{"id":231,"date":"2025-10-15T15:30:06","date_gmt":"2025-10-15T13:30:06","guid":{"rendered":"https:\/\/ipsencom-staging.go-vip.net\/maladies-rares\/"},"modified":"2026-04-17T16:13:56","modified_gmt":"2026-04-17T14:13:56","slug":"maladies-rares","status":"publish","type":"page","link":"https:\/\/ipsencom-staging.go-vip.net\/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 banner-font-color-blue\">\n            <ol class=\"breadcrumb\">\n                <li class=\"breadcrumb-item\"><a href=\"https:\/\/ipsencom-staging.go-vip.net\/fr\/\">Accueil<\/a><\/li><li class=\"breadcrumb-item visible\"><a href=\"https:\/\/ipsencom-staging.go-vip.net\/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:\/\/ipsencom-staging.go-vip.net\/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>Nous sommes fiers d\u2019avoir d\u00e9velopp\u00e9 des th\u00e9rapies approuv\u00e9es dans les maladies h\u00e9patiques rares et les maladies osseuses ultra\u2011rares.<\/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-8f761849 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 une science au service d\u2019une mission claire dans les maladies rares<\/strong><\/h3>\n<\/div>\n<\/div>\n\n\n\n<div class=\"wp-block-columns is-layout-flex wp-container-core-columns-is-layout-8f761849 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:\/\/ipsencom-staging.go-vip.net\/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 cholestatiques 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                                <\/p>\n                                                <p>Notre objectif est d\u2019\u00e9largir les possibilit\u00e9s th\u00e9rapeutiques dans les maladies rares du foie, l\u00e0 o\u00f9 les options actuelles restent limit\u00e9es.<\/p>\n<p>Nous concentrons nos efforts sur cinq maladies h\u00e9patiques rares :<br \/>\nle syndrome d\u2019Alagille (ALGS), l\u2019atr\u00e9sie biliaire (BA), la cholangite biliaire primitive (PBC), la cholestase intrah\u00e9patique familiale progressive (PFIC) et la cholangite scl\u00e9rosante primitive (PSC).<\/p>\n<p>Notre ambition est de r\u00e9tablir l\u2019\u00e9quilibre des acides biliaires en modulant trois r\u00e9cepteurs cl\u00e9s impliqu\u00e9s dans le syst\u00e8me des acides biliaires, afin de soulager des sympt\u00f4mes invalidants et d\u2019am\u00e9liorer la fonction h\u00e9patique sur le long terme<\/p>\n                            <a \n                            aria-label=\" about  La science d\u2019Ipsen dans les maladies cholestatiques 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    <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 hidden\">\n                <h3 class=\"heading-three\" role=\"heading\" aria-level=\"2\">\n                                            <\/h3>\n                <\/div>\n               \n                <!-- If description is enabled then it's visible -->\n                <div class=\"text-parbase visible\">\n                    <p><strong>La science derri\u00e8re la modulation du syst\u00e8me des acides biliaires<\/strong><\/p>\n<p>Notre engagement va au\u2011del\u00e0 du soulagement des sympt\u00f4mes : nous cherchons \u00e0 g\u00e9n\u00e9rer des avanc\u00e9es capables de transformer l\u2019avenir des traitements et de red\u00e9finir ce qui est possible pour les personnes vivant avec des maladies rares du foie.<\/p>\n<p><strong>Inhibiteurs de l\u2019IBAT<\/strong><\/p>\n<p>Nous d\u00e9veloppons des inhibiteurs du transporteur il\u00e9al des acides biliaires (IBAT) qui r\u00e9duisent le recyclage des acides biliaires de l\u2019intestin gr\u00eale vers le foie.<\/p>\n<p>En limitant leur r\u00e9absorption, les inhibiteurs de l\u2019IBAT contribuent \u00e0 pr\u00e9venir l\u2019accumulation toxique d\u2019acides biliaires responsable de d\u00e9mangeaisons s\u00e9v\u00e8res et de l\u00e9sions h\u00e9patiques.<br \/>\nNotre approche vise \u00e0 r\u00e9pondre au besoin persistant de traitements capables de r\u00e9duire les sympt\u00f4mes tout en prot\u00e9geant la fonction h\u00e9patique.<\/p>\n<p><strong>Agonistes des PPAR<\/strong><\/p>\n<p>Nous faisons progresser des agonistes PPAR \u03b1\/\u03b4 qui activent des r\u00e9gulateurs cl\u00e9s au sein des cellules h\u00e9patiques, contr\u00f4lant l\u2019\u00e9quilibre des acides biliaires, le m\u00e9tabolisme et l\u2019inflammation.<\/p>\n<p>En am\u00e9liorant la mani\u00e8re dont le foie traite les acides biliaires et en r\u00e9duisant les signaux inflammatoires, ces th\u00e9rapies visent \u00e0 am\u00e9liorer le flux biliaire et \u00e0 ralentir les l\u00e9sions pouvant conduire \u00e0 la progression de la maladie.<\/p>\n                <\/div>\n\n                <!-- If button is enabled then it's visible -->\n                \n            <\/div>\n        <\/div>\n    <\/section>\n\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:\/\/ipsencom-staging.go-vip.net\/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                            <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:\/\/ipsencom-staging.go-vip.net\/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:\/\/ipsencom-staging.go-vip.net\/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:\/\/ipsencom-staging.go-vip.net\/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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