{"id":20068,"date":"2024-04-03T10:50:12","date_gmt":"2024-04-03T08:50:12","guid":{"rendered":"https:\/\/ela-international.preprod.melisagency.fr\/?post_type=news&p=20068"},"modified":"2025-04-10T10:30:07","modified_gmt":"2025-04-10T08:30:07","slug":"libmeldy-us-approval","status":"publish","type":"news","link":"https:\/\/elainternational.eu\/en\/actualites\/libmeldy-us-approval\/","title":{"rendered":"Like 11 European countries, the United States approve Libmeldy"},"content":{"rendered":"\n\n
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Like 11 European countries, the United States approve\u00a0 Libmeldy<\/h3>\n

A rare and devastating genetic disease<\/h4>\n

Metachromatic leukodystrophy (MLD) is a rare genetic disease, occurring at a rate of 1 in 45,000 births. It can begin in childhood, adolescence, or adulthood, and leads to severe neurological dysfunction affecting motor skills and cognitive functions, and potentially to death.<\/p>\n

Libmeldy: an innovative treatment<\/h4>\n

For children unable to receive a bone marrow transplant, families\u2019 only hope lies in gene therapy: the stem cells of the patient are harvested and corrected in laboratory before being reinjected. Libmeldy was developed in Italy and required over ten years of research. Around forty European children received the treatment during clinical trials carried out to verify the safety and efficacy of the therapy. This treatment has since received European marketing authorization in 2021.<\/p>\n

A European health issue<\/strong><\/h4>\n

At the time of writing, this brings to 11 the number of European countries that have agreed to permanently reimburse this proven, life-saving gene therapy treatment: Belgium, Denmark, Finland, Germany, Iceland, Ireland, Italy, the Netherlands, Norway, Sweden and the United Kingdom.<\/p>\n

\"\"<\/p>\n

In France, treatment is available under a special dispensation: early access. This system is neither permanent nor guaranteed over time.<\/p>\n

\u201cThe patient must always come first, especially when a life is at stake, and the government must then do everything possible to ensure this. And that\u2019s why we need to cooperate much more in Europe\u2026\u201d<\/em><\/p>\n

Frank Vandenbroucke, Belgian Minister of Health and Social Affairs (January 2024)<\/p>\n

 <\/p>\n

\u201cToday\u2019s announcement of Libmeldy\u2019s reimbursement approval in our three countries gives hope to families who can now access this life-changing drug\u2026\u201d. <\/em><\/p>\n

Stephen Donnelly T.D., Irish Minister for Health (January 2024)<\/p>\n

 <\/p>\n

\u201cThis positive outcome is excellent news for MLD patients and their families. This collaboration between Belgium, Ireland and The Netherlands demonstrates once again the value of our countries working together in price negotiations\u2026\u201d<\/em><\/p>\n

Conny Helder, Dutch Minister of Health, Welfare and Sport (January 2024)<\/p>\n

A major victory for ELA’s children and families<\/strong><\/h4>\n

ELA International has funded numerous research projects on metachromatic leukodystrophy, contributing to the discovery of this new treatment. But ELA\u2019s mission is not limited to research. In the 7 countries where it is present, ELA also supports families on a daily basis.<\/p>\n

We are glad that this life-saving treatment is becoming available to a greater number of patients in Europe. France, Spain and other European countries are still missing in action, to the great dismay of the families affected. This is why we are closely monitoring the ongoing negotiations between all the parties involved.<\/p>\n\n <\/div>\n<\/div>\n\n","protected":false},"excerpt":{"rendered":"","protected":false},"author":2,"featured_media":19955,"parent":0,"template":"","news_categories":[97],"class_list":["post-20068","news","type-news","status-publish","has-post-thumbnail","hentry","news_categories-international-en"],"acf":[],"yoast_head":"\nLike 11 European countries, the United States approve Libmeldy - ELA International<\/title>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/elainternational.eu\/en\/actualites\/libmeldy-us-approval\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Like 11 European countries, the United States approve Libmeldy - ELA International\" \/>\n<meta property=\"og:url\" content=\"https:\/\/elainternational.eu\/en\/actualites\/libmeldy-us-approval\/\" \/>\n<meta property=\"og:site_name\" content=\"ELA International\" \/>\n<meta property=\"article:modified_time\" content=\"2025-04-10T08:30:07+00:00\" \/>\n<meta property=\"og:image\" content=\"https:\/\/elainternational.eu\/wp-content\/uploads\/2024\/04\/News-Libmeldy-entete.jpg\" \/>\n\t<meta property=\"og:image:width\" content=\"2000\" \/>\n\t<meta property=\"og:image:height\" content=\"626\" \/>\n\t<meta property=\"og:image:type\" content=\"image\/jpeg\" \/>\n<meta name=\"twitter:card\" content=\"summary_large_image\" \/>\n<meta name=\"twitter:label1\" content=\"Est. reading time\" \/>\n\t<meta name=\"twitter:data1\" content=\"1 minute\" \/>\n<script type=\"application\/ld+json\" class=\"yoast-schema-graph\">{\"@context\":\"https:\\\/\\\/schema.org\",\"@graph\":[{\"@type\":\"WebPage\",\"@id\":\"https:\\\/\\\/elainternational.eu\\\/en\\\/actualites\\\/libmeldy-us-approval\\\/\",\"url\":\"https:\\\/\\\/elainternational.eu\\\/en\\\/actualites\\\/libmeldy-us-approval\\\/\",\"name\":\"Like 11 European countries, the United States approve Libmeldy - 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