{"id":41,"date":"2024-11-27T07:33:34","date_gmt":"2024-11-27T05:33:34","guid":{"rendered":"https:\/\/www.ipsen.com\/middleeast\/?page_id=41"},"modified":"2025-10-07T07:31:23","modified_gmt":"2025-10-07T05:31:23","slug":"rare-disease","status":"publish","type":"page","link":"https:\/\/www.ipsen.com\/uae-gulf\/rare-disease\/","title":{"rendered":"Rare Disease"},"content":{"rendered":"    <section class=\"sub-page-banner bg-img-enabled  banner-no-filter\" style=\"background-image:url(https:\/\/www.ipsen.com\/uae-gulf\/wp-content\/uploads\/sites\/18\/2025\/10\/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\/uae-gulf\">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 banner-font-color-white\">Rare Disease<\/h1>\n            <p class=\"visible banner-font-color-white\">Developing treatment options for those with rare and ultra-rare diseases.<\/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 One Start -->\n                <div class=\"row \">\n                    <div class=\"col-xs-12 col-sm-12 col-md-6 column-1 \">\n                                                        <div class=\"no-carousel\">\n                                    <img decoding=\"async\" src=\"https:\/\/www.ipsen.com\/uae-gulf\/wp-content\/uploads\/sites\/18\/2025\/01\/Our-Commitment-to-Rare-Disease_Savannah_card-700x420pix.jpg\" alt=\"\">\n                                <\/div>\n                                        <\/div>\n                    <div class=\"col-xs-12 col-sm-12 col-md-6 column-2 \">\n                        <div class=\"section-content\">\n                                                                    <p class=\"section-head-two heading-four\">\n                                            Our commitment to rare disease                                        <\/p>\n                                                                    <p class=\"section-para \">\n                                            Ninety-four percent of rare medical conditions still have no treatment. Today our Rare Disease teams are more committed than ever to changing that. We leverage scientific expertise, our global presence, and a robust external innovation strategy, plus first-in-the-world clinical trials to develop life-changing rare disease therapeutics.                                        <\/p>\n                                                    <\/div>\n                    <\/div>\n                <\/div>\n        <!-- Ipsen Content Overlay Variation One 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\/uae-gulf\/wp-content\/uploads\/sites\/18\/2025\/01\/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                                    Listening to people living with rare diseases                                 <\/h4>\n                                                    <\/div>\n                                                <p>With unwavering focus on patient care, we strive to understand the challenges faced by those living with rare diseases. We learn from their experiences and integrate their insights into our efforts throughout the development process. As a result, we are gaining momentum toward finding\u00a0treatments for rare diseases including musculoskeletal (bone), metabolic, endocrine, gastrointestinal (liver) and neuromuscular disorders.<\/p>\n                            <a \n                            aria-label=\"Learn more about Listening to people living with rare diseases \"  \n                                role=\"link\" \n                                class=\"btn-link section-btn cta-visible\"\n                                href=\"https:\/\/www.ipsen.com\/uae-gulf\/patients\/\" \n                                target=\"_self\"\n                            > \n                                Learn more \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                                Challenging treatment paradigms                             <\/h3>\n                            <p class=\"text-graph-para charcoal-grey visible\">In 2023, Ipsen will be reporting on a Phase III clinical trial with critical implications for treating an autoimmune liver disease, Primary Biliary Cholangitis (PBC), and its debilitating symptoms.<\/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=\"Previous\" class=\"glider-graphic-card-prev\"><span><\/span><\/button>\n                                                    <button aria-hidden=\"true\" aria-label=\"Next\" 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\/uae-gulf\/wp-content\/uploads\/sites\/18\/2025\/10\/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\">Unique challenges require unique solutions <\/h4>\n                                                                                <\/div>\n                                                                                                                                                <div class=\"text-graph-desc\">\n                                                                            <p><p>In 2023, we further strengthened our Rare Disease portfolio with promising treatments and investigational assets for pediatric and adult rare liver diseases.<\/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\/uae-gulf\/wp-content\/uploads\/sites\/18\/2025\/10\/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\">An unwavering fight to meet patient needs<\/h4>\n                                                                                <\/div>\n                                                                                                                                                <div class=\"text-graph-desc\">\n                                                                            <p><p>94% of rare diseases still lack approved treatments.<\/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\/uae-gulf\/wp-content\/uploads\/sites\/18\/2025\/10\/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 but not alone<\/h4>\n                                                                                <\/div>\n                                                                                                                                                <div class=\"text-graph-desc\">\n                                                                            <p><p>350-400 million people are impacted by one of 7,000 rare diseases globally.<\/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\/uae-gulf\/wp-content\/uploads\/sites\/18\/2025\/10\/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\">Innovating for patients with rare diseases <\/h4>\n                                                                                <\/div>\n                                                                                                                                                <div class=\"text-graph-desc\">\n                                                                            <p><p>The rare disease clinical pipeline has more than doubled since 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                        Continuous pioneering in rare disease                    <\/h3>\n                <\/div>\n               \n                <!-- If description is enabled then it's visible -->\n                <div class=\"text-parbase visible\">\n                    <p>A core part of our strategy, Ipsen has focused on developing transformational treatments for rare diseases for many years.<\/p>\n                <\/div>\n\n                <!-- If button is enabled then it's visible -->\n                \n            <\/div>\n        <\/div>\n    <\/section>\n\n\n\n    <!--Accordion Cards Start Here-->\n    <section class=\"accordion bg-color-disabled\"  data-according-id=\"1821436650\">\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                            Bone disorders                        <\/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=\"accordionExample1821436650\">\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=\"#expHeading18214366500\" aria-expanded=\"false\" aria-controls=\"expHeading18214366500\">\n                                                Fibrodysplasia ossificans progressiva (FOP)                                            <\/button>\n                                        <\/p>\n                                        <div id=\"expHeading18214366500\" class=\"accordion-collapse collapse \" aria-labelledby=\"expHeading0\" data-bs-parent=\"#accordionExample1821436650\">\n                                            <div class=\"accordion-body\">\n                                                <div class=\"accordion-data\">\n                                                    <p><p>FOP causes permanent and continuous abnormal new bone formation in soft and connective tissues, like muscles, tendons and ligaments, a process known as heterotopic ossification (HO). It is an ultra-rare genetic disorder, characterized by inflammatory flare-ups that begin in childhood HO is irreversible and leads to severe functional limitations in mobility, progressive and cumulative disability, and shortened life expectancy. On average, patients are diagnosed at the age of 5, and their median life expectancy is 56. Around 900 people worldwide are currently living with an FOP diagnosis. Ipsen is harnessing novel mechanisms of action through the development of two investigational assets to bring potentially transformative treatments to those living with 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=\"1211285787\">\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                            Growth disorders                        <\/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=\"accordionExample1211285787\">\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=\"#expHeading12112857870\" aria-expanded=\"false\" aria-controls=\"expHeading12112857870\">\n                                                Acromegaly                                            <\/button>\n                                        <\/p>\n                                        <div id=\"expHeading12112857870\" class=\"accordion-collapse collapse \" aria-labelledby=\"expHeading0\" data-bs-parent=\"#accordionExample1211285787\">\n                                            <div class=\"accordion-body\">\n                                                <div class=\"accordion-data\">\n                                                    <p><p>Acromegaly is a rare disorder caused by a benign (noncancerous) tumor of the pituitary gland, called an adenoma, that promotes too much growth hormone, or GH, to be released into the bloodstream. As a consequence, the body makes more of another hormone, insulin-like growth factor 1 (IGF-1).<\/p>\n<p>&nbsp;<\/p>\n<p>Normally, IGF-1 works with GH to help control growth of many tissues in the body. With too much IGF-1 and GH, the body can undergo abnormal growth of bones, cartilage and other body tissues. While the more outward signs are enlarged hands, feet and face, more serious problems may also develop.<\/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=\"#expHeading12112857871\" aria-expanded=\"false\" aria-controls=\"expHeading12112857871\">\n                                                Growth Hormone Deficiency                                            <\/button>\n                                        <\/p>\n                                        <div id=\"expHeading12112857871\" class=\"accordion-collapse collapse \" aria-labelledby=\"expHeading1\" data-bs-parent=\"#accordionExample1211285787\">\n                                            <div class=\"accordion-body\">\n                                                <div class=\"accordion-data\">\n                                                    <p><p>Pediatric Growth Hormone Deficiency (PGHD) and Adult-onset Adult Growth Hormone Deficiency (AGHD) occur when a person is unable to produce enough growth hormone (GH), or use it effectively. GH plays a critical role in helping bodies grow and develop, including improving muscle metabolism, growing bones, and breaking down fats. When levels of GH are too low, there may be an impact on growth and bodily functions.<\/p>\n<p>&nbsp;<\/p>\n<p>PGHD may be present at birth or develop at any time during childhood or adolescence. Both can be caused by:<\/p>\n<ul>\n<li>A tumor in and around the pituitary gland<\/li>\n<li>Damage or an abnormality in the hypothalamus<\/li>\n<li>Damage to the pituitary by infection, blood vessel disease, severe head injury, or radiation treatment.<\/li>\n<\/ul>\n<p>Damage to the pituitary gland may affect the production of one or more pituitary hormones, including GH.<\/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=\"#expHeading12112857872\" aria-expanded=\"false\" aria-controls=\"expHeading12112857872\">\n                                                Turner Syndrome-Associated Short Stature                                            <\/button>\n                                        <\/p>\n                                        <div id=\"expHeading12112857872\" class=\"accordion-collapse collapse \" aria-labelledby=\"expHeading2\" data-bs-parent=\"#accordionExample1211285787\">\n                                            <div class=\"accordion-body\">\n                                                <div class=\"accordion-data\">\n                                                    <p><p>Turner Syndrome (TS) is a genetic condition that occurs when one of a girl\u2019s two X chromosomes is absent or incomplete. The most common feature of TS \u2013 affecting nearly all girls with the condition \u2013 is short stature. This is because the missing or incomplete X chromosome contains a gene that helps girls grow to a normal height. From birth through their teen years, girls with TS grow more slowly than unaffected children. The average adult height of women with TS is about 4\u20198\u201d, while unaffected women average about 5\u20194\u201d.<\/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=\"#expHeading12112857873\" aria-expanded=\"false\" aria-controls=\"expHeading12112857873\">\n                                                Chronic Kidney Disease-Associated Growth Disease                                            <\/button>\n                                        <\/p>\n                                        <div id=\"expHeading12112857873\" class=\"accordion-collapse collapse \" aria-labelledby=\"expHeading3\" data-bs-parent=\"#accordionExample1211285787\">\n                                            <div class=\"accordion-body\">\n                                                <div class=\"accordion-data\">\n                                                    <p><p>Chronic Kidney Disease (CKD) is a rare disease that causes loss of kidney function over a period of months or years. Most young people with CKD produce as much growth hormone (GH) as anyone their age, but their kidneys do not filter waste from their blood as well as they should. Because so much waste is trapped in their blood, their bodies cannot use GH effectively. Typically, GH signals insulin-like growth factor (IGF-1) to travel to muscles, organs and bones, instructing the bones to grow and helping improve muscle metabolism. CKD causes GH and IGF to not connect properly. This can lead to slowed or stopped growth, as well as improper development of muscles, organs and bones.<\/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=\"#expHeading12112857874\" aria-expanded=\"false\" aria-controls=\"expHeading12112857874\">\n                                                Severe Primary Insulin-like Growth Factor (IGF-1) Deficiency (SPIGFD)                                            <\/button>\n                                        <\/p>\n                                        <div id=\"expHeading12112857874\" class=\"accordion-collapse collapse \" aria-labelledby=\"expHeading4\" data-bs-parent=\"#accordionExample1211285787\">\n                                            <div class=\"accordion-body\">\n                                                <div class=\"accordion-data\">\n                                                    <p><p>Severe Primary Insulin-Like Growth Factor Deficiency (SPIGFD) occurs when children lack insulin-like growth factor 1 (IGF-1), a naturally occurring hormone that is vital in promoting growth. If IGF-1 levels are very low, but growth hormone levels are normal or even elevated, this can cause children to be shorter than almost all others of the same age and gender.<\/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=\"#expHeading12112857875\" aria-expanded=\"false\" aria-controls=\"expHeading12112857875\">\n                                                Central Precocious Puberty                                            <\/button>\n                                        <\/p>\n                                        <div id=\"expHeading12112857875\" class=\"accordion-collapse collapse \" aria-labelledby=\"expHeading5\" data-bs-parent=\"#accordionExample1211285787\">\n                                            <div class=\"accordion-body\">\n                                                <div class=\"accordion-data\">\n                                                    <p><p>Central precocious puberty (CPP) occurs when a child develops into a young adult at an unexpectedly early age because their growth and sex hormone glands begin to function early.<sup>\u00a0<\/sup>CPP can be caused by tumors in the central nervous system (CNS) and other CNS disorders. However, a larger number of CPP cases are of unknown origin, especially in females; research is beginning to identify rare genetic causes for some cases.<\/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=\"1736122267\">\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                            Rare liver diseases                        <\/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=\"accordionExample1736122267\">\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=\"#expHeading17361222670\" aria-expanded=\"false\" aria-controls=\"expHeading17361222670\">\n                                                Progressive familial intrahepatic cholestasis (PFIC)                                            <\/button>\n                                        <\/p>\n                                        <div id=\"expHeading17361222670\" class=\"accordion-collapse collapse \" aria-labelledby=\"expHeading0\" data-bs-parent=\"#accordionExample1736122267\">\n                                            <div class=\"accordion-body\">\n                                                <div class=\"accordion-data\">\n                                                    <p><p>PFIC is a rare inherited condition in which children are unable to drain bile from the liver even though the large bile ducts are open (cholestasis). As a result, bile acid builds up in liver cells causing liver disease and symptoms. It is estimated that PFIC occurs in 1 in 100,000 live births. The most debilitating 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. Up to 80% of \u00a0patients suffer from severe pruritus.<\/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 One Start -->\n                <div class=\"row \">\n                    <div class=\"col-xs-12 col-sm-12 col-md-6 column-1 \">\n                                                        <div class=\"no-carousel\">\n                                    <img decoding=\"async\" src=\"https:\/\/www.ipsen.com\/uae-gulf\/wp-content\/uploads\/sites\/18\/2025\/10\/Module-2-\u2013-Brief-about-neuroscience_IPSEN_Production_Signes_060.jpg\" alt=\"\">\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 class=\"heading-para heading-four\">\n                                            Ipsen rare disease clinical trials                                        <\/h4>\n                                    <\/div>\n                                                                    <p class=\"section-para \">\n                                            We are constantly striving to bring new treatments to the clinical trial stage to meet patients\u2019 unmet medical needs.                                        <\/p>\n                                                                <a aria-label=\"\" \n                                        class=\"btn-link section-btn cta-visible\" \n                                        href=\"https:\/\/www.ipsen.com\/uae-gulf\/clinical-trials\/\" \n                                        aria-label=\"\" \n                                        target=\"_self\"\n                                    >\n                                        Learn more \n                                    <\/a>\n                                                    <\/div>\n                    <\/div>\n                <\/div>\n        <!-- Ipsen Content Overlay Variation One End -->\n                <\/div>\n    <\/div>\n<\/section>\n<!-- Ipsen Content Overlay End -->\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 One 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 class=\"no-carousel\">\n                                    <img decoding=\"async\" src=\"https:\/\/www.ipsen.com\/uae-gulf\/wp-content\/uploads\/sites\/18\/2025\/10\/Module-9-\u2013-Rare-disease-pipeline_191209_0722.jpg\" alt=\"\">\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 class=\"heading-para heading-four\">\n                                            Our pipeline                                        <\/h4>\n                                    <\/div>\n                                                                    <p class=\"section-para \">\n                                            Collaborating with like-minded partners, we are focused on improving the lives of patients and their caregivers. Our teams have unrelenting energy to deliver the best possible treatment options to patients who need them most.                                        <\/p>\n                                                                <a aria-label=\"\" \n                                        class=\"btn-link section-btn cta-visible\" \n                                        href=\"https:\/\/www.ipsen.com\/uae-gulf\/pipeline\/\" \n                                        aria-label=\"\" \n                                        target=\"_self\"\n                                    >\n                                        Learn more \n                                    <\/a>\n                                                    <\/div>\n                    <\/div>\n                <\/div>\n        <!-- Ipsen Content Overlay Variation One 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-41","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 - UAE &amp; 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