{"id":12219,"date":"2025-02-27T11:44:49","date_gmt":"2025-02-27T11:44:49","guid":{"rendered":"https:\/\/www.healthnews.ie\/?p=12219"},"modified":"2025-03-03T09:20:18","modified_gmt":"2025-03-03T09:20:18","slug":"hope-for-a-future-in-which-rare-diseases-are-no-longer-overlooked","status":"publish","type":"post","link":"https:\/\/www.healthnews.ie\/rare-diseases\/hope-for-a-future-in-which-rare-diseases-are-no-longer-overlooked\/","title":{"rendered":"Hope for a future in which rare diseases are no longer overlooked"},"content":{"rendered":"\n<div style=\"background-color:#f2f2f2;color:#32373c\" class=\"wp-block-atomic-blocks-ab-profile-box round ab-has-avatar ab-font-size-14 ab-block-profile ab-profile-columns\"><div class=\"ab-profile-column ab-profile-avatar-wrap\"><div class=\"ab-profile-image-wrap\"><figure class=\"ab-profile-image-square\"><img loading=\"lazy\" decoding=\"async\" width=\"274\" height=\"275\" class=\"ab-profile-avatar wp-image-12222\" src=\"https:\/\/s3.eu-north-1.amazonaws.com\/cdn-site.mediaplanet.com\/app\/uploads\/sites\/94\/2025\/02\/27113954\/vicky-1740656393-Luke-Robinson-BioCryst-Ireland-UK-and-Nordic-Countries-VP-and-General-Manager.png\" alt=\"\" srcset=\"https:\/\/s3.eu-north-1.amazonaws.com\/cdn-site.mediaplanet.com\/app\/uploads\/sites\/94\/2025\/02\/27113954\/vicky-1740656393-Luke-Robinson-BioCryst-Ireland-UK-and-Nordic-Countries-VP-and-General-Manager.png 274w, https:\/\/s3.eu-north-1.amazonaws.com\/cdn-site.mediaplanet.com\/app\/uploads\/sites\/94\/2025\/02\/27113954\/vicky-1740656393-Luke-Robinson-BioCryst-Ireland-UK-and-Nordic-Countries-VP-and-General-Manager-125x125.png 125w\" sizes=\"auto, (max-width: 274px) 100vw, 274px\" \/><\/figure><\/div><\/div><div class=\"ab-profile-column ab-profile-content-wrap\"><h2 class=\"ab-profile-name\" style=\"color:#32373c\">Luke Robinson<\/h2><p class=\"ab-profile-title\" style=\"color:#32373c\">VP and General Manager, Ireland, UK and the Nordic Countries, BioCryst\u00a0<\/p><div class=\"ab-profile-text\"><\/div><ul class=\"ab-social-links\"><\/ul><\/div><\/div>\n\n\n\n<p><br><em>Learn how a small biotech built on a big dream considers underserved patient populations at the heart of its innovation.\u00a0<\/em><\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<p>Although each rare disease may have a small patient population, the aggregate of all rare diseases means that a substantial proportion of people are impacted. Current conservative estimates suggest that 18\u201330 million people in the EU and 263\u2013446 million people worldwide are affected by rare disease at any point in time.<sup>1&nbsp;<\/sup><\/p>\n\n\n<div id=\"healt-1559635988\"><div class=\"healt-adlabel\">Advertisements<\/div><div id=\"healt-3238672068\" style=\"margin-top: 30px;margin-bottom: 30px;\"><a data-no-instant=\"1\" href=\"https:\/\/biocryst.ie\/\" rel=\"noopener\" class=\"a2t-link\" target=\"_blank\" aria-label=\"vicky-1740657902-BioCryst desk\"><img loading=\"lazy\" decoding=\"async\" src=\"https:\/\/s3.eu-north-1.amazonaws.com\/cdn-site.mediaplanet.com\/app\/uploads\/sites\/94\/2025\/02\/27120503\/vicky-1740657902-BioCryst-desk.jpg\" alt=\"\"  width=\"750\" height=\"250\"   \/><\/a><\/div><\/div>\n\n\n<h2 class=\"wp-block-heading\" id=\"h-commitment-to-rare-disease-advancement-nbsp\"><strong>Commitment to rare disease advancement&nbsp;<\/strong><\/h2>\n\n\n\n<p><a href=\"https:\/\/biocryst.ie\/\" target=\"_blank\" rel=\"noreferrer noopener\">BioCryst<\/a>, a commercial-stage US biotech with EMEA headquarters in Dublin, is committed to discovering and developing treatments and has spent over three decades working to meet unmet needs in rare disease. Luke Robinson, VP and General Manager for Ireland, UK and Nordic Countries suggests: \u201cMany rare diseases share common challenges, such as difficulty in diagnosis, limited treatment options and a lack of awareness, making the collective experience of people living with these conditions more significant. As a result, rare diseases are a growing area of focus for researchers, policymakers and all those who support the local healthcare infrastructure.\u201d\u00a0<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"h-delivering-targeted-solutions-for-unmet-patient-need-nbsp\"><strong>Delivering targeted solutions for unmet patient need&nbsp;<\/strong><\/h2>\n\n\n\n<p>BioCryst focuses on the <a href=\"https:\/\/biocryst.ie\/\" target=\"_blank\" rel=\"noreferrer noopener\">development of therapies for rare disease<\/a>. An example of this is hereditary angioedema (HAE), a potentially life-threatening rare disease characterised by repeated painful and unpredictable swelling attacks.&nbsp;<\/p>\n\n\n\n<blockquote class=\"wp-block-quote is-layout-flow wp-block-quote-is-layout-flow\">\n<p>Rare diseases are a growing area of focus <br>for researchers, policymakers and all those <br>who support the local healthcare infrastructure.<\/p>\n<\/blockquote>\n\n\n\n<p>Researchers at its Discovery Centre of Excellence use an innovative structure-guided approach to drug design, which involves the use of computational tools and techniques to analyse the molecular structure of disease-causing proteins. By understanding the shape and dynamics of these proteins and essentially how they \u2018fit together,\u2019 BioCryst can design small molecules that bind specifically to these targets, either inhibiting their harmful activity or modulating their function in a beneficial way. This approach is particularly useful in the treatment of rare diseases, where targets may be poorly understood or difficult to modulate using traditional drug discovery methods.&nbsp;<\/p>\n\n\n<div id=\"healt-186276397\"><div class=\"healt-adlabel\">Advertisements<\/div><div id=\"healt-560699081\" style=\"margin-top: 30px;margin-bottom: 30px;\"><a data-no-instant=\"1\" href=\"https:\/\/biocryst.ie\/\" rel=\"noopener\" class=\"a2t-link\" target=\"_blank\" aria-label=\"vicky-1740657902-BioCryst desk\"><img loading=\"lazy\" decoding=\"async\" src=\"https:\/\/s3.eu-north-1.amazonaws.com\/cdn-site.mediaplanet.com\/app\/uploads\/sites\/94\/2025\/02\/27120503\/vicky-1740657902-BioCryst-desk.jpg\" alt=\"\"  width=\"750\" height=\"250\"   \/><\/a><\/div><\/div>\n\n\n<h2 class=\"wp-block-heading\" id=\"h-collaboration-supporting-innovation-nbsp\"><strong>Collaboration supporting innovation&nbsp;<\/strong><\/h2>\n\n\n\n<p>Innovation and collaboration in rare disease accelerate the development of new drugs and improve the precision of therapies. They give people living with rare disease a chance of achieving health equity, supporting the better management of their conditions and quality of life in a practical way.&nbsp;<\/p>\n\n\n\n<p>With BioCryst\u2019s approach to innovation, listening, learning and sharing, it has made strides across Europe in working with patient groups, regulators and Health Technology Assessment (HTA) bodies to bring new medicines to those who need them most. \u201cI am proud of our approach and while much has been achieved, I am looking forward to what is yet to come,\u201d concludes Robinson.&nbsp;<\/p>\n\n\n<!DOCTYPE html PUBLIC \"-\/\/W3C\/\/DTD HTML 4.0 Transitional\/\/EN\" \"http:\/\/www.w3.org\/TR\/REC-html40\/loose.dtd\">\n<html><body><div style=\"text-align:center\" class=\"wp-block-atomic-blocks-ab-button ab-block-button\"><a href=\"https:\/\/biocryst.ie\/\" target=\"_blank\" rel=\"noopener noreferrer\" class=\"ab-button ab-button-shape-rounded ab-button-size-medium fb-pixel\" style=\"color:#ffffff;background-color:#7ac79b\">Click here to learn more<\/a><\/div>\n<\/body><\/html>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<p>IE.BCX.00008&nbsp;<\/p>\n\n\n\n<p class=\"has-text-align-right has-small-font-size\"><strong>[1] <\/strong>Nguengang Wakap, S., Lambert, D.M., Olry, A. et al. Estimating cumulative point prevalence of rare diseases: analysis of the Orphanet database. Eur J Hum Genet 28, 165\u2013173 (2020).&nbsp;<\/p>\n\n\n\n<p><\/p>\n","protected":false},"excerpt":{"rendered":"<p>Learn how a small biotech built on a big dream considers underserved patient populations at the heart of its innovation.\u00a0 Although each rare disease may have a small patient population, the aggregate of all rare diseases means that a substantial proportion of people are impacted. Current conservative estimates suggest that 18\u201330 million people in the &hellip; <a href=\"https:\/\/www.healthnews.ie\/rare-diseases\/hope-for-a-future-in-which-rare-diseases-are-no-longer-overlooked\/\">Continued<\/a><\/p>\n","protected":false},"author":91,"featured_media":12223,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"footnotes":""},"categories":[5778],"tags":[5961],"class_list":["post-12219","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-rare-diseases","tag-rare-disease-day-2025"],"acf":[],"featured_image_src":"https:\/\/s3.eu-north-1.amazonaws.com\/cdn-site.mediaplanet.com\/app\/uploads\/sites\/94\/2025\/02\/27114411\/vicky-1740656641-AdobeStock_1075359599-600x400.png","featured_image_src_square":"https:\/\/s3.eu-north-1.amazonaws.com\/cdn-site.mediaplanet.com\/app\/uploads\/sites\/94\/2025\/02\/27114411\/vicky-1740656641-AdobeStock_1075359599-600x600.png","author_info":{"display_name":"henryphillips","author_link":"https:\/\/www.healthnews.ie\/author\/henryphillips\/"},"vicky_category_order":[{"category_id":5778,"order":"1"}],"vicky_index_order":"8","_links":{"self":[{"href":"https:\/\/www.healthnews.ie\/wp-json\/wp\/v2\/posts\/12219","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.healthnews.ie\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.healthnews.ie\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.healthnews.ie\/wp-json\/wp\/v2\/users\/91"}],"replies":[{"embeddable":true,"href":"https:\/\/www.healthnews.ie\/wp-json\/wp\/v2\/comments?post=12219"}],"version-history":[{"count":5,"href":"https:\/\/www.healthnews.ie\/wp-json\/wp\/v2\/posts\/12219\/revisions"}],"predecessor-version":[{"id":12236,"href":"https:\/\/www.healthnews.ie\/wp-json\/wp\/v2\/posts\/12219\/revisions\/12236"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.healthnews.ie\/wp-json\/wp\/v2\/media\/12223"}],"wp:attachment":[{"href":"https:\/\/www.healthnews.ie\/wp-json\/wp\/v2\/media?parent=12219"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.healthnews.ie\/wp-json\/wp\/v2\/categories?post=12219"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.healthnews.ie\/wp-json\/wp\/v2\/tags?post=12219"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}