d4\ubd80\ud130 \ubbf8\uad6d \uc804\uc5ed\uc5d0\uc11c \uc2dc\ud589\ub41c \uc2e0\uc0dd\uc544 \uc120\ubcc4 \uac80\uc0ac\ub294 SMA\ub97c \ubb34\uc99d\uc0c1 \ub2e8\uacc4\uc5d0\uc11c \uc2dd\ubcc4\ud558\uc5ec \uace0\ud6a8\ub2a5 \uce58\ub8cc\uc758 \ucd5c\uc801 \uc2dc\uae30\ub97c \uac00\ub2a5\ud558\uac8c \ud569\ub2c8\ub2e4. \ub300\ub9cc, \ud5dd\uac00\ub9ac, \uc138\ub974\ube44\uc544 \ub4f1\uc5d0\uc11c\ub3c4 \uc720\uc0ac\ud55c \ud504\ub85c\uadf8\ub7a8\uc774 \ub3c4\uc785\ub418\uc5b4 65\ub9cc \uba85 \uc774\uc0c1\uc758 \uc601\uc544\ub97c \uc120\ubcc4\ud588\uc73c\uba70, \uc774\ub294 \ub2e4\uc591\ud55c \ubcf4\uac74 \uc2dc\uc2a4\ud15c\uc5d0\uc11c\uc758 \ud655\uc7a5 \uac00\ub2a5\uc131\uc744 \ubcf4\uc5ec\uc90d\ub2c8\ub2e4. \uc870\uae30 \uc9c4\ub2e8\uc740 \ud604\uc800\ud788 \uac1c\uc120\ub41c \uc6b4\ub3d9 \uacb0\uacfc\ub97c \uac00\uc838\uc640 \uace0\uac00\uc758 \uce58\ub8cc\uc5d0 \ub300\ud55c \uc9c0\ubd88\uc790\uc758 \uc0c1\ud658 \uc758\uc9c0\ub97c \uac15\ud654\ud569\ub2c8\ub2e4.<br \/>\n*   \uace0\uc18c\ub4dd \uc2dc\uc7a5\uc5d0\uc11c SMN \ud45c\uc801 \uc720\uc804\uc790 \uce58\ub8cc\uc81c \ucc44\ud0dd \uc99d\uac00: \uc548\ud2f0\uc13c\uc2a4 \uc62c\ub9ac\uace0\ub274\ud074\ub808\uc624\ud0c0\uc774\ub4dc, \uc720\uc804\uc790 \ub300\uccb4 \ubca1\ud130, \uc800\ubd84\uc790 \uc2a4\ud50c\ub77c\uc774\uc2f1 \uc870\uc808\uc81c \ub4f1 SMN \ud45c\uc801 \uce58\ub8cc\uc81c\ub294 SMN \ub2e8\ubc31\uc9c8 \uc218\uce58\ub97c \ud68c\ubcf5\uc2dc\ud0a4\ub294 \ub2e4\uc591\ud55c \uacbd\ub85c\ub97c \uc81c\uacf5\ud569\ub2c8\ub2e4. \ub204\uc2dc\ub108\uc13c\uc758 \uc6a9\ub7c9 \uc99d\ub7c9\uc740 \ud608\uc7a5 \uc2e0\uacbd\uc12c\uc720 \uacbd\uc1c4(neurofilament light chain)\ub97c 94% \uac10\uc18c\uc2dc\ucf30\uc73c\uba70, \ub85c\uc288\uc758 2025\ub144 2\uc6d4 \uacbd\uad6c\uc6a9 \uc815\uc81c \uc2b9\uc778\uc740 \uce58\ub8cc \ubd80\ub2f4\uc744 \uc904\uc600\uc2b5\ub2c8\ub2e4. \ub178\ubc14\ud2f0\uc2a4\ub294 \uc218\ub9c9\uac15\ub0b4 \uc624\ub098\uc148\ub178\uc9c4 \uc544\ubca0\ud30c\ub974\ubcf4\ubca1\uc744 \uc0ac\uc6a9\ud558\uc5ec \ub098\uc774\ub4e0 \uc5b4\ub9b0\uc774\uc5d0\uac8c\uc11c HFMSE \uc810\uc218 2.39\uc810 \ud5a5\uc0c1\uc744 \ub2ec\uc131\ud558\uc5ec \uc720\uc804\uc790 \uce58\ub8cc\uc758 \uc801\uc6a9 \ub300\uc0c1\uc744 \ub113\ud614\uc2b5\ub2c8\ub2e4.<br \/>\n*   \uace0\uac00 \ud76c\uadc0 \uc9c8\ud658 \uc57d\ubb3c\uc5d0 \ub300\ud55c \uc804 \uc138\uacc4 \uc9c0\ubd88\uc790\uc758 \uc218\uc6a9 \uc99d\uac00: \ub204\uc2dc\ub108\uc13c\uc758 \ube44\uc6a9 \ud6a8\uc728\uc131 \ube44\uc728\uc774 QALY\ub2f9 21\ub9cc \ub2ec\ub7ec\ub97c \ucd08\uacfc\ud568\uc5d0\ub3c4 \ubd88\uad6c\ud558\uace0, \ub300\ubd80\ubd84\uc758 \ubbf8\uad6d \ubc0f \uc8fc\uc694 \uc720\ub7fd \uc9c0\ubd88\uc790\ub4e4\uc740 SMA \uce58\ub8cc\uc5d0 \ub300\ud55c \uc804\uad6d\uc801\uc778 \ubcf4\uc7a5\uc744 \uc720\uc9c0\ud558\uace0 \uc788\uc2b5\ub2c8\ub2e4. \uc774\ub294 \uc870\uae30 \uac1c\uc785\uc774 \ud3c9\uc0dd\uc5d0 \uac78\uce5c \uc778\uacf5\ud638\ud761\uae30 \ubc0f \uc815\ud615\uc678\uacfc\uc801 \ube44\uc6a9\uc744 \uc608\ubc29\ud558\uae30 \ub54c\ubb38\uc785\ub2c8\ub2e4. \uace0\uc544 \uc758\uc57d\ud488 \uc778\uc13c\ud2f0\ube0c, \uc2e4\uc81c \uc784\uc0c1 \ub370\uc774\ud130, \uc131\uacfc \uae30\ubc18 \uacc4\uc57d \ub4f1\uc774 \uc0c1\ud658 \uac00\ub2a5\uc131\uc744 \uc9c0\uc18d\uc801\uc73c\ub85c \uac15\ud654\ud558\uace0 \uc788\uc2b5\ub2c8\ub2e4.<br \/>\n*   \uc639\ud638 \ub2e8\uccb4\uc758 \ucd08\uae30 \ub2e8\uacc4 \uc784\uc0c1 \uc2dc\ud5d8 \uc790\uae08 \uc9c0\uc6d0 \uc99d\uac00: Cure SMA, Muscular Dystrophy UK, SMA Foundation\uacfc \uac19\uc740 \ube44\uc601\ub9ac \ub2e8\uccb4\ub4e4\uc740 RNA \ud3b8\uc9d1 \ubc0f \uc2e0\uacbd \ubcf4\ud638 \uc800\ubd84\uc790 \ud654\ud569\ubb3c\uacfc \uac19\uc740 \ud0d0\uc0c9\uc801 \uc811\uadfc \ubc29\uc2dd\uc5d0 \ubcf4\uc870\uae08\uc744 \uc9c0\uc6d0\ud569\ub2c8\ub2e4. \uc774\ub294 \uac1c\ub150 \uc99d\uba85 \ub2e8\uacc4\ub97c \uac00\uc18d\ud654\ud558\uace0 \uae30\uc220 \ud50c\ub7ab\ud3fc\uc758 \uc704\ud5d8\uc744 \uc904\uc774\uba70 \ubca4\ucc98 \uce90\ud53c\ud0c8\uc758 \ud6c4\uc18d \ud22c\uc790\ub97c \uc720\uce58\ud569\ub2c8\ub2e4.<br \/>\n*   SMN \ubc0f \ubbf8\uc624\uc2a4\ud0c0\ud2f4 \ud45c\uc801 \ud30c\uc774\ud504\ub77c\uc778 \ubcf5\ud569 \uc694\ubc95: SMN \ubc0f \ubbf8\uc624\uc2a4\ud0c0\ud2f4\uc744 \ub3d9\uc2dc\uc5d0 \ud45c\uc801\ud558\ub294 \ubcf5\ud569 \uc694\ubc95 \ud30c\uc774\ud504\ub77c\uc778\uc740 \uc2dc\uc7a5 \uc131\uc7a5\uc5d0 \uae0d\uc815\uc801\uc778 \uc601\ud5a5\uc744 \ubbf8\uce60 \uac83\uc73c\ub85c \uc608\uc0c1\ub429\ub2c8\ub2e4.<br \/>\n*   \uc7ac\ud0dd \ub514\uc9c0\ud138 \uc21c\uc751\ub3c4 \ud50c\ub7ab\ud3fc\uc744 \ud1b5\ud55c \ub9cc\uc131 \ud22c\uc5ec \uc99d\uc9c4: \uac1c\ubc1c\ub41c \uc2dc\uc7a5\uc5d0\uc11c \uc7ac\ud0dd \ub514\uc9c0\ud138 \uc21c\uc751\ub3c4 \ud50c\ub7ab\ud3fc\uc740 \ub9cc\uc131 \ud22c\uc5ec\uc758 \ud3b8\uc758\uc131\uc744 \ub192\uc5ec \uc2dc\uc7a5 \uc131\uc7a5\uc5d0 \uae30\uc5ec\ud569\ub2c8\ub2e4.<\/p>\n<p>3.2. \uc2dc\uc7a5 \uc81c\uc57d \uc694\uc778<\/p>\n<p>*   \uc218\ubc31\ub9cc \ub2ec\ub7ec\uc5d0 \ub2ec\ud558\ub294 \ub192\uc740 \uc57d\uac00 \ubc0f \ubd88\ud655\uc2e4\ud55c \uc7a5\uae30 \uc0c1\ud658: \uc878\uac90\uc2a4\ub9c8\uc640 \uac19\uc740 \uce58\ub8cc\uc81c\uc758 \uc218\ubc31\ub9cc \ub2ec\ub7ec\uc5d0 \ub2ec\ud558\ub294 \ube44\uc6a9\uacfc \ud3c9\uc0dd \ud6a8\ub2a5\uc740 \ud2b9\ud788 \uc911\uc18c\ub4dd \uad6d\uac00\uc5d0\uc11c \uc9c0\ubd88\uc790\uc758 \uc608\uc0b0\uc5d0 \ubd80\ub2f4\uc744 \uc90d\ub2c8\ub2e4. \uc804\ud1b5\uc801\uc778 \uc784\uacc4\uce58\ub97c \ucd08\uacfc\ud558\ub294 \uc810\uc9c4\uc801 \ube44\uc6a9 \ud6a8\uc728\uc131 \ube44\uc728\uc740 \uc704\ud5d8 \uacf5\uc720 \uacc4\uc57d\uc744 \uc720\ub3c4\ud558\uc5ec \ucc44\ud0dd\uc744 \uc9c0\uc5f0\uc2dc\ud0b5\ub2c8\ub2e4.<br \/>\n*   \uc218\ub9c9\uac15\ub0b4 \ud22c\uc5ec\uc758 \ubcf5\uc7a1\uc131 \ubc0f \uc8fc\uc785 \uc13c\ud130\uc758 \uc6a9\ub7c9 \uc81c\uc57d: \uc218\ub9c9\uac15\ub0b4 \ud22c\uc5ec\ub294 \ub9ce\uc740 \uc9c0\uc5ed \ubcd1\uc6d0\uc5d0\uc11c \uc774\uc6a9\ud560 \uc218 \uc5c6\ub294 \uc694\ucd94 \ucc9c\uc790 \uc804\ubb38 \uc9c0\uc2dd\uacfc \ud615\uad11 \ud22c\uc2dc \uc7a5\ube44\uac00 \ud544\uc694\ud569\ub2c8\ub2e4. \uc77c\ubd80 \ubbf8\uad6d \ubc0f \uc720\ub7fd \uc13c\ud130\uc5d0\uc11c\ub294 \uc608\uc57d \uc801\uccb4\uac00 \uba87 \uc8fc\uc5d0 \ub2ec\ud558\uc5ec \uc911\uc694\ud55c \ubb34\uc99d\uc0c1 \uae30\uac04 \ub0b4 \uce58\ub8cc \uc2dc\uc791\uc774 \uc9c0\uc5f0\ub418\uace0 \uc788\uc2b5\ub2c8\ub2e4. \uc18c\uc544 \ucc99\ucd94 \ubcc0\ud615\uc740 \uc2dc\uc220 \uc704\ud5d8\uc744 \uc99d\uac00\uc2dc\ud0a4\uace0 \uc608\uc57d \uc2dc\uac04\uc744 \ub298\ub824 \uc6a9\ub7c9\uc744 \ub354\uc6b1 \uc555\ubc15\ud569\ub2c8\ub2e4.<br \/>\n*   \uc2e0\ud765 \uacbd\uc81c\uad6d\uc758 \uc18c\uc544 \uc2e0\uacbd\uadfc \uc804\ubb38\uc758 \ubd80\uc871: \ub77c\ud2f4 \uc544\uba54\ub9ac\uce74, \ub0a8\uc544\uc2dc\uc544, \uc544\ud504\ub9ac\uce74 \ub4f1 \uc2e0\ud765 \uacbd\uc81c\uad6d\uc5d0\uc11c\ub294 \uc18c\uc544 \uc2e0\uacbd\uadfc \uc804\ubb38\uc758\uac00 \ubd80\uc871\ud558\uc5ec \uce58\ub8cc \uc811\uadfc\uc131\uc744 \uc81c\ud55c\ud569\ub2c8\ub2e4.<br \/>\n*   AAV \uacf5\uae09\uc744 \uc704\ud55c \ubc14\uc774\ub7ec\uc2a4 \ubca1\ud130 \uc81c\uc870 \ubcd1\ubaa9 \ud604\uc0c1: \uc720\uc804\uc790 \uce58\ub8cc\uc81c\uc5d0 \ud544\uc218\uc801\uc778 AAV(Adeno-Associated Virus) \ubca1\ud130 \uc81c\uc870\uc758 \ubcd1\ubaa9 \ud604\uc0c1\uc740 \uc720\uc804\uc790 \uce58\ub8cc\uc81c\uc758 \ubcf4\uae09\uc744 \uc800\ud574\ud569\ub2c8\ub2e4.<\/p>\n<p># 4. \uc138\uadf8\uba3c\ud2b8 \ubd84\uc11d<\/p>\n<p>4.1. \uc9c8\ubcd1 \uc720\ud615\ubcc4: Type 1 SMA\uac00 \uc2dc\uc7a5 \uc9d1\uc911\uc744 \uc8fc\ub3c4<\/p>\n<p>2025\ub144 Type 1 SMA\ub294 \ucc99\uc218\uc131 \uadfc\uc704\ucd95\uc99d \uce58\ub8cc \uc2dc\uc7a5\uc758 48.05%\ub97c \ucc28\uc9c0\ud588\uc2b5\ub2c8\ub2e4. \uc0dd\uba85\uc744 \uc704\ud611\ud558\ub294 \ud2b9\uc131\uc73c\ub85c \uc778\ud574 \uc989\uac01\uc801\uc778 \uce58\ub8cc \uac1c\uc2dc\uc640 \ud504\ub9ac\ubbf8\uc5c4 \uac00\uaca9 \ucc45\uc815\uc774 \uc774\ub8e8\uc5b4\uc9d1\ub2c8\ub2e4. \ubcf4\ud3b8\uc801\uc778 \uc2e0\uc0dd\uc544 \uc120\ubcc4 \uac80\uc0ac\ub294 \uc9c4\ub2e8\uc744 \ubb34\uc99d\uc0c1 \ub2e8\uacc4\ub85c \uc804\ud658\ud558\uc5ec \uc778\uacf5\ud638\ud761\uae30 \uc5c6\uc774 \uc0dd\uc874\ud558\ub294 \ube44\uc728\uc744 \uac1c\uc120\ud558\uace0 \uc870\uae30 \uc720\uc804\uc790 \uce58\ub8cc \uc0ac\uc6a9\uc5d0 \ub300\ud55c \uc784\uc0c1\uc801 \ud569\uc758\ub97c \uac15\ud654\ud588\uc2b5\ub2c8\ub2e4. Type 0\uc740 2031\ub144\uae4c\uc9c0 18.02%\uc758 CAGR\ub85c \uc131\uc7a5\ud560 \uac83\uc73c\ub85c \uc608\uc0c1\ub418\uba70, Type 2 \ubc0f 3\uc740 \ud3c9\uc0dd \uc720\uc9c0 \uc6a9\ub7c9 \ud22c\uc5ec\uac00 \ud45c\uc900 \uad00\ud589\uc73c\ub85c \ub0a8\uc544 \uafb8\uc900\ud55c \ubc18\ubcf5 \uc218\uc775\uc744 \ucc3d\ucd9c\ud569\ub2c8\ub2e4.<\/p>\n<p>4.2. \uce58\ub8cc \uc591\uc2dd\ubcc4: SMN2 \uc2a4\ud50c\ub77c\uc774\uc2f1 \uc870\uc808\uc81c\uc758 \uc9c0\ubc30\ub825\uc5d0 \ub300\ud55c \uc720\uc804\uc790 \uce58\ub8cc\uc758 \ub3c4\uc804<\/p>\n<p>2025\ub144 SMN2 \uc2a4\ud50c\ub77c\uc774\uc2f1 \uc870\uc808\uc81c\ub294 \ucc99\uc218\uc131 \uadfc\uc704\ucd95\uc99d \uce58\ub8cc \uc2dc\uc7a5 \uc810\uc720\uc728\uc758 42.35%\ub97c \ucc28\uc9c0\ud588\uc2b5\ub2c8\ub2e4. \uc774\ub294 \ubaa8\ub4e0 \ud45c\ud604\ud615\uc5d0 \ub300\ud55c \uc801\uc6a9 \uac00\ub2a5\uc131, \uacbd\uad6c \ub610\ub294 \uc218\ub9c9\uac15\ub0b4 \ud22c\uc5ec \uc635\uc158, \uadf8\ub9ac\uace0 \uc6b0\ud638\uc801\uc778 \uc548\uc804\uc131 \uae30\ub85d \ub355\ubd84\uc785\ub2c8\ub2e4. \uc720\uc804\uc790 \ub300\uccb4 \uc694\ubc95\uc740 \uc81c\uc870 \uc218\uc728 \uac1c\uc120\uacfc \ub098\uc774\ub4e0 \uc5b4\ub9b0\uc774\ub97c \uc704\ud55c \uc218\ub9c9\uac15\ub0b4 \uc81c\ud615 \uac1c\ubc1c\ub85c 18.41%\uc758 \uac00\uc7a5 \ube60\ub978 CAGR\uc744 \ubcf4\uc785\ub2c8\ub2e4. \uc548\ud2f0\uc13c\uc2a4 \uc62c\ub9ac\uace0\ub274\ud074\ub808\uc624\ud0c0\uc774\ub4dc\ub294 \uc6a9\ub7c9 \uc99d\ub7c9 \ud504\ub85c\ud1a0\ucf5c\ub85c \uafb8\uc900\ud55c \uc810\uc720\uc728\uc744 \uc720\uc9c0\ud558\uace0 \uc788\uc2b5\ub2c8\ub2e4. \uc2a4\uce7c\ub77c \ub85d\uc758 \uc544\ud53c\ud14c\uadf8\ub85c\ub9d9\uacfc \uac19\uc740 \uadfc\uc721 \ud45c\uc801 \uc81c\uc81c\ub294 SMN \ud68c\ubcf5 \uce58\ub8cc\uc640 \ubcd1\uc6a9 \uc2dc \uc2dc\ub108\uc9c0 \ud6a8\uacfc\ub97c \uc57d\uc18d\ud558\uba70, RNA \ud3b8\uc9d1 \ub3c4\uad6c\uc758 \ud30c\uc774\ud504\ub77c\uc778\uc740 \ud601\uc2e0\uc744 \uc9c0\uc18d\ud560 \uac83\uc73c\ub85c \uc608\uc0c1\ub429\ub2c8\ub2e4.<\/p>\n<p>4.3. \ud22c\uc5ec \uacbd\ub85c\ubcc4: \uc218\ub9c9\uac15\ub0b4 \uc778\ud504\ub77c\uac00 \uacbd\uad6c \uce58\ub8cc\uc81c \uc131\uc7a5\uc744 \uac00\ub2a5\ud558\uac8c \ud568<\/p>\n<p>2025\ub144 \uc218\ub9c9\uac15\ub0b4 \ud22c\uc5ec\ub294 \ucc99\uc218\uc131 \uadfc\uc704\ucd95\uc99d \uce58\ub8cc \uc2dc\uc7a5 \uaddc\ubaa8\uc758 51.05%\ub97c \ucc28\uc9c0\ud588\uc2b5\ub2c8\ub2e4. \uc624\ub79c \uae30\uac04 \ud655\ub9bd\ub41c \uc694\ucd94 \ucc9c\uc790 \ud504\ub85c\ud1a0\ucf5c\uacfc \uc8fc\uc785 \uc2dc\uc124\uc774 \uc774\ub7ec\ud55c \uad00\ud589\uc744 \uace0\ucc29\ud654\ud588\uc2b5\ub2c8\ub2e4. \uadf8\ub7ec\ub098 \uacbd\uad6c\uc6a9 \ub9ac\uc2a4\ub514\ud50c\ub78c\uacfc \uc0c8\ub85c \uc2b9\uc778\ub41c \uc5d0\ube0c\ub9ac\uc2a4\ub514 \uc815\uc81c\ub294 \uc7ac\ud0dd \ud22c\uc5ec\uc758 \ud3b8\ub9ac\ud568\uacfc \uc720\uc0ac\ud55c \uc911\ucd94\uc2e0\uacbd\uacc4 \ub178\ucd9c \ub355\ubd84\uc5d0 18.22%\uc758 CAGR\ub85c \uc131\uc7a5\ud558\uace0 \uc788\uc2b5\ub2c8\ub2e4. \uc815\ub9e5 \uc8fc\uc0ac \uc720\uc804\uc790 \uce58\ub8cc\ub294 13.5kg \ubbf8\ub9cc\uc758 \uc601\uc544\uc5d0\uac8c \uc5ec\uc804\ud788 \uc911\uc694\ud558\uc9c0\ub9cc, AAV \uc81c\uc870 \uc6a9\ub7c9 \uc81c\ud55c\uc73c\ub85c \uc778\ud574 \ud655\uc7a5 \ud55c\uacc4\uc5d0 \uc9c1\uba74\ud574 \uc788\uc2b5\ub2c8\ub2e4.<\/p>\n<p>4.4. \uc5f0\ub839 \uadf8\ub8f9\ubcc4: \uc18c\uc544 \ud658\uc790 \uc9d1\uc911\uc740 \uc870\uae30 \uac1c\uc785 \ucd08\uc810\uc744 \ubc18\uc601<\/p>\n<p>2025\ub144 6\uac1c\uc6d4\uc5d0\uc11c 12\uc138 \uc0ac\uc774\uc758 \uc18c\uc544 \ud658\uc790 \ucf54\ud638\ud2b8\ub294 \ucc99\uc218\uc131 \uadfc\uc704\ucd95\uc99d \uce58\ub8cc \uc2dc\uc7a5 \uaddc\ubaa8\uc758 61.80%\ub97c \ucc28\uc9c0\ud588\uc2b5\ub2c8\ub2e4. \uc2e0\uacbd\uacfc \uc758\uc0ac\ub4e4\uc740 \ub3cc\uc774\ud0ac \uc218 \uc5c6\ub294 \uc6b4\ub3d9 \ub2e8\uc704 \uc190\uc2e4 \uc774\uc804\uc5d0 \uc801\uadf9\uc801\uc778 \uce58\ub8cc\ub97c \uc120\ud638\ud558\ubbc0\ub85c \uc774 \ubd80\ubb38\uc774 \uc8fc\uc694 \uc218\uc775 \ub3d9\ub825\uc785\ub2c8\ub2e4. \ubb34\uc99d\uc0c1 \uc120\ubcc4 \ubc0f \uce58\ub8cc \ud504\ub85c\uadf8\ub7a8\uc5d0 \ud798\uc785\uc5b4 \uc2e0\uc0dd\uc544 \ucc98\ubc29\uc740 \ub9e4\ub144 18.05% \uc99d\uac00\ud558\uace0 \uc788\uc2b5\ub2c8\ub2e4. \uccad\uc18c\ub144 \ubc0f \uc131\uc778 \ud658\uc790\ub294 \ud6c4\uae30 \ubc1c\ubcd1 \uc0ac\ub840 \uc9c4\ub2e8 \uc54c\uace0\ub9ac\uc998\uc758 \ubc1c\uc804\uacfc \uae30\uc874 \uc7a5\uc560\uc5d0\uc11c\ub3c4 \uc0b6\uc758 \uc9c8 \ud5a5\uc0c1\uc744 \uc778\uc815\ud558\ub294 \uc9c0\ubd88\uc790 \ub355\ubd84\uc5d0 \ube44\uc911\uc774 \uc791\uc9c0\ub9cc \uc99d\uac00\ud558\uace0 \uc788\uc2b5\ub2c8\ub2e4.<\/p>\n<p>4.5. \uc720\ud1b5 \ucc44\ub110\ubcc4: \ubcd1\uc6d0 \uc57d\uad6d\uc774 \uc804\ubb38 \uce58\ub8cc \ub124\ud2b8\uc6cc\ud06c\uc758 \uc911\uc2ec<\/p>\n<p>2025\ub144 \ubcd1\uc6d0 \uc57d\uad6d\uc740 \uce58\ub8cc\uc81c\uc758 46.85%\ub97c \uc870\uc81c\ud588\uc2b5\ub2c8\ub2e4. \ucf5c\ub4dc \uccb4\uc778, \uc720\uc804\uc790 \uce58\ub8cc \ubca1\ud130 \uc7ac\uace0, \uc751\uae09 \ub300\uc751 \uc900\ube44 \ub4f1 \ubcf5\uc7a1\ud55c \ubb3c\ub958\ub294 \ubcd1\uc6d0 \uc57d\uad6d\uc758 \uc911\uc2ec \uc5ed\ud560\uc744 \uc815\ub2f9\ud654\ud569\ub2c8\ub2e4. \uc628\ub77c\uc778 \ubc0f \uc18c\ub9e4 \uc57d\uad6d\uc740 \uacbd\uad6c \uc81c\ud615\uc758 \uc7ac\ud0dd \ubc30\uc1a1 \ubc0f \uc6d0\uaca9 \uc21c\uc751\ub3c4 \ubaa8\ub2c8\ud130\ub9c1 \ub3c4\uad6c\uc5d0 \ud798\uc785\uc5b4 18.92%\uc758 CAGR\ub85c \uc131\uc7a5\ud558\uace0 \uc788\uc2b5\ub2c8\ub2e4.<\/p>\n<p># 5. \uc9c0\uc5ed \ubd84\uc11d<\/p>\n<p>*   \ubd81\ubbf8: 2025\ub144 \uc804 \uc138\uacc4 \uc218\uc775\uc758 42.30%\ub97c \ucc28\uc9c0\ud588\uc73c\uba70, \uc774\ub294 \uad11\ubc94\uc704\ud55c \uc2e0\uc0dd\uc544 \uc120\ubcc4 \uac80\uc0ac, \ubc00\uc9d1\ub41c \uc2e0\uacbd\uadfc \uc804\ubb38\uc758 \ub124\ud2b8\uc6cc\ud06c, \uadf8\ub9ac\uace0 6~7\uc790\ub9ac \uc22b\uc790\uc758 \uc57d\uac00\ub97c \uc218\uc6a9\ud558\ub294 \uc131\uc219\ud55c \uc9c0\ubd88\uc790 \uba54\ucee4\ub2c8\uc998\uc5d0 \ud798\uc785\uc740 \uacb0\uacfc\uc785\ub2c8\ub2e4. \ubbf8\uad6d CMS\uc758 \uc601\uc544 \uc120\ubcc4 \uc758\ubb34\ud654\ub294 \uac70\uc758 \ubcf4\ud3b8\uc801\uc778 \uc870\uae30 \uc9c4\ub2e8\uc744 \ubcf4\uc7a5\ud558\uba70, \ubbfc\uac04 \ubcf4\ud5d8\uc0ac\uc640 \uba54\ub514\ucf00\uc774\ub4dc \ud504\ub85c\uadf8\ub7a8\uc740 \uad11\ubc94\uc704\ud55c \uc811\uadfc\uc744 \ubcf4\uc7a5\ud558\ub294 \uc131\uacfc \uae30\ubc18 \uacc4\uc57d\uc744 \ud611\uc0c1\ud569\ub2c8\ub2e4.<br \/>\n*   \uc720\ub7fd: EMA\uc758 \uc911\uc559 \uc9d1\uc911\uc2dd \uc2b9\uc778\uc774 \ub77c\ubca8 \uc870\ud654\ub97c \uac04\uc18c\ud654\ud558\uba74\uc11c \uafb8\uc900\ud55c \uc810\uc9c4\uc801 \uc131\uc7a5\uc744 \ubcf4\uc774\uc9c0\ub9cc, \uac1c\ubcc4 \uad6d\uac00 \ud611\uc0c1\uac00\ub4e4\uc740 \uc2dc\uc7a5 \uc9c4\uc785 \uc2dc\uae30\ub97c \uc870\uc808\ud569\ub2c8\ub2e4. \ub3c5\uc77c\uacfc \ud504\ub791\uc2a4\ub294 \ubaa8\ub4e0 \uc2b9\uc778\ub41c SMA \uce58\ub8cc\uc81c\uc5d0 \ub300\ud55c \uc758\ubb34\uc801\uc778 \uc9c8\ubcd1 \uae30\uae08 \ubcf4\uc7a5\uc744 \uc2dc\ud589\ud558\uc5ec \ub192\uc740 \ucc44\ud0dd\ub960\uc744 \uc720\uc9c0\ud569\ub2c8\ub2e4.<br \/>\n*   \uc544\uc2dc\uc544 \ud0dc\ud3c9\uc591: 2031\ub144\uae4c\uc9c0 18.96%\uc758 \uac00\uc7a5 \ube60\ub978 CAGR\uc744 \uae30\ub85d\ud560 \uac83\uc73c\ub85c \uc608\uc0c1\ub429\ub2c8\ub2e4. \uc774\ub294 \uac74\uac15 \ubcf4\ud5d8 \uce68\ud22c\uc728 \uc99d\uac00, \ubd84\uc790 \uc9c4\ub2e8 \uc5ed\ub7c9 \ud655\uc7a5, \uadf8\ub9ac\uace0 \uc218\uc785 \uad00\uc138\ub97c \uba74\uc81c\ud558\ub294 \uc815\ubd80\uc758 \ud76c\uadc0 \uc9c8\ud658 \ubaa9\ub85d \ub355\ubd84\uc785\ub2c8\ub2e4. \uc77c\ubcf8\uc740 2023\ub144\ubd80\ud130 \ubaa8\ub4e0 \uc2e0\uc0dd\uc544\ub97c \uc120\ubcc4\ud558\uace0 \uc138 \uac00\uc9c0 SMN \ud45c\uc801 \uce58\ub8cc\uc81c\ub97c \ubaa8\ub450 \uc9c0\uc6d0\ud558\uba70 1\uc778\ub2f9 \uc9c0\ucd9c\uc744 \uc8fc\ub3c4\ud558\uace0 \uc788\uc2b5\ub2c8\ub2e4. \uc911\uad6d\uc740 2024\ub144 \ub204\uc2dc\ub108\uc13c\uc744 \uc2b9\uc778\ud558\uace0 NRDL(\uad6d\uac00 \uc758\uc57d\ud488 \uc0c1\ud658 \ubaa9\ub85d)\uc5d0 \ud611\uc0c1\ub41c \ud560\uc778 \uac00\uaca9\uc73c\ub85c \ud3ec\ud568\uc2dc\ucf1c \uc9c0\ubc29 \ucc44\ud0dd\uc744 \uac00\uc18d\ud654\ud588\uc2b5\ub2c8\ub2e4.<\/p>\n<p># 6. \uacbd\uc7c1 \ud658\uacbd<\/p>\n<p>\ucc99\uc218\uc131 \uadfc\uc704\ucd95\uc99d \uce58\ub8cc \uc2dc\uc7a5\uc740 \ubc14\uc774\uc624\uc820(Biogen), \ub178\ubc14\ud2f0\uc2a4(Novartis), \ub85c\uc288(Roche)\uac00 \uc804 \uc138\uacc4 \uc218\uc775\uc758 3\ubd84\uc758 2 \uc774\uc0c1\uc744 \ucc28\uc9c0\ud558\ub294 \uc0c1\uc704 \uc9d1\uc911\ud615 \uc2dc\uc7a5\uc785\ub2c8\ub2e4. \uac01 \uae30\uc5c5\uc740 \uc548\ud2f0\uc13c\uc2a4, \uc720\uc804\uc790 \uce58\ub8cc, \uacbd\uad6c \uc2a4\ud50c\ub77c\uc774\uc2f1 \uc870\uc808 \ub4f1 \uace0\uc720\ud55c \uba54\ucee4\ub2c8\uc998 \ud2c8\uc0c8\uc2dc\uc7a5\uc744 \uc810\uc720\ud558\uc5ec \uc9c1\uc811\uc801\uc778 \ub300\uccb4 \uacbd\uc7c1\uc744 \ucd5c\uc18c\ud654\ud558\uace0 \uc788\uc2b5\ub2c8\ub2e4. \ubc29\uc5b4 \uc804\ub7b5\uc5d0\ub294 \uc218\uba85 \uc8fc\uae30 \uad00\ub9ac(\uace0\uc6a9\ub7c9 \ub204\uc2dc\ub108\uc13c, \uc218\ub9c9\uac15\ub0b4 \uc720\uc804\uc790 \ubca1\ud130) \ubc0f \uc0c1\ud658\uc774 \uc544\uc9c1 \uc9c4\ud654 \uc911\uc778 \uc2e0\ud765 \uc9c0\uc5ed\uc73c\ub85c\uc758 \uc9c0\ub9ac\uc801 \ud655\uc7a5\uc774 \ud3ec\ud568\ub429\ub2c8\ub2e4.<\/p>\n<p>\ud30c\uc774\ud504\ub77c\uc778\uc758 \ud601\uc2e0 \uae30\uc5c5\ub4e4\uc740 \uc815\uba74 \uacbd\uc7c1\ubcf4\ub2e4\ub294 \ubcf4\uc644\uc801\uc778 \uc0dd\ubb3c\ud559\uc801 \uacbd\ub85c\ub97c \ud45c\uc801\ud569\ub2c8\ub2e4. \uc2a4\uce7c\ub77c \ub85d(Scholar Rock)\uc758 \uc544\ud53c\ud14c\uadf8\ub85c\ub9d9(\ud56d-\ubbf8\uc624\uc2a4\ud0c0\ud2f4 \ud56d\uccb4)\uc740 SMN \uce58\ub8cc\uc640 \ubcd1\uc6a9 \uc2dc 30.4%\uc758 \ubc18\uc751\ub960\uc744 \ubcf4\uc5ec \ucd5c\ucd08\uc758 \ucd94\uac00 \uadfc\uc721 \ud615\uc131 \uc635\uc158\uc73c\ub85c \uc790\ub9ac\ub9e4\uae40\ud588\uc2b5\ub2c8\ub2e4. \ub85c\uc288\ub294 \ub9ac\uc2a4\ub514\ud50c\ub78c\uacfc RO7204239\ub97c \ubcd1\uc6a9\ud558\ub294 \uc784\uc0c1 2\/3\uc0c1 \uc5f0\uad6c\ub97c \uc2dc\uc791\ud558\uc5ec \uc774\uc911 \uacbd\ub85c \uc774\uc810\uc744 \uc815\ub7c9\ud654\ud558\ub294 \uac83\uc744 \ubaa9\ud45c\ub85c \ud569\ub2c8\ub2e4. \uc81c\uc870 \ud601\uc2e0 \ub610\ud55c \uacbd\uc7c1 \uc6b0\uc704\ub97c \uc7ac\ud3b8\ud558\uace0 \uc788\uc2b5\ub2c8\ub2e4. \ub178\ubc14\ud2f0\uc2a4\ub294 2025\ub144 \ubd80\uc720 HEK293 \uc2dc\uc2a4\ud15c\uc744 \ud1b5\ud569\ud558\uc5ec AAV \ubca1\ud130 \uc218\uc728\uc744 \ub450 \ubc30\ub85c \ub298\ub824 \uc6a9\ub7c9\ub2f9 \ube44\uc6a9\uc744 \uc808\uac10\ud558\uace0 \uacf5\uae09 \uc81c\uc57d\uc744 \uc644\ud654\ud588\uc2b5\ub2c8\ub2e4.<\/p>\n<p>\uc804\ub7b5\uc801 \uc81c\ud734\ub294 \uae00\ub85c\ubc8c \uc785\uc9c0\ub97c \ud655\uc7a5\ud569\ub2c8\ub2e4. \ubc14\uc774\uc624\uc820\uc740 UPS \ud5ec\uc2a4\ucf00\uc5b4\uc640 \ubb3c\ub958 \ud30c\ud2b8\ub108\uc2ed\uc744 \uac31\uc2e0\ud558\uc5ec \ub77c\ud2f4 \uc544\uba54\ub9ac\uce74\ub85c\uc758 \ucf5c\ub4dc \uccb4\uc778 \uc2e0\ub8b0\uc131\uc744 \ub192\uc600\uc73c\uba70, \ub85c\uc288\ub294 \uc911\uad6d CDMO\uc640 \ub77c\uc774\uc120\uc2a4 \uacc4\uc57d\uc744 \uccb4\uacb0\ud558\uc5ec \uc9c0\uc5ed\ubcc4 \ucda9\uc804 \ubc0f \ub9c8\uac10 \uc791\uc5c5\uc744 \ucc98\ub9ac\ud558\uace0 NRDL \uad00\ub828 \uacf5\uae09 \uc77c\uc815\uc744 \uac00\uc18d\ud654\ud588\uc2b5\ub2c8\ub2e4. M&#038;A \uad00\uc2ec\uc740 \ud50c\ub7ab\ud3fc \uac15\ud654\uc5d0 \uc9d1\uc911\ub418\uc5b4 \uc788\uc73c\uba70, \ubbf8\uc624\uc2a4\ud0c0\ud2f4 \ubc0f \uc2e0\uacbd \ubcf4\ud638 \uc800\ubd84\uc790 \ud654\ud569\ubb3c \uc2a4\ud0c0\ud2b8\uc5c5\ub4e4\uc740 \ud22c\uc790 \uc720\uce58 \ud65c\ub3d9\uc774 \uc99d\uac00\ud558\uace0 \uc788\uc2b5\ub2c8\ub2e4. \uc804\ubc18\uc801\uc73c\ub85c \uacbd\uc7c1\uc740 \uac00\uaca9 \ud560\uc778\ubcf4\ub2e4\ub294 \ubcf5\ud569 \uc694\ubc95\uc758 \ub2e4\uc591\uc131, \uacf5\uae09 \ubcf4\uc548, \uc9c0\ubd88\uc790 \uc5f0\uacc4 \ud658\uc790 \uc9c0\uc6d0 \uc778\ud504\ub77c\uc5d0 \uc911\uc810\uc744 \ub450\uc5b4 \ucc99\uc218\uc131 \uadfc\uc704\ucd95\uc99d \uce58\ub8cc \uc2dc\uc7a5 \uc804\ubc18\uc5d0 \uac78\uccd0 \uac74\uc804\ud55c \ub9c8\uc9c4\uc744 \uc720\uc9c0\ud558\uace0 \uc788\uc2b5\ub2c8\ub2e4.<\/p>\n<p>\uc8fc\uc694 \uae30\uc5c5: \ubc14\uc774\uc624\uc820(Biogen), F. \ud638\ud504\ub9cc-\ub77c \ub85c\uc288(F. Hoffmann &#8211; La Roche Ltd), \uc0ac\uc774\ud1a0\ud0a4\ub124\ud2f1\uc2a4(Cytokinetics, Inc.), \ub178\ubc14\ud2f0\uc2a4(Novartis AG), \uce74\ud0c8\ub9ac\uc2a4\ud2b8 \ud30c\ub9c8\uc288\ud2f0\uceec(Catalyst Pharmaceutical)<\/p>\n<p># 7. \ucd5c\uadfc \uc0b0\uc5c5 \ub3d9\ud5a5<\/p>\n<p>*   2025\ub144 3\uc6d4: \uc2a4\uce7c\ub77c \ub85d\uc740 \uc544\ud53c\ud14c\uadf8\ub85c\ub9d9 BLA\uc5d0 \ub300\ud574 FDA \uc6b0\uc120 \uc2ec\uc0ac\ub97c \ubc1b\uc558\uc73c\uba70, PDUFA \ub0a0\uc9dc\ub294 2025\ub144 9\uc6d4 22\uc77c\uc785\ub2c8\ub2e4.<br \/>\n*   2025\ub144 2\uc6d4: \ub85c\uc288\ub294 SMA \ucd5c\ucd08\uc758 \uace0\ud615 \uc81c\ud615\uc778 \uc5d0\ube0c\ub9ac\uc2a4\ub514 5mg \uc815\uc81c\uc5d0 \ub300\ud574 FDA \uc2b9\uc778\uc744 \ud655\ubcf4\ud588\uc2b5\ub2c8\ub2e4.<br \/>\n*   2025\ub144 1\uc6d4: \ub178\ubc14\ud2f0\uc2a4\ub294 \uc218\ub9c9\uac15\ub0b4 \uc624\ub098\uc148\ub178\uc9c4 \uc544\ubca0\ud30c\ub974\ubcf4\ubca1\uc5d0 \ub300\ud55c \uae0d\uc815\uc801\uc778 \uc784\uc0c1 3\uc0c1 STEER \ub370\uc774\ud130\ub97c \ubc1c\ud45c\ud558\uba70 HFMSE \uc810\uc218 2.39\uc810 \ud5a5\uc0c1\uc744 \uae30\ub85d\ud588\uc2b5\ub2c8\ub2e4.<br \/>\n*   2024\ub144 9\uc6d4: \ubc14\uc774\uc624\uc820\uc740 \uace0\uc6a9\ub7c9 \ub204\uc2dc\ub108\uc13c\uc5d0 \ub300\ud55c DEVOTE \uc5f0\uad6c \uacb0\uacfc\ub97c \ubcf4\uace0\ud588\uc73c\uba70, \uc2e0\uacbd\uc12c\uc720 \uac10\uc18c\uc728 94%\ub85c \uc6b0\uc218\ud55c \ud6a8\ub2a5\uc744 \ubcf4\uc600\uc2b5\ub2c8\ub2e4.<\/p>\n<p>\uc774 \ubcf4\uace0\uc11c\ub294 5q \ucc99\uc218\uc131 \uadfc\uc704\ucd95\uc99d(SMA) \uce58\ub8cc \uc2dc\uc7a5\uc5d0 \ub300\ud55c \uc2ec\uce35 \ubd84\uc11d\uc744 \uc81c\uacf5\ud569\ub2c8\ub2e4. SMA\ub294 SMN1 \uc720\uc804\uc790 \ub3cc\uc5f0\ubcc0\uc774\ub85c \uc778\ud574 \ubc1c\uc0dd\ud558\ub294 \uc9c4\ud589\uc131 \uc2e0\uacbd\uadfc\uc721 \uc9c8\ud658\uc73c\ub85c, \uc6b4\ub3d9 \uc2e0\uacbd \ud1f4\ud589\uc744 \uc720\ubc1c\ud558\uba70 \ubc1c\ubcd1 \uc2dc\uae30\uc640 \uc2ec\uac01\ub3c4\uc5d0 \ub530\ub77c \ub2e4\uc591\ud55c \uc591\uc0c1\uc744 \ubcf4\uc785\ub2c8\ub2e4.<\/p>\n<p>\uc2dc\uc7a5 \uaddc\ubaa8 \ubc0f \uc131\uc7a5 \uc804\ub9dd:<br \/>\n\uae00\ub85c\ubc8c SMA \uce58\ub8cc \uc2dc\uc7a5\uc740 2026\ub144 60\uc5b5 6\ucc9c\ub9cc \ub2ec\ub7ec\uc5d0\uc11c 2031\ub144 134\uc5b5 8\ucc9c\ub9cc \ub2ec\ub7ec\ub85c \uc131\uc7a5\ud560 \uac83\uc73c\ub85c \uc608\uc0c1\ub418\uba70, \ud574\ub2f9 \uae30\uac04 \ub3d9\uc548 \uc5f0\ud3c9\uade0 \uc131\uc7a5\ub960(CAGR)\uc740 17.33%\uc5d0 \ub2ec\ud560 \uac83\uc785\ub2c8\ub2e4.<\/p>\n<p>\uc8fc\uc694 \uc2dc\uc7a5 \ub3d9\uc778:<br \/>\n\uc2e0\uc0dd\uc544 \uc120\ubcc4\uac80\uc0ac \uc758\ubb34\ud654\ub85c \uce58\ub8cc \uac00\ub2a5\ud55c \ud658\uc790\uad70\uc774 \ud655\ub300\ub418\uace0 \uc788\uc73c\uba70, \uace0\uc18c\ub4dd \uad6d\uac00\uc5d0\uc11c SMN \ud45c\uc801 \uc720\uc804\uc790 \uce58\ub8cc\uc81c\uc758 \ucc44\ud0dd\uc774 \ube60\ub974\uac8c \uc99d\uac00\ud558\uace0 \uc788\uc2b5\ub2c8\ub2e4. \ub610\ud55c, \uace0\uac00 \ud76c\uadc0\uc9c8\ud658 \uc758\uc57d\ud488\uc5d0 \ub300\ud55c \uae00\ub85c\ubc8c \uc9c0\ubd88\uc790\uc758 \uc218\uc6a9\ub3c4 \uc99d\uac00, \ucd08\uae30 \ub2e8\uacc4 \uc784\uc0c1\uc2dc\ud5d8\uc5d0 \ub300\ud55c \uc639\ud638 \ub2e8\uccb4\uc758 \uc790\uae08 \uc9c0\uc6d0 \ud655\ub300, SMN \ubc0f \ubbf8\uc624\uc2a4\ud0c0\ud2f4\uc744 \ud45c\uc801\uc73c\ub85c \ud558\ub294 \ud30c\uc774\ud504\ub77c\uc778 \ubcf5\ud569 \uc694\ubc95 \uac1c\ubc1c, \uadf8\ub9ac\uace0 \ub9cc\uc131 \ud22c\uc5ec\ub97c \ucd09\uc9c4\ud558\ub294 \uc7ac\ud0dd \ub514\uc9c0\ud138 \ubcf5\uc57d \uc21c\uc751\ub3c4 \ud50c\ub7ab\ud3fc\uc758 \ub4f1\uc7a5\uc774 \uc2dc\uc7a5 \uc131\uc7a5\uc744 \uacac\uc778\ud558\uace0 \uc788\uc2b5\ub2c8\ub2e4.<\/p>\n<p>\uc8fc\uc694 \uc2dc\uc7a5 \uc81c\uc57d \uc694\uc778:<br \/>\n\uc218\ubc31\ub9cc \ub2ec\ub7ec\uc5d0 \ub2ec\ud558\ub294 \uce58\ub8cc \ube44\uc6a9\uacfc \ubd88\ud655\uc2e4\ud55c \uc7a5\uae30 \uc0c1\ud658 \uc815\ucc45\uc740 \uc2dc\uc7a5\uc758 \uc8fc\uc694 \uc81c\uc57d \uc694\uc778\uc785\ub2c8\ub2e4. \uc774\uc640 \ub354\ubd88\uc5b4, \uc218\ub9c9\uac15\ub0b4(intrathecal) \ud22c\uc5ec\uc758 \ubcf5\uc7a1\uc131\uacfc \uc8fc\uc785 \uc13c\ud130\uc758 \uc6a9\ub7c9 \uc81c\uc57d, \uc2e0\ud765 \uacbd\uc81c\uad6d\uc758 \uc18c\uc544 \uc2e0\uacbd\uadfc\uc721 \uc804\ubb38\uc758 \ubd80\uc871, \uadf8\ub9ac\uace0 AAV(\uc544\ub370\ub178 \uad00\ub828 \ubc14\uc774\ub7ec\uc2a4) \uacf5\uae09\uc744 \uc704\ud55c \ubc14\uc774\ub7ec\uc2a4 \ubca1\ud130 \uc81c\uc870 \ubcd1\ubaa9 \ud604\uc0c1\ub3c4 \uc2dc\uc7a5 \uc131\uc7a5\uc744 \uc800\ud574\ud558\ub294 \uc694\uc18c\ub85c \uc791\uc6a9\ud569\ub2c8\ub2e4.<\/p>\n<p>\uc2dc\uc7a5 \uc138\ubd84\ud654:<br \/>\n\ubcf8 \ubcf4\uace0\uc11c\ub294 \uc2dc\uc7a5\uc744 \uc9c8\ubcd1 \uc720\ud615(0\ud615, 1\ud615, 2\ud615, 3\ud615, 4\ud615), \uce58\ub8cc \ubc29\uc2dd(\uc720\uc804\uc790 \ub300\uccb4 \uc694\ubc95, SMN2 \uc2a4\ud50c\ub77c\uc774\uc2f1 \uc870\uc808\uc81c, \uc548\ud2f0\uc13c\uc2a4 \uc62c\ub9ac\uace0\ub274\ud074\ub808\uc624\ud0c0\uc774\ub4dc \uc694\ubc95, \uadfc\uc721 \ud45c\uc801\/\ubbf8\uc624\uc2a4\ud0c0\ud2f4 \uc5b5\uc81c\uc81c, \ubcf4\uc870 \ubc0f \uc9c0\uc9c0 \uc694\ubc95), \ud22c\uc5ec \uacbd\ub85c(\uacbd\uad6c, \uc218\ub9c9\uac15\ub0b4, \uc815\ub9e5), \uc5f0\ub839 \uadf8\ub8f9(\uc2e0\uc0dd\uc544, \uc18c\uc544, \uccad\uc18c\ub144, \uc131\uc778), \uc720\ud1b5 \ucc44\ub110(\ubcd1\uc6d0 \uc57d\uad6d, \uc804\ubb38 \ud074\ub9ac\ub2c9, \uc628\ub77c\uc778 \ubc0f \uc18c\ub9e4 \uc57d\uad6d) \ubc0f \uc9c0\uc5ed(\ubd81\ubbf8, \uc720\ub7fd, \uc544\uc2dc\uc544-\ud0dc\ud3c9\uc591, \uc911\ub3d9 \ubc0f \uc544\ud504\ub9ac\uce74, \ub0a8\ubbf8)\ubcc4\ub85c \uc0c1\uc138\ud558\uac8c \ubd84\uc11d\ud569\ub2c8\ub2e4.<\/p>\n<p>\uc9c0\uc5ed\ubcc4 \ud1b5\ucc30:<br \/>\n\uc544\uc2dc\uc544-\ud0dc\ud3c9\uc591 \uc9c0\uc5ed\uc740 \uac74\uac15 \ubcf4\ud5d8 \uc801\uc6a9 \ubc94\uc704 \ud655\ub300, \ubd84\uc790 \uc9c4\ub2e8 \uc5ed\ub7c9 \uc99d\ub300, \ud76c\uadc0 \uc9c8\ud658 \uc758\uc57d\ud488\uc5d0 \ub300\ud55c \uc815\ubd80 \uc778\uc13c\ud2f0\ube0c\uc5d0 \ud798\uc785\uc5b4 2031\ub144\uae4c\uc9c0 18.96%\uc758 \uac00\uc7a5 \ube60\ub978 \uc5f0\ud3c9\uade0 \uc131\uc7a5\ub960\uc744 \ubcf4\uc77c \uac83\uc73c\ub85c \uc608\uc0c1\ub429\ub2c8\ub2e4.<\/p>\n<p>\uce58\ub8cc \ubc29\uc2dd\ubcc4 \ud1b5\ucc30:<br \/>\nSMN2 \uc2a4\ud50c\ub77c\uc774\uc2f1 \uc870\uc808\uc81c\ub294 \ud655\ub9bd\ub41c \uc548\uc804\uc131 \ud504\ub85c\ud30c\uc77c\uacfc \ubaa8\ub4e0 SMA \uc720\ud615\uc5d0 \uc801\uc6a9 \uac00\ub2a5\ud558\ub2e4\ub294 \uc7a5\uc810\uc73c\ub85c 2025\ub144 \uc2dc\uc7a5 \uc810\uc720\uc728 42.35%\ub97c \ucc28\uc9c0\ud558\uba70 \uc120\ub450\ub97c \uc720\uc9c0\ud588\uc2b5\ub2c8\ub2e4.<\/p>\n<p>\uc8fc\uc694 \uacbd\uc7c1\uc0ac:<br \/>\n\uc2dc\uc7a5\uc758 \uc8fc\uc694 \ucc38\uc5ec \uae30\uc5c5\uc73c\ub85c\ub294 Biogen Inc., Roche Holding AG (Genentech), Novartis International AG, Ionis Pharmaceuticals Inc., PTC Therapeutics Inc., Scholar Rock Inc., Cytokinetics Inc., Sarepta Therapeutics Inc., NMD Pharma A\/S, Astellas Pharma Inc., Pfizer Inc., Chugai Pharmaceutical Co., Ltd., Sanofi S.A. (Genzyme), Catalyst Pharmaceuticals \ub4f1\uc774 \uc788\uc2b5\ub2c8\ub2e4.<\/p>\n<p>\uacb0\ub860:<br \/>\n\ubcf8 \ubcf4\uace0\uc11c\ub294 SMA \uce58\ub8cc \uc2dc\uc7a5\uc758 \ud604\uc7ac \uc0c1\ud669, \ubbf8\ub798 \uc131\uc7a5 \uc804\ub9dd, \uc8fc\uc694 \ub3d9\uc778 \ubc0f \uc81c\uc57d \uc694\uc778, \uadf8\ub9ac\uace0 \uacbd\uc7c1 \ud658\uacbd\uc5d0 \ub300\ud55c \ud3ec\uad04\uc801\uc778 \uc774\ud574\ub97c \uc81c\uacf5\ud558\uba70, \ubbf8\ucda9\uc871 \uc218\uc694 \ubc0f \ubbf8\ub798 \uae30\ud68c\uc5d0 \ub300\ud55c \ud3c9\uac00\ub3c4 \ud3ec\ud568\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-54111","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\/54111"}],"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=54111"}],"version-history":[{"count":0,"href":"https:\/\/www.globalresearch.co.kr\/insight\/wp-json\/wp\/v2\/posts\/54111\/revisions"}],"wp:attachment":[{"href":"https:\/\/www.globalresearch.co.kr\/insight\/wp-json\/wp\/v2\/media?parent=54111"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.globalresearch.co.kr\/insight\/wp-json\/wp\/v2\/categories?post=54111"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.globalresearch.co.kr\/insight\/wp-json\/wp\/v2\/tags?post=54111"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}