{"id":231,"date":"2025-10-15T15:30:06","date_gmt":"2025-10-15T13:30:06","guid":{"rendered":"https:\/\/www.ipsen.com\/maladies-rares\/"},"modified":"2025-11-05T08:00:54","modified_gmt":"2025-11-05T06:00:54","slug":"maladies-rares","status":"publish","type":"page","link":"https:\/\/www.ipsen.com\/fr\/science\/maladies-rares\/","title":{"rendered":"Maladies rares"},"content":{"rendered":"    <section class=\"sub-page-banner bg-color-disabled  banner-no-filter\" style=\"background: linear-gradient(93.64deg, #224A81 2.99%, #2797D3 97.01%)\">\n                <div class=\"container content-sec\">\n            <nav aria-label=\"breadcrumb\">\n            <ol class=\"breadcrumb\">\n                <li class=\"breadcrumb-item\"><a href=\"https:\/\/www.ipsen.com\/fr\/\">Accueil<\/a><\/li><li class=\"breadcrumb-item visible\"><a href=\"https:\/\/www.ipsen.com\/fr\/science\/\">Science<\/a><\/li><li class=\"breadcrumb-item active\"><a class=\"link-pointer-events-none\" href=\"#\" aria-current=\"page\">Maladies rares<\/a><\/li><\/ol>\n            <\/nav>            <h1 class=\"visible banner-font-color-white\">Maladies rares<\/h1>\n            <p class=\"visible banner-font-color-white\">Chez Ipsen, nous ouvrons de nouvelles voies dans les maladies rares des os et du foie, en apportant des solutions qui modifient l\u2019\u00e9volution de ces maladies et am\u00e9liorent les sympt\u00f4mes souvent invalidants qui affectent la vie quotidienne. <\/p>\n        <\/div>\n    <\/section>\n\n\n\n    <section class=\"title-text img-bg\">\n        <div class=\"container\">\n            <div class=\"title-text-inner\" style=\"background-image:url(https:\/\/www.ipsen.com\/wp-content\/uploads\/2025\/07\/Join-Us.png)\">\n\n                <!-- If heading is enabled then it's visible -->\n                <div class=\"text-head-dash visible\">\n                <h3 class=\"heading-three\" role=\"heading\" aria-level=\"2\">\n                        La raison d\u2019\u00eatre d\u2019Ipsen dans les maladies rares                    <\/h3>\n                <\/div>\n               \n                <!-- If description is enabled then it's visible -->\n                <div class=\"text-parbase visible\">\n                    <p><span data-contrast=\"auto\">Nous travaillons \u00e0 d\u00e9voiler la science derri\u00e8re les maladies rares. Nous identifions des cibles th\u00e9rapeutiques bien d\u00e9finies afin de d\u00e9velopper des m\u00e9dicaments qui modifient l\u2019\u00e9volution des maladies rares et soulagent les sympt\u00f4mes pour am\u00e9liorer la vie quotidienne des patients.<\/span><span data-ccp-props=\"{&quot;134233117&quot;:false,&quot;134233118&quot;:false,&quot;201341983&quot;:0,&quot;335559738&quot;:240,&quot;335559739&quot;:240,&quot;335559740&quot;:240}\">\u00a0<\/span><\/p>\n<p><span data-contrast=\"auto\">Nous sommes fiers d\u2019avoir d\u00e9velopp\u00e9 de nombreuses indications et traitements dans les maladies rares du foie, ainsi que d\u2019avoir fourni le premier traitement approuv\u00e9 pour une maladie osseuse ultra-rare.<\/span><span data-ccp-props=\"{&quot;134233117&quot;:false,&quot;134233118&quot;:false,&quot;201341983&quot;:0,&quot;335559738&quot;:240,&quot;335559739&quot;:240,&quot;335559740&quot;:240}\">\u00a0<\/span><\/p>\n<p><span data-contrast=\"auto\">En mettant en \u0153uvre la science translationnelle et en faisant progresser les th\u00e9rapies cibl\u00e9es, notre recherche se concentre sur l\u2019intersection entre l\u2019exp\u00e9rience v\u00e9cue des patients et la science innovante, afin de proposer des traitements porteurs de sens.<\/span><span data-ccp-props=\"{&quot;134233117&quot;:false,&quot;134233118&quot;:false,&quot;201341983&quot;:0,&quot;335559738&quot;:240,&quot;335559739&quot;:240,&quot;335559740&quot;:240}\">\u00a0<\/span><\/p>\n                <\/div>\n\n                <!-- If button is enabled then it's visible -->\n                \n            <\/div>\n        <\/div>\n    <\/section>\n\n\n\n<div class=\"wp-block-columns is-layout-flex wp-container-core-columns-is-layout-9d6595d7 wp-block-columns-is-layout-flex\">\n<div class=\"wp-block-column is-layout-flow wp-block-column-is-layout-flow\" style=\"flex-basis:100%\">\n<h3 class=\"wp-block-heading\"><strong>Comment Ipsen fait progresser la science dans les maladies rares avec d\u00e9termination<\/strong>&nbsp;<\/h3>\n<\/div>\n<\/div>\n\n\n\n<div class=\"wp-block-columns is-layout-flex wp-container-core-columns-is-layout-9d6595d7 wp-block-columns-is-layout-flex\">\n<div class=\"wp-block-column is-layout-flow wp-block-column-is-layout-flow\" style=\"flex-basis:1000%\"><!-- Text & Media Block Code -->\n<section class=\"two-col-section text-media-section\">\n    <div class=\"container two-section-innner \">\n        <div class=\"response-inner\" style=\"background-image: url('')\">\n            <div class=\"row \">\n                <div class=\"col-xs-12 col-sm-12 col-md-6\">\n                                                    <div class=\"img-video-section\">\n                                <img decoding=\"async\" \n                                    src=\"https:\/\/www.ipsen.com\/wp-content\/uploads\/2025\/10\/IPSEN_250226_SCIENCE-2_oncology-RVB-V2-1-7.png\" \n                                    alt=\"\" \n                                >\n                            <\/div>\n                                        <\/div>\n                <div class=\" col-xs-12 col-sm-12 col-md-6\">\n                    <div class=\"section-content\">\n                        <div class=\"heading-dash\">\n                                                            <span class=\"dash\"><\/span>\n                                <h4 class=\"heading-para heading-four\" role=\"heading\" aria-level=\"2\">\n                                    La science d\u2019Ipsen dans les maladies rares du foie                                <\/h4>\n                                                    <\/div>\n                                                    <p class=\"section-head-two heading-para\" role=\"heading\" aria-level=\"3\">\n                                    Utiliser la science pour r\u00e9tablir l\u2019\u00e9quilibre\u00a0                                <\/p>\n                                                <p><span data-contrast=\"none\">Nous concentrons nos efforts sur l\u2019\u00e9largissement des options th\u00e9rapeutiques pour les maladies rares du foie, l\u00e0 o\u00f9 les solutions actuelles demeurent limit\u00e9es.<\/span><\/p>\n<p><span data-contrast=\"none\">Nous ciblons cinq maladies rares du foie : ALGS, BA, PBC, PFIC et PSC, et visons \u00e0 r\u00e9tablir l\u2019\u00e9quilibre biliaire en modulant trois r\u00e9cepteurs impliqu\u00e9s dans le syst\u00e8me des acides biliaires, afin de soulager les sympt\u00f4mes invalidants et d\u2019am\u00e9liorer la sant\u00e9 h\u00e9patique \u00e0 long terme.<\/span><\/p>\n<p><span data-contrast=\"none\">Les inhibiteurs IBAT et ASBT r\u00e9duisent la r\u00e9absorption des acides biliaires dans l\u2019intestin, diminuant ainsi l\u2019accumulation toxique de bile responsable du prurit et des l\u00e9sions h\u00e9patiques. Les agonistes PPAR activent des r\u00e9cepteurs dans les cellules h\u00e9patiques pour r\u00e9guler le m\u00e9tabolisme des acides biliaires, r\u00e9duire l\u2019inflammation et contribuer \u00e0 ralentir la progression de la maladie.<\/span><\/p>\n<p><span data-contrast=\"none\">Notre engagement va au-del\u00e0 du soulagement des sympt\u00f4mes \u2013 nous visons des avanc\u00e9es qui red\u00e9finissent l\u2019avenir du traitement et \u00e9largissent le champ des possibles pour les personnes atteintes de maladies rares du foie<\/span><\/p>\n                            <a \n                            aria-label=\" about La science d\u2019Ipsen dans les maladies rares du foie\"  \n                                role=\"link\" \n                                class=\"btn-link hidden\"\n                                href=\"\" \n                                target=\"_self\"\n                            > \n                                 \n                            <\/a>\n\n                    <\/div>\n                <\/div>\n            <\/div>\n        <\/div>\n    <\/div>\n<\/section>\n\n<!-- Text & Media Block Code -->\n<section class=\"two-col-section text-media-section\">\n    <div class=\"container two-section-innner section-reverse\">\n        <div class=\"response-inner\" style=\"background-image: url('')\">\n            <div class=\"row section-reverse\">\n                <div class=\"col-xs-12 col-sm-12 col-md-6\">\n                                                    <div class=\"img-video-section\">\n                                <img decoding=\"async\" \n                                    src=\"https:\/\/www.ipsen.com\/wp-content\/uploads\/2025\/10\/Generic_Science_Key_Visual_\u2013_Science_Precision_Digital-none.jpg\" \n                                    alt=\"\" \n                                >\n                            <\/div>\n                                        <\/div>\n                <div class=\" col-xs-12 col-sm-12 col-md-6\">\n                    <div class=\"section-content\">\n                        <div class=\"heading-dash\">\n                                                            <span class=\"dash\"><\/span>\n                                <h4 class=\"heading-para heading-four\" role=\"heading\" aria-level=\"2\">\n                                    La science d\u2019Ipsen dans les maladies rares des os\u00a0                                <\/h4>\n                                                    <\/div>\n                                                    <p class=\"section-head-two heading-para\" role=\"heading\" aria-level=\"3\">\n                                    Cibler la source de la croissance osseuse anormale\u00a0                                <\/p>\n                                                <p><span data-contrast=\"none\">Nous faisons progresser l\u2019innovation th\u00e9rapeutique dans la fibrodysplasie ossifiante progressive (FOP), une maladie g\u00e9n\u00e9tique ultra-rare et lourdement invalidante, caract\u00e9ris\u00e9e par la formation anormale d\u2019os dans les tissus mous. Cette pathologie progressive entra\u00eene une perte s\u00e9v\u00e8re de mobilit\u00e9, des difficult\u00e9s respiratoires et une esp\u00e9rance de vie significativement r\u00e9duite.<\/span><\/p>\n<p><span data-contrast=\"none\">La FOP est caus\u00e9e par une activit\u00e9 excessive d\u2019un r\u00e9cepteur appel\u00e9 ALK2, faisant partie de la voie de signalisation des prot\u00e9ines morphog\u00e9n\u00e9tiques osseuses (BMP) \u2013 un syst\u00e8me qui r\u00e9gule normalement le d\u00e9veloppement osseux sain.\u00a0<\/span><\/p>\n<p><span data-contrast=\"none\">Nos recherches in\u00e9dites ciblent plusieurs points de la voie BMP pour bloquer les signaux anormaux qui alimentent la maladie. En capitalisant sur le premier traitement approuv\u00e9 et en ouvrant de nouvelles pistes d\u2019investigation, nous visons \u00e0 poursuivre notre r\u00f4le de leader dans la FOP et \u00e0 apporter un changement durable dans la vie des patients et de leurs familles.<\/span><span data-ccp-props=\"{&quot;134233117&quot;:true,&quot;134233118&quot;:true,&quot;201341983&quot;:0,&quot;335559739&quot;:0,&quot;335559740&quot;:240}\">\u00a0<\/span><\/p>\n                            <a \n                            aria-label=\" about La science d\u2019Ipsen dans les maladies rares des os\u00a0\"  \n                                role=\"link\" \n                                class=\"btn-link hidden\"\n                                href=\"\" \n                                target=\"_self\"\n                            > \n                                 \n                            <\/a>\n\n                    <\/div>\n                <\/div>\n            <\/div>\n        <\/div>\n    <\/div>\n<\/section><\/div>\n<\/div>\n\n\n    <section class=\"title-text \">\n        <div class=\"container\">\n            <div class=\"title-text-inner\" style=\"background-color:#EDEBE4;\">\n\n                <!-- If heading is enabled then it's visible -->\n                <div class=\"text-head-dash visible\">\n                <h3 class=\"heading-three\" role=\"heading\" aria-level=\"2\">\n                        Rencontrez l\u2019\u00e9quipe                    <\/h3>\n                <\/div>\n               \n                <!-- If description is enabled then it's visible -->\n                <div class=\"text-parbase visible\">\n                    <p><span data-contrast=\"none\">D\u00e9couvrez les personnes qui font avancer la R&amp;D chez Ipsen, en transformant la science en impact pour les patients, de la d\u00e9couverte \u00e0 la mise \u00e0 disposition des traitements.<\/span><\/p>\n                <\/div>\n\n                <!-- If button is enabled then it's visible -->\n                                        <div class=\"btn-txt\">\n                            <a \n                                aria-label=\"\" \n                                class=\"btn-link section-btn cta-visible\"  \n                                role=\"link\"\n                                href=\"https:\/\/www.ipsen.com\/fr\/profils\/\" target=\"_self\">\n                                En savoir plus                            <\/a>\n                        <\/div>\n                \n            <\/div>\n        <\/div>\n    <\/section>\n\n\n    <section class=\"title-text img-bg\">\n        <div class=\"container\">\n            <div class=\"title-text-inner\" style=\"background-image:url(https:\/\/www.ipsen.com\/wp-content\/uploads\/2025\/07\/Join-Us.png)\">\n\n                <!-- If heading is enabled then it's visible -->\n                <div class=\"text-head-dash visible\">\n                <h3 class=\"heading-three\" role=\"heading\" aria-level=\"2\">\n                        La science guid\u00e9e par le sens                    <\/h3>\n                <\/div>\n               \n                <!-- If description is enabled then it's visible -->\n                <div class=\"text-parbase visible\">\n                    <p><span data-contrast=\"none\">Nous d\u00e9veloppons des m\u00e9dicaments en oncologie, en maladies rares et en neurosciences gr\u00e2ce \u00e0 une innovation guid\u00e9e par la science. Explorez notre domaine des Neurosciences pour d\u00e9couvrir comment nous transformons l\u2019innovation en traitement.<\/span><\/p>\n                <\/div>\n\n                <!-- If button is enabled then it's visible -->\n                                        <div class=\"btn-txt\">\n                            <a \n                                aria-label=\"\" \n                                class=\"btn-link section-btn cta-visible\"  \n                                role=\"link\"\n                                href=\"https:\/\/www.ipsen.com\/fr\/science-2\/neurosciences\/\" target=\"_self\">\n                                En savoir plus sur les Neurosciences                            <\/a>\n                        <\/div>\n                \n            <\/div>\n        <\/div>\n    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