{"id":35,"date":"2024-01-17T13:52:03","date_gmt":"2024-01-17T11:52:03","guid":{"rendered":"https:\/\/www.ipsen.com\/it\/?page_id=35"},"modified":"2026-07-27T16:24:37","modified_gmt":"2026-07-27T14:24:37","slug":"malattie-rare","status":"publish","type":"page","link":"https:\/\/www.ipsen.com\/it\/malattie-rare\/","title":{"rendered":"Malattie Rare"},"content":{"rendered":"    <section class=\"sub-page-banner bg-img-enabled  banner-no-filter\" style=\"background-image:url(https:\/\/www.ipsen.com\/it\/wp-content\/uploads\/sites\/9\/2025\/07\/Health-Rare-Disease.jpg)\">\n\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:\/\/www.ipsen.com\/it\">Home<\/a><\/li><li class=\"breadcrumb-item active\"><a href=\"#\" aria-current=\"page\">Aree Terapeutiche &#8211; Malattie Rare<\/a><\/li><\/ol>\n            <\/nav>            <h1 class=\"visible banner-font-color-white\">Malattie Rare<\/h1>\n            <p class=\"visible banner-font-color-white\">Sviluppiamo terapie per chi vive con malattie rare e ultra-rare<\/p>\n        <\/div>\n    <\/section>\n\n\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            <div class=\"row \">\n                <div class=\"col-xs-12 col-sm-12 col-md-6 column-1 \">\n                                                                <div class=\"content-overlay-image\">\n                                        <img decoding=\"async\" src=\"https:\/\/www.ipsen.com\/it\/wp-content\/uploads\/sites\/9\/2024\/03\/Our-Commitment-to-Rare-Disease_Savannah_card-700x420pix.jpg\" alt=\"Media Image\">\n                                    <\/div>\n                                            <\/div>\n\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                                    Il nostro impegno nelle malattie rare                                <\/h4>\n                            <\/div>\n                                                \n                                                \n                                                    <p class=\"section-para \">\n                                Il 94% delle malattie rare non ha ancora alcuna opzione di trattamento. Oggi i nostri team sono pi\u00f9 determinati che mai a cambiare questa difficile realt\u00e0 facendo leva sulla nostra expertise in ambito scientifico e sulla presenza globale.                            <\/p>\n                                                \n                                            <\/div>\n                <\/div>\n            <\/div>\n            \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\/it\/wp-content\/uploads\/sites\/9\/2024\/03\/Module-2_Our-commitment-ro-rare-disease-Brief-about-rare-disease_20201010_5529-HD_03-scaled-1.jpg\" \n                                    alt=\"Ascoltiamo i bisogni di chi vive con una malattia rara\" \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                                    Ascoltiamo i bisogni di chi vive con una malattia rara                                <\/h4>\n                                                    <\/div>\n                                                <p>Ci dedichiamo con passione a comprendere i bisogni e le sfide che le persone con malattie rare affrontano ogni giorno. Impariamo dalle loro esperienze e integriamo i loro spunti e le loro storie in tutte le nostre attivit\u00e0 di sviluppo medico-scientifico, dall\u2019inizio alla fine del processo. Questa modalit\u00e0 di azione ci ha permesso di fare avanzare la ricerca e lo sviluppo di nuovi trattamenti per malattie rare in ambito metabolico, endocrino, gastrointestinale e neuromuscolare.<\/p>\n                            <a \n                            aria-label=\"Scopri di pi\u00f9 about Ascoltiamo i bisogni di chi vive con una malattia rara\"  \n                                role=\"link\" \n                                class=\"btn-link section-btn cta-visible\"\n                                href=\"https:\/\/www.ipsen.com\/it\/iniziative\/\" \n                                target=\"_self\"\n                            > \n                                Scopri di pi\u00f9 \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                                Mettiamo in discussione i paradigmi terapeutici                             <\/h3>\n                            <p class=\"text-graph-para charcoal-grey visible\">Nel 2023 Ipsen ha annunciato i risultati di uno studio clinico di Fase III con importanti implicazioni per il trattamento di una malattia epatica autoimmune, la colangite biliare primitiva (PBC) che provoca sintomi debilitanti.<\/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=\"Precedente\" tabindex = \"-1\" class=\"glider-graphic-card-prev\"><span><\/span><\/button>\n                                                    <button aria-hidden=\"true\" aria-label=\"Prossimo\" tabindex = \"-1\" 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\/it\/wp-content\/uploads\/sites\/9\/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\">Sfide uniche richiedono soluzioni uniche <\/h4>\n                                                                        <\/div>\n                                                                                                                                <div class=\"text-graph-desc\">\n                                                                    <p>Nel 2023 abbiamo ulteriormente ampliato il nostro portfolio terapeutico nell&#8217;ambito delle malattie rare con terapie promettenti e molecole sperimentali per le malattie epatiche rare dei bambini e degli adulti.<sup>1<\/sup><\/p>\n                                                                <\/div>\n                                                            <\/div>\n                                                        <\/div>\n                                                        <!-- Graphic Card With OLD layout end here -->\n                                                                                            <\/div>\n\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\/it\/wp-content\/uploads\/sites\/9\/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\">Una sfida senza sosta per rispondere ai bisogni dei pazienti<\/h4>\n                                                                        <\/div>\n                                                                                                                                <div class=\"text-graph-desc\">\n                                                                    <p>Il 94% delle malattie rare non dispone di un trattamento approvato.<sup>2<\/sup><\/p>\n                                                                <\/div>\n                                                            <\/div>\n                                                        <\/div>\n                                                        <!-- Graphic Card With OLD layout end here -->\n                                                                                            <\/div>\n\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\/it\/wp-content\/uploads\/sites\/9\/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\">Rari ma non soli<\/h4>\n                                                                        <\/div>\n                                                                                                                                <div class=\"text-graph-desc\">\n                                                                    <p>Nel mondo oltre 350-400 milioni di persone vivono con una delle 7.000 malattie classificate come rare.<sup>3<\/sup><\/p>\n                                                                <\/div>\n                                                            <\/div>\n                                                        <\/div>\n                                                        <!-- Graphic Card With OLD layout end here -->\n                                                                                            <\/div>\n\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\/it\/wp-content\/uploads\/sites\/9\/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\">Portiamo innovazione ai pazienti con malattie rare <\/h4>\n                                                                        <\/div>\n                                                                                                                                <div class=\"text-graph-desc\">\n                                                                    <p>La nostra pipeline nell&#8217;ambito delle malattie rare \u00e8 pi\u00f9 che raddoppiata dal 2020 a oggi.<\/p>\n                                                                <\/div>\n                                                            <\/div>\n                                                        <\/div>\n                                                        <!-- Graphic Card With OLD layout end here -->\n                                                                                            <\/div>\n\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\n    <!--Accordion Cards Start Here-->\n    <section class=\"accordion \"  data-according-id=\"1298362962\">        \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 hidden heading-three\" role=\"heading\" aria-level=\"2\">\n                                                    <\/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=\"accordionExample1298362962\">\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 \"  data-according-id=\"1698337234\">        \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                            Malattie Rare del Fegato                        <\/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=\"accordionExample1698337234\">\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=\"#expHeading16983372340\" aria-expanded=\"false\" aria-controls=\"expHeading16983372340\">\n                                                \u00a0Colestasi intraepatica familiare progressiva (PFIC)                                            <\/button>\n                                        <\/p>\n                                        <div id=\"expHeading16983372340\" class=\"accordion-collapse collapse \" aria-labelledby=\"expHeading0\" data-bs-parent=\"#accordionExample1698337234\">\n                                            <div class=\"accordion-body\">\n                                                <div class=\"accordion-data\">\n                                                    <p>La PFIC \u00e8 una condizione ereditaria rara che impedisce ai bambini di drenare la bile dal fegato anche se i dotti biliari pi\u00f9 grandi sono aperti (colestasi). Di conseguenza, gli acidi biliari si accumulano nelle cellule epatiche causando sintomi e malattie nel fegato. Si stima che la PFIC abbia un\u2019incidenza pari a 1 su 100.000 nati vivi. Il sintomo pi\u00f9 debilitante della PFIC \u00e8 il prurito che pu\u00f2 essere cos\u00ec grave da portare a mutilazioni cutanee, insonnia, irritabilit\u00e0, deficit di attenzione e peggioramento del rendimento scolastico. Fino all\u201980% dei pazienti affetti da PFIC soffre di prurito grave.<sup>4<\/sup><\/p>\n                                                    <div class=\"cta-block hidden\" style=\"text-align: left;\">\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<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            <div class=\"row \">\n                <div class=\"col-xs-12 col-sm-12 col-md-6 column-1 \">\n                                                                <div class=\"content-overlay-image\">\n                                        <img decoding=\"async\" src=\"https:\/\/www.ipsen.com\/it\/wp-content\/uploads\/sites\/9\/2025\/07\/Module-2-\u2013-Brief-about-neuroscience_IPSEN_Production_Signes_060.jpg\" alt=\"Media Image\">\n                                    <\/div>\n                                            <\/div>\n\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                                    Gli studi clinici di Ipsen nelle malattie rare                                 <\/h4>\n                            <\/div>\n                                                \n                                                \n                                                    <p class=\"section-para \">\n                                Ci impegniamo senza sosta nella ricerca e sviluppo di nuovi trattamenti che offrano risposte a bisogni clinici non ancora soddisfatti.                            <\/p>\n                                                \n                                                    <a \n                                role=\"link\" \n                                class=\"btn-link section-btn cta-visible\" \n                                href=\"https:\/\/www.ipsen.com\/clinical-trials\/\" \n                                aria-label=\"\" \n                                target=\"_blank\"\n                            >\n                                Scopri di pi\u00f9                            <\/a>    \n                                            <\/div>\n                <\/div>\n            <\/div>\n            \n        <\/div>\n    <\/div>\n<\/section>\n\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            <div class=\"row section-bg-reverse\">\n                <div class=\"col-xs-12 col-sm-12 col-md-6 column-1 order-2\">\n                                                                <div class=\"content-overlay-image\">\n                                        <img decoding=\"async\" src=\"https:\/\/www.ipsen.com\/it\/wp-content\/uploads\/sites\/9\/2025\/07\/Module-9-\u2013-Rare-disease-pipeline_191209_0722.jpg\" alt=\"Media Image\">\n                                    <\/div>\n                                            <\/div>\n\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                                    La nostra pipeline                                <\/h4>\n                            <\/div>\n                                                \n                                                \n                                                    <p class=\"section-para \">\n                                Collaboriamo con chi come noi ha lo sguardo rivolto al futuro per generare innovazione per sviluppare terapie  che in grado di migliorare la vita di chi affronta malattie complesse.                            <\/p>\n                                                \n                                                    <a \n                                role=\"link\" \n                                class=\"btn-link section-btn cta-visible\" \n                                href=\"https:\/\/www.ipsen.com\/pipeline\/\" \n                                aria-label=\"\" \n                                target=\"_blank\"\n                            >\n                                Scopri di pi\u00f9                            <\/a>    \n                                            <\/div>\n                <\/div>\n            <\/div>\n            \n        <\/div>\n    <\/div>\n<\/section>\n\n\n    <!--Accordion Cards Start Here-->\n    <section class=\"accordion \"  data-according-id=\"1568371451\">        \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 hidden heading-three\" role=\"heading\" aria-level=\"2\">\n                                                    <\/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=\"accordionExample1568371451\">\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=\"#expHeading15683714510\" aria-expanded=\"false\" aria-controls=\"expHeading15683714510\">\n                                                Riferimenti bibliografici                                            <\/button>\n                                        <\/p>\n                                        <div id=\"expHeading15683714510\" class=\"accordion-collapse collapse \" aria-labelledby=\"expHeading0\" data-bs-parent=\"#accordionExample1568371451\">\n                                            <div class=\"accordion-body\">\n                                                <div class=\"accordion-data\">\n                                                    <ol>\n<li><a href=\"\/?p=38553\"><strong>Ipsen nella colangite biliare primitiva<\/strong><\/a>. Ultimo accesso maggio 2024<\/li>\n<li>Ref. C. P. Austin et al., Clin Transl Sci. 2018 Jan; 11(1): 21\u201327<\/li>\n<li>Ref. T. Adachi et al., Int J Environ Res Public Health. 2023 Mar; 20(6): 4732<\/li>\n<li>Ref. M.Gunaydin Hepatic Medicine: Evidence and Research 2018:10 95\u2013104<\/li>\n<\/ol>\n                                                    <div class=\"cta-block hidden\" style=\"text-align: left;\">\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<!--Alert Card Layout Start Here-->\n<section class=\"alertcard-section\">\n      <div class=\"container\">\n        <!--Alert Box HTML Start Here-->\n        <div class=\"alert-box general-box\">\n                    <div class=\"info-icon\">\n              <span class=\"info-circle\"><img decoding=\"async\" src=\"https:\/\/www.ipsen.com\/it\/wp-content\/themes\/ipsen-main\/dist\/images\/alert-info-icon.png\" alt=\"Alert\"\/><\/i>\n            <\/div>\n                    <div class=\"info-contents\">\n              <h4 role=\"heading\" aria-level=\"2\" class=\"info-head heading-four heading-four\">\n                                <\/h4>\n              <p class=\"info-sub-head-para hidden heading-para\">\n                                <\/p>\n              <div class=\"alert-content visible\">\n                  <p>Le informazioni presenti in questa pagina non sostituiscono il parere medico<\/p>\n              <\/div>\n          <\/div>\n        <\/div>\n        <!--Alert Box HTML Start Here-->\n      <\/div>\n    <\/section>\n<!--Alert Card Layout End 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