{"id":255434,"date":"2023-01-23T13:47:13","date_gmt":"2023-01-23T12:47:13","guid":{"rendered":"https:\/\/www.fqeuskadi.org\/?p=255434"},"modified":"2023-01-23T13:52:22","modified_gmt":"2023-01-23T12:52:22","slug":"kb407-ak-sendagai-orphan-en-estatusa-lortu-du-europan","status":"publish","type":"post","link":"https:\/\/www.fqeuskadi.org\/eu\/kb407-ak-sendagai-orphan-en-estatusa-lortu-du-europan\/","title":{"rendered":"KB407-ak, \u201corphan medikamentu\u201d-aren estatusa lortu du Europan"},"content":{"rendered":"<p>[et_pb_section fb_built=&#8221;1&#8243; admin_label=&#8221;section&#8221; _builder_version=&#8221;4.16&#8243; global_colors_info=&#8221;{}&#8221;][et_pb_row admin_label=&#8221;row&#8221; _builder_version=&#8221;4.16&#8243; background_size=&#8221;initial&#8221; background_position=&#8221;top_left&#8221; background_repeat=&#8221;repeat&#8221; global_colors_info=&#8221;{}&#8221;][et_pb_column type=&#8221;4_4&#8243; _builder_version=&#8221;4.16&#8243; custom_padding=&#8221;|||&#8221; global_colors_info=&#8221;{}&#8221; custom_padding__hover=&#8221;|||&#8221;][et_pb_text _builder_version=&#8221;4.19.4&#8243; _module_preset=&#8221;default&#8221; global_colors_info=&#8221;{}&#8221;]<\/p>\n<h3 class=\"p1\">Terapia potentzial honek badu izendapen hori AEBetan.<\/h3>\n<p class=\"p1\">Europako Batzordeak (EB) \u201cOrphan\u201d medikamentu izendatu du <b>KB407<\/b>, Fibrosi Kistikoaren aurkako Krystal Biotech-en<b> terapia potentzial inhalatuari.<\/b><\/p>\n<p class=\"p1\">\u201cOrphan&#8221; medikamentu estatusaren helburua<b> gaixotasun arraroentzako terapien garapena sustatzea <\/b>da. Onespenaren ondoren merkatuan 10 urteko esklusibotasuna bezalako onurak ekartzen ditu.<\/p>\n<p class=\"p1\">Fibrosi Kistikoa CFTR genean gertatzen diren <b>mutazioek<\/b> eragiten dute. Mutazio honek CFTR proteina forma okerrean eratzea edo proteina honen erabateko falta dakar. CFTR-ak uraren eta <b>kloruro-ioien fluxua kontrolatzen du<\/b> zelulen barruan eta kanpoan, baina CFTR genaren mutazioak gehiegizko <b>muki lodia<\/b>ren ekoizpena eragiten du.<\/p>\n<blockquote>\n<p class=\"p1\">KB407a dosi errepikatuko FKaren <b>terapia genikoa da<\/b>. CFTRren bi kopia osasuntsu arnasbideetako zelulei helarazteko, herpes sinplearen birusaren (HSV-1) bertsio aldatua erabiltzen du. Terapiaren helburua,<b> ioien eta uraren fluxua<\/b> biriketako zeluletan eta hauetatik kanpo <b>berezartzea<\/b> da, pazienteek duten CFTR mutazio mota edozein dela ere (2000 mutazio baino gehiago deskribatu dira).<\/p>\n<\/blockquote>\n<p class=\"p1\">Animalietan egindako ikerketetan, KB407 birikei <b>inhalazio bidez<\/b> helarazi zitzaien, nebulizatzaile baten laguntzarekin.<span class=\"Apple-converted-space\">\u00a0 <\/span>Honek ere, biriketako zeluletan <b>CFTR proteina normalaren ekoizpena<\/b> sustatu zuen, baita pazienteetatik eratorritako organoideetan ere: 3D miniorganoak, ehun gaixoa hobeto imitatzen dutenak.<\/p>\n<p><a href=\"https:\/\/cysticfibrosisnewstoday.com\/news\/potential-cf-gene-therapy-kb407-orphan-drug-europe\/\">Jatorrizko albistea<\/a><\/p>\n<p>Beste albisteak: <a href=\"https:\/\/www.fqeuskadi.org\/eu\/pseudomonasei-aurre-egiteko-inhalatutako-antibiotikoak-segurtasuna-erakusten-du-aurretiko-saiakuntzan\/\">Pseudomonasei aurre egiteko inhalatutako antibiotikoak segurtasuna erakusten du aurretiko saiakuntzan<\/a><\/p>\n<p>[\/et_pb_text][\/et_pb_column][\/et_pb_row][\/et_pb_section]<\/p>\n","protected":false},"excerpt":{"rendered":"<p>Terapia potentzial honek badu izendapen hori AEBetan. Europako Batzordeak (EB) \u201cOrphan\u201d medikamentu izendatu du KB407, Fibrosi Kistikoaren aurkako Krystal Biotech-en terapia potentzial inhalatuari. \u201cOrphan&#8221; medikamentu estatusaren helburua gaixotasun arraroentzako terapien garapena sustatzea da. Onespenaren ondoren merkatuan 10 urteko esklusibotasuna bezalako onurak ekartzen ditu. Fibrosi Kistikoa CFTR genean gertatzen diren mutazioek eragiten dute. Mutazio honek CFTR [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":255432,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_et_pb_use_builder":"on","_et_pb_old_content":"","_et_gb_content_width":"","footnotes":""},"categories":[47],"tags":[],"class_list":["post-255434","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-fq-berriak"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v24.9 - https:\/\/yoast.com\/wordpress\/plugins\/seo\/ -->\n<title>KB407-ak, \u201corphan medikamentu\u201d-aren estatusa lortu du Europan<\/title>\n<meta name=\"description\" content=\"\u201cOrphan sendagai\u201d estatusaren helburua onuera ezberdinen bidez gaixotasun arraroentzako terapien garapena sustatzea da.\" \/>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/www.fqeuskadi.org\/eu\/kb407-ak-sendagai-orphan-en-estatusa-lortu-du-europan\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"KB407-ak, \u201corphan medikamentu\u201d-aren estatusa lortu du Europan\" \/>\n<meta property=\"og:description\" content=\"\u201cOrphan sendagai\u201d estatusaren helburua onuera ezberdinen bidez gaixotasun arraroentzako terapien garapena sustatzea da.\" \/>\n<meta property=\"og:url\" content=\"https:\/\/www.fqeuskadi.org\/eu\/kb407-ak-sendagai-orphan-en-estatusa-lortu-du-europan\/\" \/>\n<meta property=\"og:site_name\" content=\"FQ EUSKADI - 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