{"id":27286,"date":"2025-06-09T15:53:56","date_gmt":"2025-06-09T08:53:56","guid":{"rendered":"https:\/\/quanghung.name.vn\/suc-khoe\/viet-nam-thu-nghiem-thuoc-dieu-tri-teo-co-tuy-50-ti-dong-lieu\/"},"modified":"2025-06-09T15:53:56","modified_gmt":"2025-06-09T08:53:56","slug":"viet-nam-thu-nghiem-thuoc-dieu-tri-teo-co-tuy-50-ti-dong-lieu","status":"publish","type":"post","link":"https:\/\/quanghung.name.vn\/?p=27286","title":{"rendered":"Vi\u1ec7t Nam th\u1eed nghi\u1ec7m thu\u1ed1c \u0111i\u1ec1u tr\u1ecb teo c\u01a1 t\u1ee7y 50 t\u1ec9 \u0111\u1ed3ng\/li\u1ec1u"},"content":{"rendered":"\n<div class=\"detail-content afcbc-body\" data-role=\"content\" itemprop=\"articleBody\" data-io-article-url=\"https:\/\/thanhnien.vn\/viet-nam-thu-nghiem-thuoc-dieu-tri-teo-co-tuy-50-ti-dong-lieu-185250606170232942.htm\">\n<div data-check-position=\"body_start\"><\/div>\n<p>Th\u00f4ng tin tr\u00ean \u0111\u01b0\u1ee3c PGS-TS, b\u00e1c s\u0129 V\u0169 Ch\u00ed D\u0169ng, Gi\u00e1m \u0111\u1ed1c Trung t\u00e2m N\u1ed9i ti\u1ebft, chuy\u1ec3n h\u00f3a, di truy\u1ec1n v\u00e0 li\u1ec7u ph\u00e1p ph\u00e2n t\u1eed, B\u1ec7nh vi\u1ec7n Nhi T.\u01af, cho bi\u1ebft t\u1ea1i di\u1ec5n \u0111\u00e0n \u0110\u1ed5i m\u1edbi s\u00e1ng t\u1ea1o\u00a0chuy\u1ec3n \u0111\u1ed5i s\u1ed1 trong y t\u1ebf, do Pharma Group ph\u1ed1i h\u1ee3p v\u1edbi FPT t\u1ed5 ch\u1ee9c ng\u00e0y 6.6 t\u1ea1i H\u00e0 N\u1ed9i.<\/p>\n<figure class=\"VCSortableInPreviewMode\" type=\"Photo\" style=\"\">\n<div><img decoding=\"async\" loading=\"lazy\" data-author=\"\" src=\"https:\/\/images2.thanhnien.vn\/thumb_w\/640\/528068263637045248\/2025\/6\/6\/benh-hiem-17492030568291483137505.jpg\" id=\"img_160792539354636288\" w=\"664\" h=\"447\" alt=\"Tr\u1ebb m\u1eafc b\u1ec7nh hi\u1ebfm t\u1ea1i Vi\u1ec7t Nam th\u1eed nghi\u1ec7m d\u00f9ng thu\u1ed1c 51 t\u1ef7 \u0111\u1ed3ng\/ca b\u1ec7nh - \u1ea2nh 1.\" title=\"\" rel=\"lightbox\" photoid=\"160792539354636288\" data-original=\"https:\/\/images2.thanhnien.vn\/528068263637045248\/2025\/6\/6\/benh-hiem-17492030568291483137505.jpg\" type=\"photo\" style=\"max-width:100%;\" width=\"664\" height=\"447\"><\/div><figcaption class=\"PhotoCMS_Caption\">\n<p data-placeholder=\"Nh\u1eadp ch\u00fa th\u00edch \u1ea3nh\">Thu\u1ed1c Zolgensma c\u00f3 gi\u00e1 kho\u1ea3ng h\u01a1n 50 t\u1ec9 \u0111\u1ed3ng\/l\u1ea7n d\u00f9ng duy nh\u1ea5t \u0111\u01b0\u1ee3c h\u1ed7 tr\u1ee3 mi\u1ec5n ph\u00ed m\u1ed9t s\u1ed1 tr\u1ebb b\u1ecb teo c\u01a1 t\u1ee7y<\/p>\n<\/figcaption><div class=\"PhotoCMS_Author\">\n<p data-placeholder=\"Nh\u1eadp t\u00e1c gi\u1ea3\" class=\"\">\u1ea2NH: B\u1ec6NH VI\u1ec6N NHI T.\u01af<\/p>\n<\/div>\n<\/figure>\n<p>C\u00e1c tr\u1ebb tham gia th\u1eed nghi\u1ec7m v\u00e0o n\u0103m 2022. B\u1ec7nh vi\u1ec7n Nhi T.\u01af l\u00e0 \u0111\u01a1n v\u1ecb tham gia th\u1eed nghi\u1ec7m l\u00e2m s\u00e0ng thu\u1ed1c n\u00e0y. Vi\u1ec7t Nam l\u00e0 m\u1ed9t trong 15 n\u01b0\u1edbc tham gia, nghi\u00ean c\u1ee9u \u0111\u00e3 ho\u00e0n t\u1ea5t v\u00e0o ng\u00e0y 30.5 v\u1eeba qua.<\/p>\n<p>Thu\u1ed1c th\u1eed nghi\u1ec7m l\u00e0 Zolgensma, \u0111i\u1ec1u tr\u1ecb b\u1ec7nh di truy\u1ec1n teo c\u01a1 t\u1ee7y, c\u00f3 gi\u00e1 kho\u1ea3ng h\u01a1n 50 t\u1ec9 \u0111\u1ed3ng. Thu\u1ed1c d\u00f9ng m\u1ed9t l\u1ea7n trong \u0111\u1eddi, c\u01a1 ch\u1ebf ho\u1ea1t \u0111\u1ed9ng l\u00e0 thay th\u1ebf gene b\u1ecb b\u1ec7nh, \u0111\u01b0a gen b\u00ecnh th\u01b0\u1eddng v\u00e0o trong th\u1ea7n kinh t\u1ee7y.<\/p>\n<p>V\u1edbi c\u00e1c tr\u1ebb \u0111\u00e3 tham gia th\u1eed nghi\u1ec7m thu\u1ed1c, sau khi k\u1ebft th\u00fac d\u00f9ng thu\u1ed1c \u0111\u01b0\u1ee3c chuy\u1ec3n sang giai \u0111o\u1ea1n theo d\u00f5i 5 n\u0103m ti\u1ebfp theo \u0111\u1ec3 ti\u1ebfp t\u1ee5c \u0111\u00e1nh gi\u00e1 hi\u1ec7u qu\u1ea3 c\u1ee7a thu\u1ed1c.<\/p>\n<p>Teo c\u01a1 t\u1ee7y l\u00e0 b\u1ec7nh di truy\u1ec1n g\u00e2y tho\u00e1i h\u00f3a v\u1eadn \u0111\u1ed9ng v\u00e0 t\u1eed vong s\u1edbm do li\u1ec7t c\u01a1 h\u00f4 h\u1ea5p \u1edf tr\u1ebb nh\u1ecf.<\/p>\n<h2>Th\u1eed nghi\u1ec7m l\u00e2m s\u00e0ng th\u00eam c\u01a1 h\u1ed9i ti\u1ebfp c\u1eadn thu\u1ed1c m\u1edbi\u00a0<\/h2>\n<p>B\u00e1c s\u0129 D\u0169ng cho hay, th\u1eed nghi\u1ec7m l\u00e2m s\u00e0ng thu\u1ed1c n\u00eau tr\u00ean d\u00e0nh cho nh\u1eefng tr\u1ebb tr\u00ean 2 tu\u1ed5i. Thu\u1ed1c do T\u1eadp \u0111o\u00e0n d\u01b0\u1ee3c ph\u1ea9m Novartis (Th\u1ee5y S\u0129) nghi\u00ean c\u1ee9u ph\u00e1t tri\u1ec3n, c\u00f3 gi\u00e1 2,1 tri\u1ec7u USD (kho\u1ea3ng h\u01a1n 50 t\u1ec9 \u0111\u1ed3ng) m\u1ed9t li\u1ec1u, \u0111\u1eaft nh\u1ea5t th\u1ebf gi\u1edbi v\u00e0o th\u1eddi \u0111i\u1ec3m ra \u0111\u1eddi. Th\u1ef1c t\u1ebf, thu\u1ed1c Zolgensma \u0111\u00e3 \u0111\u01b0\u1ee3c FDA M\u1ef9 ph\u00ea chu\u1ea9n t\u1eeb th\u00e1ng 5.2020 d\u00e0nh cho nh\u00f3m tr\u1ebb d\u01b0\u1edbi 2 tu\u1ed5i.<\/p>\n<p>N\u0103m 2021, B\u1ec7nh vi\u1ec7n Nhi T.\u01af \u0111\u00e3 k\u00fd bi\u00ean b\u1ea3n ghi nh\u1edb v\u1edbi C\u00f4ng ty TNHH Novartis Vi\u1ec7t Nam v\u1ec1 \u201cT\u0103ng c\u01b0\u1eddng ch\u0103m s\u00f3c cho b\u1ec7nh nh\u00e2n teo c\u01a1 t\u1ee7y v\u00e0 qu\u1ea3n l\u00fd b\u1ec7nh hi\u1ebfm t\u1ea1i Vi\u1ec7t Nam\u201d trong \u0111\u00f3 h\u1ed7 tr\u1ee3 thu\u1ed1c Zolgensma mi\u1ec5n ph\u00ed cho 10 b\u1ec7nh nh\u00e2n teo c\u01a1 t\u1ee7y d\u01b0\u1edbi 2 tu\u1ed5i.\u00a0<\/p>\n<p>T\u1ea1i Vi\u1ec7t Nam, c\u00f3 46 tr\u1ebb d\u01b0\u1edbi 2 tu\u1ed5i t\u1ea1i B\u1ec7nh vi\u1ec7n Nhi T.\u01af v\u00e0 B\u1ec7nh vi\u1ec7n Nhi \u0111\u1ed3ng (TP.HCM) \u0111\u00e3 \u0111\u01b0\u1ee3c s\u1eed d\u1ee5ng thu\u1ed1c n\u00e0y t\u1eeb vi\u1ec7n tr\u1ee3 kh\u00f4ng ho\u00e0n l\u1ea1i.<\/p>\n<p>T\u1ea1i di\u1ec5n \u0111\u00e0n \u0110\u1ed5i m\u1edbi s\u00e1ng t\u1ea1o trong y t\u1ebf, c\u00e1c chuy\u00ean gia c\u0169ng nh\u1ea5n m\u1ea1nh vai tr\u00f2 c\u1ee7a AI trong tri\u1ec3n khai c\u00e1c th\u1eed nghi\u1ec7m l\u00e2m s\u00e0ng. \u0110\u1eb7c bi\u1ec7t, c\u00e1c \u0111\u01a1n v\u1ecb c\u00f4ng ngh\u1ec7 trong n\u01b0\u1edbc \u0111ang x\u00e2y d\u1ef1ng AI lab, d\u00e0nh cho c\u00e1c nghi\u00ean c\u1ee9u th\u1eed nghi\u1ec7m l\u00e2m s\u00e0ng, gi\u00fap r\u00fat ng\u1eafn th\u1eddi gian nghi\u00ean c\u1ee9u, t\u0103ng c\u01a1 h\u1ed9i ti\u1ebfp c\u1eadn thu\u1ed1c m\u1edbi trong \u0111i\u1ec1u tr\u1ecb.<\/p>\n<p><\/p>\n<p><\/p>\n<p><\/p>\n<p><\/p>\n<p><\/p>\n<p><\/p>\n<p><\/p>\n<p><\/p>\n<p><\/p>\n<p><!--Fri Jun 06 2025 18:10:43 GMT+0700 (Indochina Time) --  -- Fri Jun 06 2025 18:10:43 GMT+0700 (Indochina Time)--><br \/>\n                                        <script>\n    \/\/Ch\u00e8n ads gi\u1eefa b\u00e0i\n    (runinit = window.runinit || 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t\u1ebf, do Pharma Group ph\u1ed1i h\u1ee3p v\u1edbi FPT t\u1ed5 ch\u1ee9c ng\u00e0y 6.6 t\u1ea1i<\/p>\n","protected":false},"author":1,"featured_media":24681,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"googlesitekit_rrm_CAow44a6DA:productID":"","footnotes":""},"categories":[6],"tags":[7468,7467,7470,7469],"class_list":["post-27286","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-suc-khoe","tag-di-truyen","tag-pharma-group","tag-thu-nghiem-lam-sang","tag-thuoc-zolgensma"],"_links":{"self":[{"href":"https:\/\/quanghung.name.vn\/index.php?rest_route=\/wp\/v2\/posts\/27286","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/quanghung.name.vn\/index.php?rest_route=\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/quanghung.name.vn\/index.php?rest_route=\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/quanghung.name.vn\/index.php?rest_route=\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/quanghung.name.vn\/index.php?rest_route=%2Fwp%2Fv2%2Fcomments&post=27286"}],"version-history":[{"count":0,"href":"https:\/\/quanghung.name.vn\/index.php?rest_route=\/wp\/v2\/posts\/27286\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/quanghung.name.vn\/index.php?rest_route=\/wp\/v2\/media\/24681"}],"wp:attachment":[{"href":"https:\/\/quanghung.name.vn\/index.php?rest_route=%2Fwp%2Fv2%2Fmedia&parent=27286"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/quanghung.name.vn\/index.php?rest_route=%2Fwp%2Fv2%2Fcategories&post=27286"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/quanghung.name.vn\/index.php?rest_route=%2Fwp%2Fv2%2Ftags&post=27286"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}