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De farnesyltransferaseremmer (FTI) lonafarnib (onder de merknaam Zokinvy) is de eerste en enige bekende medicamenteuze behandeling voor kinderen met Progeria.<\/p>\n
Geschiedenis achter deze historische ontdekking: In augustus 2005 en februari 2006 publiceerden onderzoekers studies die een mogelijke medicijnbehandeling voor kinderen met Progeria ondersteunden. Oorspronkelijk ontwikkeld om kanker mogelijk te behandelen, keerde lonafarnib de dramatische afwijkingen in de nucleaire structuur terug die kenmerkend zijn voor cellen van kinderen met Progeria. Bovendien verbeterde dit FTI-medicijn enkele tekenen van ziekte in een Progeria-achtig muismodel.<\/p>\n
Waarom dachten onderzoekers dat dit medicijn zou werken bij Progeria? Het eiwit waarvan wij denken dat het verantwoordelijk is voor Progeria, heet progerine. Om de normale celfunctie te blokkeren en Progeria te veroorzaken, moet een molecuul genaamd een "farnesylgroep" worden gehecht aan het progerine-eiwit. FTI's werken door de hechting van de farnesylgroep aan progerine te blokkeren (remmen). Dus als het FTI-medicijn deze farnesylgroephechting bij kinderen met Progeria kan blokkeren, dan kan progerine "verlamd" worden en Progeria verbeterd.<\/p>\n
Voor het eerst hadden we een mogelijke behandeling voor kinderen met Progeria voor ons. allereerste klinische proef met Progeria-medicijnen<\/a> begon in 2007 en in 2012 werden de onderzoeksresultaten gepubliceerd, waaruit bleek dat elk kind verbetering ervoer op een of meer gebieden, waaronder het vitale cardiovasculaire systeem. In mei 2014 onthulde verder onderzoek dat lonafarnib de geschatte levensduur met ten minste 1,6 jaar verlengt (wat later zou worden verhoogd, naarmate het onderzoek verder vorderde), en in april 2018 werd een onderzoek gepubliceerd in Het tijdschrift van de American Medical Association (JAMA)<\/em> meldden dat lonafarnib alleen de overleving van kinderen met Progeria verlengde. \u00a0Klik hier<\/a> voor details over het onderzoek uit 2012,\u00a0hier <\/a>voor details over de bevindingen van 2014, en hier<\/a> voor meer informatie over het onderzoek uit 2018.<\/p>\n In mei 2018, kort na de studie die in JAMA<\/em>PRF en Eiger Biopharmaceuticals zijn een samenwerkings- en leveringsovereenkomst aangegaan voor de ontwikkeling en het nastreven van beoordeling door de Amerikaanse Food and Drug Administration en mogelijke goedkeuring van lonafarnib voor de behandeling van Progeria bij kinderen. De indiening werd voltooid op 23 maart 2020. <\/a>In november 2020 schreef lonafarnib geschiedenis als de allereerste behandeling die goedkeuring krijgt van de Amerikaanse Food and Drug Administration (FDA)<\/a> voor Progeria en Progero\u00efde Laminopathie\u00ebn.<\/p>\n We hebben nu \u00e9\u00e9n behandeling voor Progeria, maar het is geen genezing. Daarom blijven we onderzoek doen naar medicijnen die nog effectiever zijn en uiteindelijk Progeria genezen.<\/p>\n [\/et_pb_text][\/et_pb_column_inner][\/et_pb_row_inner][\/et_pb_column][\/et_pb_section][et_pb_section fb_built=\u201d1\u2033 module_class=\u201dfooter\u201d _builder_version=\u201d4.3.4\u2033 background_color=\u201d#29327a\u201d custom_margin=\u201d25px|||||\u201d custom_padding=\u201d0|0px|0|0px|false|false\u201d z_index_tablet=\u201d500\u2033 border_width_top=\u201d12px\u201d border_color_top=\u201d#00b2e2\u2033 global_module=\u201d133\u2033 locked=\u201duit\u201d global_colors_info=\u201d{}\u201d][et_pb_row column_structure=\u201d1_4,1_4,1_2\u2033 make_equal=\u201daan\u201d module_class=\u201det_pb_row_fullwidth\u201d _builder_version=\u201d4.16\u2033 width=\u201d89%\u201d width_tablet=\u201d80%\u201d width_phone=\u201d\u201d width_last_edited=\u201daan|desktop\u201d max_width=\u201d89%\u201d max_width_tablet=\u201d80%\u201d max_width_phone=\u201d\u201d 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global_colors_info=\u201d{}\u201d][\/et_pb_image][\/et_pb_column][\/et_pb_row][\/et_pb_section]<\/p>","protected":false},"excerpt":{"rendered":" [et_pb_section fb_built=\u201d1\u2033 fullwidth=\u201don\u201d disabled_on=\u201duit|uit|uit\u201d _builder_version=\u201d4.16\u2033 border_width_bottom=\u201d55px\u201d border_color_bottom=\u201d#29327a\u201d global_colors_info=\u201d{}\u201d][et_pb_fullwidth_header _builder_version=\u201d4.16\u2033 title_font_size=\u201d55\u2033 background_color=\u201d#29327a\u201d background_image=\u201dhttps:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2019\/04\/About-Header.jpg\u201d background_position=\u201dcenter_left\u201d custom_padding=\u201d9vw||9vw||true\u201d custom_padding_tablet=\u201d\u201d custom_padding_phone=\u201d|56px||\u201d custom_padding_last_edited=\u201dop|bureaublad\u201d title_font_size_tablet=\u201d45px\u201d title_font_size_phone=\u201d40px\u201d title_font_size_last_edited=\u201dop|telefoon\u201d z_index_tablet=\u201d500\u2033 custom_css_main_element=\u201dachtergrondpositie: midden 18% !belangrijk;\u201d global_colors_info=\u201d{}\u201d] Het FTI-medicijn [\/et_pb_fullwidth_header][\/et_pb_section][et_pb_section fb_built=\u201d1\u2033 use_custom_gutter=\u201don\u201d gutter_width=\u201d1\u2033 specialty=\u201don\u201d padding_left_1=\u201d35px\u201d padding_left_2=\u201d35px\u201d padding_2_tablet=\u201d|||0px\u201d padding_2_phone=\u201d|||0px\u201d padding_2_last_edited=\u201don|desktop\u201d module_class_1=\u201dsidebar-secondary-nav\u201d module_class=\u201dhandprint-bg\u201d _builder_version=\u201d4.16\u2033 background_image=\u201dhttps:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2019\/04\/blue-handprint-only.png\u201d parallax=\u201daan\u201d parallax_method=\u201duit\u201d inner_width=\u201d100%\u201d inner_max_width=\u201d100%\u201d custom_padding=\u201d0|0px|54px|0px|false|false\u201d z_index_tablet=\u201d500\u2033 border_width_top=\u201d10px\u201d border_color_top=\u201d#8fd2ed\u201d use_custom_width=\u201daan\u201d width_unit=\u201duit\u201d custom_width_percent=\u201d100%\u201d global_colors_info=\u201d{}\u201d][et_pb_column [\u2026]<\/p>","protected":false},"author":1,"featured_media":0,"parent":0,"menu_order":0,"comment_status":"closed","ping_status":"closed","template":"","meta":{"_et_pb_use_builder":"on","_et_pb_old_content":"\t\t\t\t[vc_row][vc_column][vc_custom_heading text=\"The FTI Drug Lonafarnib\" font_container=\"tag:h1|text_align:center\" use_theme_fonts=\"yes\"][vc_custom_heading text=\"The FTI lonafarnib \u2013 the first and only known drug treatment for children with Progeria\" use_theme_fonts=\"yes\"][vc_column_text]In August 2005 and February 2006, researchers published studies that supported a potential drug treatment for children with Progeria. Farnesyltransferase inhibitors (FTIs)<\/b>, originally developed to potentially treat cancer, reversed the dramatic nuclear structure abnormalities that are the hallmark of cells from children with Progeria. In addition, this FTI drug improves some signs of disease in a Progeria-like mouse model.\r\n\r\nWhy did researchers think this drug would work in Progeria?<\/b> The protein that we believe is responsible for Progeria is called progerin.\u00a0In order to block normal cell function and cause Progeria, a molecule called a \u201cfarnesyl group\u201d must be attached to the progerin protein. FTIs act by blocking (inhibiting) the attachment of the farnesyl group onto progerin. \u00a0So if the FTI drug can block this farnesyl group attachment in children with Progeria, then progerin may be \u201cparalyzed\u201d and Progeria improved.\r\n\r\nFor the first time, we had in front of us a possible treatment for children with Progeria.\u00a0<\/b>A first-ever clinical drug trial<\/b><\/a>\u00a0began in 2007, and in 2012 <\/b>the study results were published, demonstrating that every child experienced improvement in one or more areas, including the vital cardiovascular system.\u00a0 In May 2014, further study revealed lonafarnib increases estimated lifespan by at least 1.6 years (time will tell if that number increases \u2013 it just hasn\u2019t been long enough to determine)\u00a0and in April 2018 a study published in The Journal of the American Medical Association (JAMA)<\/em> reported that lonafarnib alone extended survival in children with Progeria. \u00a0Click here<\/b><\/a> for details on the 2012 study,\u00a0here<\/b> <\/a>for details on the 2014 findings, and here<\/a><\/strong> for details on the 2018 study.\r\n\r\nIn May 2018, on the heels of the study published in JAMA<\/em>, PRF and Eiger Biopharmaceuticals entered into collaboration and supply agreement for the development and pursuit of U.S. Food and Drug Administration review and potential approval of lonafarnib for the treatment of Progeria in children. Lonafarnib will mark the first therapy submitted to the FDA for the treatment of Progeria.\r\n\r\nWe now have one treatment for Progeria but it is not a cure, so we continue to drive the research to discover drugs that will be even more effective, and ultimately cure Progeria.[\/vc_column_text][\/vc_column][\/vc_row]\t\t","_et_gb_content_width":"","footnotes":"","_links_to":"","_links_to_target":""},"class_list":["post-796","page","type-page","status-publish","hentry"],"yoast_head":"\nAanmelden<\/h2>\n
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