{"id":43,"date":"2024-10-29T06:54:14","date_gmt":"2024-10-29T04:54:14","guid":{"rendered":"https:\/\/www.ipsen.com\/uk\/?page_id=43"},"modified":"2026-02-26T16:25:50","modified_gmt":"2026-02-26T14:25:50","slug":"rare-disease","status":"publish","type":"page","link":"https:\/\/www.ipsen.com\/uk-ireland\/rare-disease\/","title":{"rendered":"Rare Disease"},"content":{"rendered":"    <section class=\"sub-page-banner \" style=\"background: linear-gradient(93.64deg, #224A81 2.99%, #2797D3 97.01%)\">\n        <div class=\"container content-sec\">\n            <nav aria-label=\"breadcrumb\">\n            <ol class=\"breadcrumb\">\n                <li class=\"breadcrumb-item\"><a href=\"https:\/\/www.ipsen.com\/uk-ireland\">Home<\/a><\/li><li class=\"breadcrumb-item active\"><a href=\"#\" aria-current=\"page\">Science &#8211; Rare Disease<\/a><\/li><\/ol>\n            <\/nav>            <h1 class=\"visible\">Rare Disease<\/h1>\n            <p class=\"hidden\"><\/p>\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 \">\n        <div class=\"response-inner\" style=\"background-image: url('')\">\n            <div class=\"row \">\n                <div class=\"col-xs-12 col-sm-12 col-md-6\">\n                                                    <div class=\"img-video-section\">\n                                <img decoding=\"async\" \n                                    src=\"https:\/\/www.ipsen.com\/uk-ireland\/wp-content\/uploads\/sites\/19\/2025\/01\/13322-IPS-UKI-Website-TA-Images-6_.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                                    Rare Diseases                                <\/h4>\n                                                    <\/div>\n                                                <p><strong>In the EU, a disease is considered to be a rare disease when the number of people affected is no more than 5 person in 10,000.<sup>i<\/sup>\u00a0Ipsen is committed to translating scientific progress into clinically meaningful medicines for people suffering from rare conditions.<\/strong><\/p>\n<p><sup>i<\/sup>European Union: EU Research on Rare Diseases. Accessed June 2025.<\/p>\n                            <a \n                            aria-label=\" about Rare Diseases\"  \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\n<div class=\"wp-block-group\"><div class=\"wp-block-group__inner-container is-layout-constrained wp-block-group-is-layout-constrained\">\n<h3 class=\"wp-block-heading\">Addressing areas of unmet need<\/h3>\n\n\n\n<p>Ipsen\u2019s work in rare diseases currently focuses on how we can address areas of high unmet need for people affected by hormone and metabolic imbalances and rare liver disorders. We strive to understand the challenges faced by those living with rare diseases, learning from their experiences and integrating their insights into our efforts throughout the development process.<\/p>\n<\/div><\/div>\n\n\n\n    <!--Accordion Cards Start Here-->\n    <section class=\"accordion bg-color-disabled\"  data-according-id=\"283134932\">\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                            Areas we work in within rare diseases                        <\/h3>\n                        <p class=\"accordian-summary visible\">Ipsen continues to develop innovative medicines in rare diseases to address conditions with high unmet needs, with specific investment in pituitary pathologies and liver disorders.<\/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=\"accordionExample283134932\">\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=\"faqHeading0\">\n                                            <button class=\"accordion-button accor-faq-btn collapsed\" type=\"button\" data-bs-toggle=\"collapse\" data-bs-target=\"#faqHeading2831349320\" aria-expanded=\"false\" aria-controls=\"faqHeading2831349320\">\n                                                Acromegaly                                            <\/button>\n                                        <\/p>\n                                        <div id=\"faqHeading2831349320\" class=\"accordion-collapse collapse \" aria-labelledby=\"faqHeading0\" data-bs-parent=\"#accordionExample283134932\">\n                                            <div class=\"accordion-body\">\n                                                <div class=\"accordion-data\">\n                                                    <p><p><strong>Acromegaly is a rare condition caused by excess production of growth hormone by the pituitary gland, resulting in body tissues and bones growing more quickly.<sup>i<\/sup><\/strong><\/p>\n<p>&nbsp;<\/p>\n<p>About 2\u20134 people in a million develop acromegaly each year in the U.K.<sup>ii<\/sup>\u00a0The condition can cause a wide range of symptoms, however acromegaly literally means \u2018enlarged extremities\u2019 with large hands and feet a typical feature, however there are many other symptoms which usually progress slowly over time, including thickened vocal cords, facial changes (e.g. thickened lips and nose) and enlarged tongue.<sup>ii<\/sup><\/p>\n<p>&nbsp;<\/p>\n<p><sup>i<\/sup>NHS: Acromegaly. Accessed June 2025.<br \/>\n<sup>ii<\/sup>Payne J.: Acromegaly. Accessed June 2025.<\/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=\"faqHeading1\">\n                                            <button class=\"accordion-button accor-faq-btn collapsed\" type=\"button\" data-bs-toggle=\"collapse\" data-bs-target=\"#faqHeading2831349321\" aria-expanded=\"false\" aria-controls=\"faqHeading2831349321\">\n                                                Precocious puberty                                            <\/button>\n                                        <\/p>\n                                        <div id=\"faqHeading2831349321\" class=\"accordion-collapse collapse \" aria-labelledby=\"faqHeading1\" data-bs-parent=\"#accordionExample283134932\">\n                                            <div class=\"accordion-body\">\n                                                <div class=\"accordion-data\">\n                                                    <p><p><strong>Precocious puberty refers to the onset of puberty at an abnormally early age; before age 8 in girls, and 9 in boys.<sup>i<\/sup>\u00a0It is not always clear what causes early puberty, however it can be a familial tendency.<sup>i<\/sup><\/strong><\/p>\n<p>&nbsp;<\/p>\n<p>Medical treatment for early puberty aims to address any underlying cause or reduce hormone levels to pause sexual development for a few years to avoid emotional or physical issues.<sup>i<\/sup><\/p>\n<p>&nbsp;<\/p>\n<p><sup>i<\/sup>NHS: Early or Delayed Puberty. Accessed June 2025.<\/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=\"faqHeading2\">\n                                            <button class=\"accordion-button accor-faq-btn collapsed\" type=\"button\" data-bs-toggle=\"collapse\" data-bs-target=\"#faqHeading2831349322\" aria-expanded=\"false\" aria-controls=\"faqHeading2831349322\">\n                                                Progressive familial intrahepatic cholestasis                                            <\/button>\n                                        <\/p>\n                                        <div id=\"faqHeading2831349322\" class=\"accordion-collapse collapse \" aria-labelledby=\"faqHeading2\" data-bs-parent=\"#accordionExample283134932\">\n                                            <div class=\"accordion-body\">\n                                                <div class=\"accordion-data\">\n                                                    <p><p><strong>Progressive familial intrahepaftic cholestasis (PFIC) is a rare inherited condition in which bile is unable to drain from the liver into the gastrointestinal tract.<sup>i<\/sup>\u00a0As a result, bile acid builds up in liver cells (cholestasis) causing scaring and symptoms.<sup>i<\/sup>\u00a0The global prevalence of PFIC is estimated between 1 in 50,000 to 1 in 100,000 live births.<sup>i<\/sup><\/strong><\/p>\n<p>&nbsp;<\/p>\n<p>The most bothersome symptom of PFIC is pruritus (itching), which may be so severe that it leads to skin mutilation, loss of sleep, irritability, poor attention and impaired school performance.<sup>ii<\/sup><\/p>\n<p>&nbsp;<\/p>\n<p><sup>i<\/sup>NICE Guidance HST17: 2. The Condition. Accessed June 2025.<br \/>\n<sup>ii<\/sup>Srivastava A.\u00a0<em>J Clin Exp Hepatol<\/em>. 2014;4( 1 ):25\u201336.<\/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<div class=\"wp-block-group\"><div class=\"wp-block-group__inner-container is-layout-constrained wp-block-group-is-layout-constrained\">\n<p>ALLSC-GB-001301<\/p>\n\n\n\n<p>January 2026<\/p>\n<\/div><\/div>\n","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-43","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>Rare disease<\/title>\n<meta name=\"description\" content=\"Ipsen is dedicated to developing innovative medicines for rare diseases, focusing on three main areas: bone and endocrine, neuromuscular and liver.\" \/>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/www.ipsen.com\/uk-ireland\/rare-disease\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" 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