{"id":26674,"date":"2025-12-19T10:55:35","date_gmt":"2025-12-19T09:55:35","guid":{"rendered":"https:\/\/www.unither-pharma.com\/journee-des-maladies-rares\/"},"modified":"2026-02-25T14:16:32","modified_gmt":"2026-02-25T13:16:32","slug":"journee-des-maladies-rares","status":"publish","type":"post","link":"https:\/\/www.unither-pharma.com\/fr\/journee-des-maladies-rares\/","title":{"rendered":"Journ\u00e9e des maladies rares"},"content":{"rendered":"<h2>Journ\u00e9e des maladies rares : Un appel \u00e0 l\u2019innovation dans le d\u00e9veloppement pharmaceutique<\/h2>\n<p>&nbsp;<\/p>\n<p><em><strong data-start=\"0\" data-end=\"20\" data-is-only-node=\"\">Marianne Henrion<\/strong> est <strong data-start=\"25\" data-end=\"51\">Directrice du site R&amp;D<\/strong> chez <strong data-start=\"57\" data-end=\"84\">Unither Pharmaceuticals<\/strong> depuis 2024. Forte de plus de 25 ans d\u2019exp\u00e9rience dans le d\u00e9veloppement pharmaceutique, elle a occup\u00e9 des postes strat\u00e9giques chez Ethypharm, Famar et Catalent, contribuant \u00e0 l\u2019innovation et \u00e0 la mise sur le march\u00e9 de solutions th\u00e9rapeutiques \u00e0 l\u2019\u00e9chelle internationale.<\/em><\/p>\n<p id=\"ember50\" class=\"ember-view reader-text-block__paragraph\">Chaque ann\u00e9e, la Journ\u00e9e des maladies rares nous rappelle \u00e0 quel point l\u2019innovation est essentielle pour r\u00e9pondre aux besoins des <strong>300 millions de personnes concern\u00e9es<\/strong> par une maladie rare. Derri\u00e8re ce chiffre, ce sont des patients et des familles confront\u00e9s \u00e0 des parcours m\u00e9dicaux complexes, des diagnostics tardifs et des solutions th\u00e9rapeutiques souvent inexistantes.<\/p>\n<p id=\"ember51\" class=\"ember-view reader-text-block__paragraph\"><strong>Les principaux d\u00e9fis sont :<\/strong><\/p>\n<ul>\n<li>Un diagnostic long et complexe, avec un <strong>d\u00e9lai moyen de 5 ans en Europe<\/strong> et <strong>18 mois en France<\/strong>, laissant de nombreux patients sans prise en charge adapt\u00e9e pendant des ann\u00e9es. <strong>70 % des patients attendent plus d\u2019un an<\/strong> apr\u00e8s une premi\u00e8re consultation avant d\u2019obtenir un diagnostic confirm\u00e9.<\/li>\n<li>Des essais cliniques difficiles \u00e0 mener, en raison du faible nombre de patients et de leur dispersion g\u00e9ographique, rendant le recrutement particuli\u00e8rement complexe.<\/li>\n<li>Des capacit\u00e9s de d\u00e9veloppement sp\u00e9cifiques pour mettre au point et fabriquer des traitements adapt\u00e9s \u00e0 des maladies qui, dans 95% des cas, ne disposent d\u2019aucune option curative.<\/li>\n<\/ul>\n<p id=\"ember53\" class=\"ember-view reader-text-block__paragraph\">Face \u00e0 ces enjeux, l\u2019innovation en biotechnologie, en formulation et en production pharmaceutique est essentielle pour acc\u00e9l\u00e9rer l\u2019acc\u00e8s aux traitements et r\u00e9pondre aux besoins des patients.<\/p>\n<h3>Une mobilisation collective pour acc\u00e9l\u00e9rer l\u2019innovation<\/h3>\n<p id=\"ember55\" class=\"ember-view reader-text-block__paragraph\">D\u00e9velopper un m\u00e9dicament pour une maladie rare demande une collaboration \u00e9troite entre les chercheurs, les entreprises sp\u00e9cialis\u00e9es en biotechnologies, les industriels et les autorit\u00e9s de sant\u00e9.<\/p>\n<ul>\n<li><strong>Les laboratoires acad\u00e9miques<\/strong> jouent un r\u00f4le cl\u00e9 dans la compr\u00e9hension des m\u00e9canismes biologiques de ces maladies.<\/li>\n<li><strong>Les entreprises sp\u00e9cialis\u00e9es en biotechnologies<\/strong> d\u00e9veloppent des approches th\u00e9rapeutiques de pointe.<\/li>\n<li><strong>Les industriels sp\u00e9cialis\u00e9s<\/strong> assurent la transposition et l\u2019acc\u00e8s des traitements aux patients en ma\u00eetrisant les contraintes r\u00e9glementaires et de fabrication.<\/li>\n<\/ul>\n<p id=\"ember57\" class=\"ember-view reader-text-block__paragraph\">Cette synergie est essentielle pour transformer les d\u00e9couvertes scientifiques en traitements concrets.<\/p>\n<h3 id=\"ember58\" class=\"ember-view reader-text-block__heading-3\">Un rappel de l\u2019accessibilit\u00e9 des solutions de sant\u00e9 pour tous<\/h3>\n<p id=\"ember59\" class=\"ember-view reader-text-block__paragraph\">Chez Unither Pharmaceuticals, notre vision est claire : rendre accessible des solutions de sant\u00e9 qui am\u00e9liorent et simplifient la vie des patients, y compris ceux atteints de maladies rares.<\/p>\n<p id=\"ember60\" class=\"ember-view reader-text-block__paragraph\">L\u2019innovation ne doit pas \u00eatre frein\u00e9e par la complexit\u00e9 du d\u00e9veloppement pharmaceutique ou par des mod\u00e8les \u00e9conomiques inadapt\u00e9s. Elle doit, au contraire, s\u2019adapter aux besoins des patients et explorer de nouvelles voies, qu\u2019il s\u2019agisse de th\u00e9rapies g\u00e9niques, de nouvelles formes gal\u00e9niques ou de solutions facilitant l\u2019acc\u00e8s aux traitements.<\/p>\n<p id=\"ember61\" class=\"ember-view reader-text-block__paragraph\"><strong>Le r\u00f4le d\u2019UDB au sein du Groupe Unither<\/strong><\/p>\n<p id=\"ember62\" class=\"ember-view reader-text-block__paragraph\">Au sein d\u2019Unither D\u00e9veloppement Bordeaux (UDB), centre R&amp;D d\u2019Unither Pharmaceuticals, nous accompagnons les entreprises pharmaceutiques et biotechnologiques dans la mise au point de nouveaux traitements. Sp\u00e9cialis\u00e9 dans le d\u00e9veloppement de formes liquides, semi-solides et solides, nous avons la capacit\u00e9 de produire des petits lots, un \u00e9l\u00e9ment cl\u00e9 pour les essais cliniques et pour la commercialisation des traitements des maladies rares.<\/p>\n<p id=\"ember63\" class=\"ember-view reader-text-block__paragraph\">Nous couvrons l\u2019ensemble du cycle de d\u00e9veloppement, de la pr\u00e9-formulation \u00e0 la formulation gal\u00e9nique, au d\u00e9veloppement analytique, en passant par la validation des m\u00e9thodes, les \u00e9tudes de stabilit\u00e9 conformes aux r\u00e9glementations internationales jusqu\u2019\u00e0 la fabrication de lots pr\u00e9cliniques et cliniques. De plus, UDB est accr\u00e9dit\u00e9 au Cr\u00e9dit d\u2019Imp\u00f4t Recherche (CIR).<\/p>\n<p id=\"ember64\" class=\"ember-view reader-text-block__paragraph\">Chaque avanc\u00e9e commence par un engagement en faveur des patients. En cette Journ\u00e9e des maladies rares, nous saluons la r\u00e9silience des patients et de leurs familles et r\u00e9affirmons notre volont\u00e9 d\u2019innover pour mettre au point de nouveaux traitements.<\/p>\n<p id=\"ember65\" class=\"ember-view reader-text-block__paragraph\">De la recherche \u00e0 la commercialisation, les efforts se poursuivent pour rendre ces th\u00e9rapies accessibles \u00e0 ceux qui en ont le plus besoin, afin que m\u00eame les maladies les plus rares ne soient pas oubli\u00e9es.<\/p>\n<p id=\"ember66\" class=\"ember-view reader-text-block__paragraph\"><strong>Sources<\/strong><\/p>\n<p id=\"ember67\" class=\"ember-view reader-text-block__paragraph\">\u00b7 <em>Estimating cumulative point prevalence of rare diseases: analysis of the Orphanet database, European Journal of Human Genetics (2019)<\/em><\/p>\n<p id=\"ember68\" class=\"ember-view reader-text-block__paragraph\">\u00b7 <em>Maladies Rares Info \u2013 Les chiffres cl\u00e9s des maladies rares <\/em><a class=\"BefCxZGaSWcPFahNZGheKUNEpFkmNKjephLXQ \" href=\"http:\/\/www.maladiesraresinfo.org\/informer\/information-generale-sur-les-maladies-rares\/les-chiffres-clefs-des-maladies-rares.html\" target=\"_self\" data-test-app-aware-link=\"\"><em>www.maladiesraresinfo.org\/informer\/information-generale-sur-les-maladies-rares\/les-chiffres-clefs-des-maladies-rares.html<\/em><\/a><\/p>\n<p id=\"ember69\" class=\"ember-view reader-text-block__paragraph\">\u00b7 <em>Eurordis \u2013 Screening rare diseases at birth! <\/em><a class=\"BefCxZGaSWcPFahNZGheKUNEpFkmNKjephLXQ \" href=\"http:\/\/www.eurordis.org\/publications\/rb-nbs-survey-results\/\" target=\"_self\" data-test-app-aware-link=\"\"><em>www.eurordis.org\/publications\/rb-nbs-survey-results\/<\/em><\/a><\/p>\n<p id=\"ember70\" class=\"ember-view reader-text-block__paragraph\">\u00b7 <em>Alcimed \u2013 Essais cliniques et maladies rares : quels d\u00e9fis ? <\/em><a class=\"BefCxZGaSWcPFahNZGheKUNEpFkmNKjephLXQ \" href=\"http:\/\/www.alcimed.com\/fr\/insights\/essais-cliniques-maladies-rares-defis-2\/\" target=\"_self\" data-test-app-aware-link=\"\"><em>www.alcimed.com\/fr\/insights\/essais-cliniques-maladies-rares-defis-2\/<\/em><\/a><\/p>\n","protected":false},"excerpt":{"rendered":"<p>Journ\u00e9e des maladies rares : Un appel \u00e0 l\u2019innovation dans le d\u00e9veloppement pharmaceutique &nbsp; Marianne Henrion est Directrice du site R&amp;D chez Unither Pharmaceuticals depuis 2024. Forte de plus de 25 ans d\u2019exp\u00e9rience dans le d\u00e9veloppement pharmaceutique, elle a occup\u00e9 des postes strat\u00e9giques chez Ethypharm, Famar et Catalent, contribuant \u00e0 l\u2019innovation et \u00e0 la mise [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":20005,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"footnotes":""},"categories":[19,148],"tags":[],"class_list":["post-26674","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-actualites","category-corporate"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v27.2 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>Journ\u00e9e des maladies rares | Unither<\/title>\n<meta name=\"description\" content=\"Journ\u00e9e des maladies rares | Unither\" \/>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, 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