{"id":101,"date":"2023-12-22T06:53:39","date_gmt":"2023-12-22T04:53:39","guid":{"rendered":"https:\/\/www.ipsen.com\/be\/?page_id=101"},"modified":"2025-07-29T11:00:34","modified_gmt":"2025-07-29T09:00:34","slug":"maladies-rares","status":"publish","type":"page","link":"https:\/\/www.ipsen.com\/be\/fr\/maladies-rares\/","title":{"rendered":"Maladies rares"},"content":{"rendered":"    <section class=\"sub-page-banner bg-img-enabled  banner-no-filter\" style=\"background-image:url(https:\/\/www.ipsen.com\/be\/wp-content\/uploads\/sites\/7\/2025\/07\/Health-Rare-Disease.jpg)\">\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\/be\/fr\/\">Accueil<\/a><\/li><li class=\"breadcrumb-item active\"><a href=\"#\" aria-current=\"page\">Science &#8211; 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\">\u00a0D\u00e9velopper des solutions th\u00e9rapeutiques pour les personnes vivant avec des maladies rares et ultra-rares<\/p>\n        <\/div>\n    <\/section>\n\n\n\n<!-- Ipsen Content Overlay Start -->\n<section class=\"two-col-image-section\">\n    <div class=\"container two-img-section-innner\"  >\n        <div class=\"content-overlay-inner bg-color-enabled\" style=\"background-color:#EDEBE4\">\n                <!-- Ipsen Content Overlay Variation Two Start -->\n                <div class=\"row \">\n                    <div class=\"col-xs-12 col-sm-12 col-md-6 column-1 \">\n                        <div id=\"image-carousel\" class=\"img-video-section carousel\" data-bs-ride=\"carousel\">\n                            <div class=\"carousel-inner\">\n                                                                        <div class=\"carousel-item active\">\n                                            <img decoding=\"async\" src=\"https:\/\/www.ipsen.com\/be\/wp-content\/uploads\/sites\/7\/2024\/08\/Image21.jpg\" alt=\"Media Image\" >\n                                        <\/div>\n                                                            <\/div>\n                                                    <\/div>\n                    <\/div>\n                    <div class=\" col-xs-12 col-sm-12 col-md-6 column-2 \">\n                        <div class=\" section-content\">\n                                                                <div class=\"heading-dash\">\n                                        <span class=\"dash\"><\/span>\n                                        <h4 role=\"heading\" aria-level=\"2\" class=\"heading-para-right heading-four\">\n                                            Notre engagement dans les maladies rares                                        <\/h4>\n                                    <\/div>\n                                                                                                                        <p class=\"section-para \">\n                                        94\u00a0% des maladies rares n&#8217;ont toujours pas de traitement. Aujourd&#8217;hui, nos \u00e9quipes d\u00e9di\u00e9es aux maladies rares sont plus que jamais d\u00e9termin\u00e9es \u00e0 y rem\u00e9dier. Fort de notre expertise scientifique et de notre pr\u00e9sence mondiale, nous nous appuyons sur une&#8230;                                    <\/p>\n                                                                                <\/div>\n                    <\/div>\n                    <!-- Ipsen Content Overlay Variation Two End -->\n                <\/div>\n        <!-- Ipsen Content Overlay Variation two End -->\n                <\/div>\n    <\/div>\n<\/section>\n<!-- Ipsen Content Overlay End -->\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\/be\/wp-content\/uploads\/sites\/7\/2024\/02\/Module-2_Our-commitment-ro-rare-disease-Brief-about-rare-disease_20201010_5529-HD_03-scaled-1.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                                    \u00c9couter les personnes atteintes de maladies rares                                <\/h4>\n                                                    <\/div>\n                                                <p>Centr\u00e9s sur la prise en charge des patients, nous nous effor\u00e7ons de comprendre les d\u00e9fis auxquels les personnes qui vivent avec des maladies rares font face. Nous apprenons de leurs exp\u00e9riences et int\u00e9grons leurs \u00e9clairages tout au long du processus de d\u00e9veloppement. Cette approche nous a permis d\u2019acc\u00e9l\u00e9rer notre dynamique pour la mise au point de traitements pour les maladies et les troubles osseux extr\u00eamement rares, les maladies rares du foie et les troubles de la croissance.<\/p>\n                            <a \n                            aria-label=\" En savoir plus  about \u00c9couter les personnes atteintes de maladies rares\"  \n                                role=\"link\" \n                                class=\"btn-link section-btn cta-visible\"\n                                href=\"https:\/\/www.ipsen.com\/fr\/patients\/\" \n                                target=\"_blank\"\n                            > \n                                 En savoir plus  \n                            <\/a>\n\n                    <\/div>\n                <\/div>\n            <\/div>\n        <\/div>\n    <\/div>\n<\/section>\n\n<!--Graphics Cards Carousel Start Here-->\n\n    <section class=\"graphic-card graphics-old-layout bg-color-disabled bg-image-disabled bg-video-disabled bg-mobile-video-img-disabled\"    data-displaycards=\"3\">\n                <div class=\"container\">\n            <div class=\"text-graph-inner\">\n                <div class=\"row\">\n                    <!--Graphic info section-->\n                    <div class=\"column-1 col-xl-12 col-md-12 col-sm-12\">\n                        <div class=\"text-graph-heading\">\n                        <h3 class=\"charcoal-grey visible heading-three\" role=\"heading\" aria-level=\"2\">\n                                Des paradigmes de traitement complexes                             <\/h3>\n                            <p class=\"text-graph-para charcoal-grey visible\">Ipsen communiquera sur les donn\u00e9es d&#8217;un essai clinique de Phase III qui sont critiques pour le traitement de la cholangite biliaire primitive (CBP), une maladie auto-immune du foie, et la prise en charge de ses sympt\u00f4mes invalidants. <\/p>\n                        <\/div>\n                    <\/div><!--Graphic info section over here -->\n\n                    <!--Graphic detail section-->\n                    <div class=\"column-2 col-xl-12 col-md-12 col-sm-12\">\n                        <!-- graphic-card carousel start here -->\n                                                    <div class=\"text-graph-carousel\">\n                                <div class=\"glider-contain multiple\">\n                                                                                    <div class=\"text-graph-content-btn\">\n                                                    <button aria-hidden=\"true\" aria-label=\"Pr\u00e9c\u00e9dent\" class=\"glider-graphic-card-prev\"><span><\/span><\/button>\n                                                    <button aria-hidden=\"true\" aria-label=\"Suivant\" class=\"glider-graphic-card-next\"><span><\/span><\/button>\n                                                <\/div>\n                                    \n                                <div class=\"glider-graphic-card\" tabindex=\"-1\">\n                                                                                                                        <div class=\"text-graphic\">\n                                                    <div class=\"text-graph-row\">\n                                                        \n                                                                <!-- Graphic Card With OLD layout start here -->\n                                                                <div class=\"column-1\">\n                                                                                                                                                <div class=\"text-graph-img visible graphic-icon-enabled \">\n                                                                                                                                                                    <img decoding=\"async\" src=\"https:\/\/www.ipsen.com\/be\/wp-content\/uploads\/sites\/7\/2025\/07\/Portfolio-1.png\" alt=\"\" \/> \n                                                                                                                                                            <\/div>\n                                                                                                                                    <\/div>\n                                                                <div class=\"column-2\">\n                                                                    <div class=\"text-graph-content\">\n                                                                                                                                                        <div class=\"text-graph-title\">\n                                                                                <h4 role=\"heading\" aria-level=\"3\">Des probl\u00e9matiques uniques qui exigent des solutions uniques <\/h4>\n                                                                                <\/div>\n                                                                                                                                                <div class=\"text-graph-desc\">\n                                                                            <p><p>En 2023, nous avons int\u00e9gr\u00e9 \u00e0 notre portefeuille Maladies Rares des traitements prometteurs et des actifs exp\u00e9rimentaux pour des maladies rares du foie chez l&#8217;enfant et l&#8217;adulte.<\/p>\n<\/p>\n                                                                        <\/div>\n                                                                    <\/div>\n                                                                <\/div>\n                                                                <!-- Graphic Card With OLD layout end here -->\n                                                        \n                                                    <\/div>\n                                                <\/div>\n                                                                                                                                                                    <div class=\"text-graphic\">\n                                                    <div class=\"text-graph-row\">\n                                                        \n                                                                <!-- Graphic Card With OLD layout start here -->\n                                                                <div class=\"column-1\">\n                                                                                                                                                <div class=\"text-graph-img visible graphic-icon-enabled \">\n                                                                                                                                                                    <img decoding=\"async\" src=\"https:\/\/www.ipsen.com\/be\/wp-content\/uploads\/sites\/7\/2025\/07\/People.png\" alt=\"\" \/> \n                                                                                                                                                            <\/div>\n                                                                                                                                    <\/div>\n                                                                <div class=\"column-2\">\n                                                                    <div class=\"text-graph-content\">\n                                                                                                                                                        <div class=\"text-graph-title\">\n                                                                                <h4 role=\"heading\" aria-level=\"3\">Une lutte permanente pour r\u00e9pondre aux besoins des patients<\/h4>\n                                                                                <\/div>\n                                                                                                                                                <div class=\"text-graph-desc\">\n                                                                            <p><p>94 % des maladies rares n&#8217;ont toujours pas de traitement approuv\u00e9.<\/p>\n<\/p>\n                                                                        <\/div>\n                                                                    <\/div>\n                                                                <\/div>\n                                                                <!-- Graphic Card With OLD layout end here -->\n                                                        \n                                                    <\/div>\n                                                <\/div>\n                                                                                                                                                                    <div class=\"text-graphic\">\n                                                    <div class=\"text-graph-row\">\n                                                        \n                                                                <!-- Graphic Card With OLD layout start here -->\n                                                                <div class=\"column-1\">\n                                                                                                                                                <div class=\"text-graph-img visible graphic-icon-enabled \">\n                                                                                                                                                                    <img decoding=\"async\" src=\"https:\/\/www.ipsen.com\/be\/wp-content\/uploads\/sites\/7\/2025\/07\/Family.png\" alt=\"\" \/> \n                                                                                                                                                            <\/div>\n                                                                                                                                    <\/div>\n                                                                <div class=\"column-2\">\n                                                                    <div class=\"text-graph-content\">\n                                                                                                                                                        <div class=\"text-graph-title\">\n                                                                                <h4 role=\"heading\" aria-level=\"3\">Rare, mais pas Seul.e<\/h4>\n                                                                                <\/div>\n                                                                                                                                                <div class=\"text-graph-desc\">\n                                                                            <p><p>350 \u00e0 400 millions de personnes sont touch\u00e9es par 7 000 maladies rares dans le monde.<\/p>\n<\/p>\n                                                                        <\/div>\n                                                                    <\/div>\n                                                                <\/div>\n                                                                <!-- Graphic Card With OLD layout end here -->\n                                                        \n                                                    <\/div>\n                                                <\/div>\n                                                                                                                                                                    <div class=\"text-graphic\">\n                                                    <div class=\"text-graph-row\">\n                                                        \n                                                                <!-- Graphic Card With OLD layout start here -->\n                                                                <div class=\"column-1\">\n                                                                                                                                                <div class=\"text-graph-img visible graphic-icon-enabled \">\n                                                                                                                                                                    <img decoding=\"async\" src=\"https:\/\/www.ipsen.com\/be\/wp-content\/uploads\/sites\/7\/2025\/07\/Clinical-Studies.png\" alt=\"\" \/> \n                                                                                                                                                            <\/div>\n                                                                                                                                    <\/div>\n                                                                <div class=\"column-2\">\n                                                                    <div class=\"text-graph-content\">\n                                                                                                                                                        <div class=\"text-graph-title\">\n                                                                                <h4 role=\"heading\" aria-level=\"3\">Innover pour les patients atteints de maladies rares <\/h4>\n                                                                                <\/div>\n                                                                                                                                                <div class=\"text-graph-desc\">\n                                                                            <p><p>Notre portefeuille de produits au stade de d\u00e9veloppement clinique dans les maladies rares a plus que doubl\u00e9 depuis 2020.<\/p>\n<\/p>\n                                                                        <\/div>\n                                                                    <\/div>\n                                                                <\/div>\n                                                                <!-- Graphic Card With OLD layout end here -->\n                                                        \n                                                    <\/div>\n                                                <\/div>\n                                                                                                                <\/div>\n                            <\/div>\n                                            <\/div> <!-- graphic-card carousel end here -->\n                <\/div> <!--Graphic detail section over here with 12 grid -->   \n            <\/div><!--row ended here-->\n        <\/div> <!-- graph-inner ended here -->\n        \n            <div class=\"more-result-btn\">\n                                            <\/div>\n        <\/div> <!--Container class ended here -->\n    <\/section>\n<!--Graphics Cards Carousel End Here-->\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                        Des travaux pionniers 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>Au c\u0153ur de sa strat\u00e9gie, Ipsen se concentre depuis de nombreuses ann\u00e9es sur le d\u00e9veloppement de traitements transformateurs pour les patients atteints de maladies rares.<\/p>\n                <\/div>\n\n                <!-- If button is enabled then it's visible -->\n                \n            <\/div>\n        <\/div>\n    <\/section>\n\n\n \n\n\n    <!--Accordion Cards Start Here-->\n    <section class=\"accordion bg-color-disabled\"  data-according-id=\"43083567\">\n        <div class=\"container\">\n            <div class=\"row\">\n                <!--Accordion info section-->\n                <div class=\"column-1 col-xl-12 col-md-12 col-sm-12\">\n                    <div class=\"accordian-info\">\n                        <h3 class=\"accordian-heading visible heading-three\" role=\"heading\" aria-level=\"2\">\n                            Troubles osseux                        <\/h3>\n                        <p class=\"accordian-summary hidden\"><\/p>\n                    <\/div>\n                <\/div><!--Accordion info section over here -->\n\n                <div class=\"col-xl-12 col-lg-12 col-md-12 col-sm-12\">\n                    <div class=\"accordion accor-group\" id=\"accordionExample43083567\">\n                         \n                                    <!-- Accordion with Expandable panel & FAQs -->\n                                    <div class=\"accordion-item accor-item\">\n                                        <p  role=\"heading\" aria-level=\"3\" class=\"accordion-header accor-head-para\" id=\"expHeading0\">\n                                            <button class=\"accordion-button accor-btn collapsed\" type=\"button\" data-bs-toggle=\"collapse\" data-bs-target=\"#expHeading430835670\" aria-expanded=\"false\" aria-controls=\"expHeading430835670\">\n                                                Fibrodysplasie ossifiante progressive (FOP)                                             <\/button>\n                                        <\/p>\n                                        <div id=\"expHeading430835670\" class=\"accordion-collapse collapse \" aria-labelledby=\"expHeading0\" data-bs-parent=\"#accordionExample43083567\">\n                                            <div class=\"accordion-body\">\n                                                <div class=\"accordion-data\">\n                                                    <p><p>La FOP provoque une formation osseuse continue et permanente dans les tissus mous et conjonctifs, comme les muscles, les tendons et les ligaments. Elle est \u00e9galement connue sous le nom d&#8217;ossification h\u00e9t\u00e9rotopique ou OH. Il s&#8217;agit d&#8217;une maladie g\u00e9n\u00e9tique ultra-rare, caract\u00e9ris\u00e9e par des pouss\u00e9es inflammatoires qui commencent d\u00e8s l&#8217;enfance. L&#8217;OH est irr\u00e9versible et entra\u00eene de graves limitations fonctionnelles de la mobilit\u00e9, une invalidit\u00e9 progressive et cumulative ainsi qu&#8217;une diminution de l\u2019esp\u00e9rance de vie. La maladie est diagnostiqu\u00e9e en moyenne \u00e0 l&#8217;\u00e2ge de 5 ans. \u00c0 terme, la FOP abr\u00e8ge l&#8217;esp\u00e9rance de vie m\u00e9diane pour la ramener \u00e0 56 ans. Environ 900 personnes dans le monde vivent avec la FOP. Ipsen exploite de nouveaux m\u00e9canismes d\u2019action avec le d\u00e9veloppement de deux actifs exp\u00e9rimentaux pour apporter potentiellement des traitements innovants aux personnes atteintes de FOP.<\/p>\n<\/p>\n                                                    <div class=\"cta-block hidden\" >\n                                                                                                            <\/div>\n                                                <\/div>\n                                            <\/div>\n                                        <\/div>\n                                    <\/div>\n                                                <\/div>\n                <\/div> \n            <\/div>\n        <\/div>\n    <\/section>\n    <!--Accordion Cards End Here-->\n\n\n\n    <!--Accordion Cards Start Here-->\n    <section class=\"accordion bg-color-disabled\"  data-according-id=\"363565906\">\n        <div class=\"container\">\n            <div class=\"row\">\n                <!--Accordion info section-->\n                <div class=\"column-1 col-xl-12 col-md-12 col-sm-12\">\n                    <div class=\"accordian-info\">\n                        <h3 class=\"accordian-heading visible heading-three\" role=\"heading\" aria-level=\"2\">\n                            Troubles de la croissance                        <\/h3>\n                        <p class=\"accordian-summary hidden\"><\/p>\n                    <\/div>\n                <\/div><!--Accordion info section over here -->\n\n                <div class=\"col-xl-12 col-lg-12 col-md-12 col-sm-12\">\n                    <div class=\"accordion accor-group\" id=\"accordionExample363565906\">\n                         \n                                    <!-- Accordion with Expandable panel & FAQs -->\n                                    <div class=\"accordion-item accor-item\">\n                                        <p  role=\"heading\" aria-level=\"3\" class=\"accordion-header accor-head-para\" id=\"expHeading0\">\n                                            <button class=\"accordion-button accor-btn collapsed\" type=\"button\" data-bs-toggle=\"collapse\" data-bs-target=\"#expHeading3635659060\" aria-expanded=\"false\" aria-controls=\"expHeading3635659060\">\n                                                Acrom\u00e9galie                                            <\/button>\n                                        <\/p>\n                                        <div id=\"expHeading3635659060\" class=\"accordion-collapse collapse \" aria-labelledby=\"expHeading0\" data-bs-parent=\"#accordionExample363565906\">\n                                            <div class=\"accordion-body\">\n                                                <div class=\"accordion-data\">\n                                                    <p><p>L\u2019acrom\u00e9galie est une maladie rare caus\u00e9e par une tumeur b\u00e9nigne (non canc\u00e9reuse) de l\u2019hypophyse, appel\u00e9e ad\u00e9nome, qui provoque la production excessive d\u2019hormone de croissance circulant dans le sang. Par cons\u00e9quent, le corps fabrique davantage une autre hormone, le facteur de croissance insulino-mim\u00e9tique recombinant (IGF-1). Normalement, l\u2019IGF-1 fonctionne avec l\u2019hormone de croissance pour contr\u00f4ler la croissance de nombreux tissus de l\u2019organisme. Lorsque la production d\u2019IGF-1 et d\u2019hormone de croissance est excessive, le corps peut subir une croissance anormale des os, du cartilage et d\u2019autres tissus corporels. Outre les signes ext\u00e9rieurs tels que l\u2019\u00e9largissement des mains, des pieds et du visage, ce trouble peut entra\u00eener des probl\u00e8mes de sant\u00e9 plus graves.<\/p>\n<\/p>\n                                                    <div class=\"cta-block hidden\" >\n                                                                                                            <\/div>\n                                                <\/div>\n                                            <\/div>\n                                        <\/div>\n                                    <\/div>\n                             \n                                    <!-- Accordion with Expandable panel & FAQs -->\n                                    <div class=\"accordion-item accor-item\">\n                                        <p  role=\"heading\" aria-level=\"3\" class=\"accordion-header accor-head-para\" id=\"expHeading1\">\n                                            <button class=\"accordion-button accor-btn collapsed\" type=\"button\" data-bs-toggle=\"collapse\" data-bs-target=\"#expHeading3635659061\" aria-expanded=\"false\" aria-controls=\"expHeading3635659061\">\n                                                D\u00e9ficit en hormone de croissance                                             <\/button>\n                                        <\/p>\n                                        <div id=\"expHeading3635659061\" class=\"accordion-collapse collapse \" aria-labelledby=\"expHeading1\" data-bs-parent=\"#accordionExample363565906\">\n                                            <div class=\"accordion-body\">\n                                                <div class=\"accordion-data\">\n                                                    <p><p>Le d\u00e9ficit en hormone de croissance chez l\u2019enfant (PGHD) et le d\u00e9ficit en hormone de croissance chez l\u2019adulte (AGHD) surviennent lorsque le corps est incapable de produire suffisamment d\u2019hormone de croissance ou de l\u2019utiliser efficacement. L\u2019hormone de croissance joue un r\u00f4le essentiel dans la croissance et le d\u00e9veloppement, notamment pour l\u2019am\u00e9lioration du m\u00e9tabolisme musculaire, la croissance des os et l\u2019assimilation des graisses. Une s\u00e9cr\u00e9tion insuffisante d\u2019hormone de croissance peut avoir un impact sur la croissance et les fonctions corporelles.<\/p>\n<p>&nbsp;<\/p>\n<p>Le PGHD peut se d\u00e9clarer d\u00e8s la naissance ou se d\u00e9velopper \u00e0 n\u2019importe quel moment au cours de l\u2019enfance ou de l\u2019adolescence. Dans les deux cas, le PGHD peut \u00eatre caus\u00e9 par :<\/p>\n<ul>\n<li>une tumeur dans et autour de l\u2019hypophyse ;<\/li>\n<li>des dommages ou une anomalie au niveau de l\u2019hypothalamus ;<\/li>\n<li>des dommages au niveau de l\u2019hypophyse li\u00e9s \u00e0 une infection, une maladie vasculaire, un traumatisme cr\u00e2nien grave ou une radioth\u00e9rapie.<\/li>\n<\/ul>\n<p>Les dommages de l\u2019hypophyse peuvent affecter la production d\u2019une ou plusieurs hormones, y compris l\u2019hormone de croissance.<\/p>\n<\/p>\n                                                    <div class=\"cta-block hidden\" >\n                                                                                                            <\/div>\n                                                <\/div>\n                                            <\/div>\n                                        <\/div>\n                                    <\/div>\n                             \n                                    <!-- Accordion with Expandable panel & FAQs -->\n                                    <div class=\"accordion-item accor-item\">\n                                        <p  role=\"heading\" aria-level=\"3\" class=\"accordion-header accor-head-para\" id=\"expHeading2\">\n                                            <button class=\"accordion-button accor-btn collapsed\" type=\"button\" data-bs-toggle=\"collapse\" data-bs-target=\"#expHeading3635659062\" aria-expanded=\"false\" aria-controls=\"expHeading3635659062\">\n                                                Petite taille associ\u00e9e au syndrome de Turner                                            <\/button>\n                                        <\/p>\n                                        <div id=\"expHeading3635659062\" class=\"accordion-collapse collapse \" aria-labelledby=\"expHeading2\" data-bs-parent=\"#accordionExample363565906\">\n                                            <div class=\"accordion-body\">\n                                                <div class=\"accordion-data\">\n                                                    <p><p>Le syndrome de Turner est une maladie g\u00e9n\u00e9tique qui survient lorsque l&#8217;un des deux chromosomes X chez la fille est absent ou incomplet. La caract\u00e9ristique la plus courante du syndrome de Turner est la petite taille, qui affecte presque toutes les filles atteintes de ce syndrome. En effet, le chromosome X manquant ou incomplet contient un g\u00e8ne qui contribue \u00e0 atteindre une taille normale. De la naissance \u00e0 l\u2019adolescence, les filles atteintes du syndrome de Turner grandissent plus lentement que les enfants non atteints. La taille moyenne adulte des femmes atteintes du syndrome de Turner est de l\u2019ordre de 145 cm, tandis que les femmes non affect\u00e9es mesurent en moyenne environ 165 cm.<\/p>\n<\/p>\n                                                    <div class=\"cta-block hidden\" >\n                                                                                                            <\/div>\n                                                <\/div>\n                                            <\/div>\n                                        <\/div>\n                                    <\/div>\n                             \n                                    <!-- Accordion with Expandable panel & FAQs -->\n                                    <div class=\"accordion-item accor-item\">\n                                        <p  role=\"heading\" aria-level=\"3\" class=\"accordion-header accor-head-para\" id=\"expHeading3\">\n                                            <button class=\"accordion-button accor-btn collapsed\" type=\"button\" data-bs-toggle=\"collapse\" data-bs-target=\"#expHeading3635659063\" aria-expanded=\"false\" aria-controls=\"expHeading3635659063\">\n                                                Retard de croissance associ\u00e9 \u00e0 une insuffisance r\u00e9nale chronique                                            <\/button>\n                                        <\/p>\n                                        <div id=\"expHeading3635659063\" class=\"accordion-collapse collapse \" aria-labelledby=\"expHeading3\" data-bs-parent=\"#accordionExample363565906\">\n                                            <div class=\"accordion-body\">\n                                                <div class=\"accordion-data\">\n                                                    <p><p>L\u2019insuffisance r\u00e9nale chronique est une maladie rare qui cause la perte de la fonction r\u00e9nale sur une p\u00e9riode allant de plusieurs mois \u00e0 plusieurs ann\u00e9es. La plupart des jeunes atteints d\u2019insuffisance r\u00e9nale chronique produisent autant d\u2019hormone de croissance que n&#8217;importe quelle personne de leur \u00e2ge, mais leurs reins n\u2019\u00e9liminent pas suffisamment les d\u00e9chets pr\u00e9sents dans le sang. \u00c0 cause de ces d\u00e9chets non \u00e9vacu\u00e9s, le corps ne parvient pas \u00e0 utiliser efficacement les hormones de croissance. En r\u00e8gle g\u00e9n\u00e9rale, l\u2019hormone de croissance signale au facteur de croissance analogue \u00e0 l&#8217;insuline (IGF-1) de se d\u00e9placer vers les muscles, les organes et les os, indique aux os de se d\u00e9velopper et aide \u00e0 am\u00e9liorer le m\u00e9tabolisme musculaire. \u00c0 cause de l\u2019insuffisance r\u00e9nale chronique, l\u2019hormone de croissance et l\u2019IGF ne fonctionnent pas correctement ensemble. Cela peut entra\u00eener un retard ou un arr\u00eat de la croissance, ainsi que des troubles du d\u00e9veloppement des muscles, des organes et des os.<\/p>\n<\/p>\n                                                    <div class=\"cta-block hidden\" >\n                                                                                                            <\/div>\n                                                <\/div>\n                                            <\/div>\n                                        <\/div>\n                                    <\/div>\n                             \n                                    <!-- Accordion with Expandable panel & FAQs -->\n                                    <div class=\"accordion-item accor-item\">\n                                        <p  role=\"heading\" aria-level=\"3\" class=\"accordion-header accor-head-para\" id=\"expHeading4\">\n                                            <button class=\"accordion-button accor-btn collapsed\" type=\"button\" data-bs-toggle=\"collapse\" data-bs-target=\"#expHeading3635659064\" aria-expanded=\"false\" aria-controls=\"expHeading3635659064\">\n                                                D\u00e9ficit primaire s\u00e9v\u00e8re en facteur de croissance analogue \u00e0 l\u2019insuline 1 (IGF-1) (SPIGFD)                                            <\/button>\n                                        <\/p>\n                                        <div id=\"expHeading3635659064\" class=\"accordion-collapse collapse \" aria-labelledby=\"expHeading4\" data-bs-parent=\"#accordionExample363565906\">\n                                            <div class=\"accordion-body\">\n                                                <div class=\"accordion-data\">\n                                                    <p><p>Le d\u00e9ficit primaire s\u00e9v\u00e8re en IGF-1 (SPIGFD) survient chez l\u2019enfant lorsque le facteur de croissance analogue \u00e0 l\u2019insuline (IGF-1), une hormone naturelle essentielle pour favoriser la croissance de l\u2019enfant, est produite en quantit\u00e9 insuffisante. Il est possible que des taux faibles d\u2019IGF-1, en pr\u00e9sence de taux d\u2019hormone de croissance normaux voire \u00e9lev\u00e9s, expliquent pourquoi les enfants atteints sont plus petits que presque tous les autres enfants du m\u00eame \u00e2ge et du m\u00eame sexe.<\/p>\n<\/p>\n                                                    <div class=\"cta-block hidden\" >\n                                                                                                            <\/div>\n                                                <\/div>\n                                            <\/div>\n                                        <\/div>\n                                    <\/div>\n                             \n                                    <!-- Accordion with Expandable panel & FAQs -->\n                                    <div class=\"accordion-item accor-item\">\n                                        <p  role=\"heading\" aria-level=\"3\" class=\"accordion-header accor-head-para\" id=\"expHeading5\">\n                                            <button class=\"accordion-button accor-btn collapsed\" type=\"button\" data-bs-toggle=\"collapse\" data-bs-target=\"#expHeading3635659065\" aria-expanded=\"false\" aria-controls=\"expHeading3635659065\">\n                                                 Pubert\u00e9 centrale pr\u00e9coce                                            <\/button>\n                                        <\/p>\n                                        <div id=\"expHeading3635659065\" class=\"accordion-collapse collapse \" aria-labelledby=\"expHeading5\" data-bs-parent=\"#accordionExample363565906\">\n                                            <div class=\"accordion-body\">\n                                                <div class=\"accordion-data\">\n                                                    <p><p>La pubert\u00e9 centrale pr\u00e9coce (PCC) survient lorsqu\u2019un enfant devient un jeune adulte \u00e0 un \u00e2ge pr\u00e9coce inhabituel, car ses glandes de croissance et d\u2019hormones sexuelles commencent \u00e0 fonctionner t\u00f4t. La pubert\u00e9 centrale pr\u00e9coce peut \u00eatre caus\u00e9e par des tumeurs au niveau du syst\u00e8me nerveux central et d\u2019autres troubles du syst\u00e8me nerveux central. Cependant, un grand nombre de cas de pubert\u00e9 centrale pr\u00e9coce sont d\u2019origine inconnue, en particulier chez les femmes. Les chercheurs commencent \u00e0 identifier des causes g\u00e9n\u00e9tiques rares dans certains cas.<\/p>\n<\/p>\n                                                    <div class=\"cta-block hidden\" >\n                                                                                                            <\/div>\n                                                <\/div>\n                                            <\/div>\n                                        <\/div>\n                                    <\/div>\n                                                <\/div>\n                <\/div> \n            <\/div>\n        <\/div>\n    <\/section>\n    <!--Accordion Cards End Here-->\n\n\n\n    <!--Accordion Cards Start Here-->\n    <section class=\"accordion bg-color-disabled\"  data-according-id=\"646203835\">\n        <div class=\"container\">\n            <div class=\"row\">\n                <!--Accordion info section-->\n                <div class=\"column-1 col-xl-12 col-md-12 col-sm-12\">\n                    <div class=\"accordian-info\">\n                        <h3 class=\"accordian-heading visible heading-three\" role=\"heading\" aria-level=\"2\">\n                            \u00a0Maladies rares du foie                        <\/h3>\n                        <p class=\"accordian-summary hidden\"><\/p>\n                    <\/div>\n                <\/div><!--Accordion info section over here -->\n\n                <div class=\"col-xl-12 col-lg-12 col-md-12 col-sm-12\">\n                    <div class=\"accordion accor-group\" id=\"accordionExample646203835\">\n                         \n                                    <!-- Accordion with Expandable panel & FAQs -->\n                                    <div class=\"accordion-item accor-item\">\n                                        <p  role=\"heading\" aria-level=\"3\" class=\"accordion-header accor-head-para\" id=\"expHeading0\">\n                                            <button class=\"accordion-button accor-btn collapsed\" type=\"button\" data-bs-toggle=\"collapse\" data-bs-target=\"#expHeading6462038350\" aria-expanded=\"false\" aria-controls=\"expHeading6462038350\">\n                                                \u00a0Cholestase intrah\u00e9patique familiale progressive (PFIC)                                            <\/button>\n                                        <\/p>\n                                        <div id=\"expHeading6462038350\" class=\"accordion-collapse collapse \" aria-labelledby=\"expHeading0\" data-bs-parent=\"#accordionExample646203835\">\n                                            <div class=\"accordion-body\">\n                                                <div class=\"accordion-data\">\n                                                    <p><p>La PFIC est une maladie h\u00e9r\u00e9ditaire rare chez l&#8217;enfant dans laquelle l&#8217;organisme est incapable d&#8217;\u00e9vacuer la bile du foie m\u00eame si les grandes voies biliaires sont ouvertes (cholestase). L\u2019accumulation d&#8217;acides biliaires qui en r\u00e9sulte dans les cellules du foie provoque une maladie du foie et des sympt\u00f4mes h\u00e9patiques. L\u2019incidence estim\u00e9e de la PFIC est de 1 cas sur 100 000 naissances. Le sympt\u00f4me le plus invalidant de la PFIC est le prurit (d\u00e9mangeaisons), qui peut atteindre un stade si grave qu\u2019il entra\u00eene une mutilation de la peau, une perte de sommeil, de l\u2019irritabilit\u00e9, un d\u00e9ficit de l\u2019attention et une alt\u00e9ration des performances scolaires. Jusqu\u2019\u00e0 80 % des patients atteints de PFIC souffrent de prurit s\u00e9v\u00e8re.<\/p>\n<\/p>\n                                                    <div class=\"cta-block hidden\" >\n                                                                                                            <\/div>\n                                                <\/div>\n                                            <\/div>\n                                        <\/div>\n                                    <\/div>\n                                                <\/div>\n                <\/div> \n            <\/div>\n        <\/div>\n    <\/section>\n    <!--Accordion Cards End Here-->\n\n\n<!-- Ipsen Content Overlay Start -->\n<section class=\"two-col-image-section\">\n    <div class=\"container two-img-section-innner\"  >\n        <div class=\"content-overlay-inner bg-color-enabled\" style=\"background-color:#EDEBE4\">\n                <!-- Ipsen Content Overlay Variation Two Start -->\n      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                                          <div class=\"heading-dash\">\n                                        <span class=\"dash\"><\/span>\n                                        <h4 role=\"heading\" aria-level=\"2\" class=\"heading-para-right heading-four\">\n                                            Les essais cliniques d&#8217;Ipsen dans les maladies rares                                        <\/h4>\n                                    <\/div>\n                                                                                                                        <p class=\"section-para \">\n                                        Nous travaillons sans rel\u00e2che pour amener de nouveaux traitements au stade d&#8217;essai clinique et ainsi r\u00e9pondre aux besoins m\u00e9dicaux non satisfaits des patients.                                    <\/p>\n                                                                                                <a \n                            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two-img-section-innner\"  >\n        <div class=\"content-overlay-inner bg-color-enabled\" style=\"background-color:#EDEBE4\">\n                <!-- Ipsen Content Overlay Variation Two Start -->\n                <div class=\"row section-bg-reverse\">\n                    <div class=\"col-xs-12 col-sm-12 col-md-6 column-1 order-2\">\n                        <div id=\"image-carousel\" class=\"img-video-section carousel\" data-bs-ride=\"carousel\">\n                            <div class=\"carousel-inner\">\n                                                                        <div class=\"carousel-item active\">\n                                            <img decoding=\"async\" src=\"https:\/\/www.ipsen.com\/be\/wp-content\/uploads\/sites\/7\/2025\/07\/Module-9-\u2013-Rare-disease-pipeline_191209_0722.jpg\" alt=\"Media Image\" >\n                                        <\/div>\n                                                            <\/div>\n                                                    <\/div>\n                    <\/div>\n                    <div class=\" col-xs-12 col-sm-12 col-md-6 column-2 order-1\">\n                        <div class=\" section-content\">\n                                                                <div class=\"heading-dash\">\n                                        <span class=\"dash\"><\/span>\n                                        <h4 role=\"heading\" aria-level=\"2\" class=\"heading-para-right heading-four\">\n                                            Notre portefeuille de produits en R&amp;D                                        <\/h4>\n                                    <\/div>\n                                                                                                                        <p class=\"section-para \">\n                                        Aux c\u00f4t\u00e9s de partenaires qui partagent notre vision, nous mettons tout en \u0153uvre pour am\u00e9liorer le quotidien des patients et de leurs aidants. Nos \u00e9quipes s&#8217;emploient jour apr\u00e8s jour \u00e0 apporter les meilleures options de traitement possibles aux patients&#8230;                                    <\/p>\n                                                                                                <a \n                                            role=\"link\" \n                                            class=\"btn-link section-btn cta-visible\" \n                                            href=\"https:\/\/www.ipsen.com\/fr\/portefeuille-de-produits-r-et-d\/\" \n                                            aria-label=\"\" \n                                            target=\"_blank\"\n                                        >\n                                            En savoir plus                                         <\/a>    \n                                    <\/button>\n                                                    <\/div>\n                    <\/div>\n                    <!-- Ipsen Content Overlay Variation Two End -->\n                <\/div>\n        <!-- Ipsen Content Overlay Variation two End -->\n                <\/div>\n    <\/div>\n<\/section>\n<!-- Ipsen Content Overlay End -->","protected":false},"excerpt":{"rendered":"","protected":false},"author":2,"featured_media":0,"parent":0,"menu_order":0,"comment_status":"closed","ping_status":"closed","template":"","meta":{"_acf_changed":false,"footnotes":""},"class_list":["post-101","page","type-page","status-publish","hentry","entry"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v26.8 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>Des traitements pionniers dans les maladies rares<\/title>\n<meta name=\"description\" content=\"Ipsen se focalise sur le d\u00e9veloppement de m\u00e9dicaments innovants dans trois principales cat\u00e9gories de maladies rares : les maladies osseuses et endocriniennes, neuromusculaires et h\u00e9patiques.\" \/>\n<meta name=\"robots\" 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