{"id":5172,"date":"2025-05-06T11:43:45","date_gmt":"2025-05-06T09:43:45","guid":{"rendered":"https:\/\/rijetke-bolesti.com\/?p=5172"},"modified":"2025-10-17T13:06:00","modified_gmt":"2025-10-17T11:06:00","slug":"nova-terapija-za-duchenne-misicnu-distrofiju","status":"publish","type":"post","link":"https:\/\/rijetke-bolesti.com\/en\/nova-terapija-za-duchenne-misicnu-distrofiju\/","title":{"rendered":"New Therapy for Duchenne Muscular Dystrophy"},"content":{"rendered":"<div class=\"wp-block-image\">\n<figure class=\"aligncenter size-full\"><img loading=\"lazy\" decoding=\"async\" width=\"391\" height=\"592\" src=\"https:\/\/rijetke-bolesti.com\/wp-content\/uploads\/2019\/03\/Duvyzat.png\" alt=\"\" class=\"wp-image-5781\" srcset=\"https:\/\/rijetke-bolesti.com\/wp-content\/uploads\/2019\/03\/Duvyzat.png 391w, https:\/\/rijetke-bolesti.com\/wp-content\/uploads\/2019\/03\/Duvyzat-198x300.png 198w, https:\/\/rijetke-bolesti.com\/wp-content\/uploads\/2019\/03\/Duvyzat-8x12.png 8w\" sizes=\"auto, (max-width: 391px) 100vw, 391px\" \/><\/figure>\n<\/div>\n\n\n<p>The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion recommending the conditional marketing authorization of givinostat for the treatment of ambulant patients aged six years and older with Duchenne muscular dystrophy (DMD). <\/p>\n\n\n\n<p>The European Commission is expected to make a final decision on the authorization of the medicine within the European Union in July. <\/p>\n\n\n\n<p>Duchenne muscular dystrophy is a rare, progressive neuromuscular disorder caused by mutations in the DMD gene, which make muscle fibers more vulnerable to damage and prevent the activation of key genes essential for muscle maintenance and repair. The continuous cycle of muscle injury leads to chronic inflammation, impaired regeneration, and the gradual replacement of muscle tissue with connective and fatty tissue.\nThe condition primarily affects boys, with symptoms typically appearing between the ages of two and five. Over time, muscle weakness worsens, affecting mobility and eventually involving the heart and respiratory muscles, often leading to premature death. DMD is among the most common types of childhood muscular dystrophy, with a global incidence of approximately 1 in 5,050 boys. <\/p>\n\n\n\n<p>For more information, please contact Tina Vu\u010dkovi\u0107, President of DMD Hrvatska (Duchenne Muscular Dystrophy Association of Croatia), by phone at +385 91 9138 010 or via email at dmdhrvatska@gmail.com. <a>dmdhrvatska@gmail.com<\/a>.<\/p>\n\n\n\n<figure class=\"wp-block-gallery has-nested-images columns-default is-cropped wp-block-gallery-1 is-layout-flex wp-block-gallery-is-layout-flex\"><\/figure>","protected":false},"excerpt":{"rendered":"<p>The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion recommending the conditional marketing authorization of givinostat for the treatment of ambulant patients aged six years and older with Duchenne muscular dystrophy (DMD).<\/p>","protected":false},"author":3,"featured_media":0,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"content-type":"","footnotes":""},"categories":[1],"tags":[],"class_list":["post-5172","post","type-post","status-publish","format-standard","hentry","category-uncategorized"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v26.0 - https:\/\/yoast.com\/wordpress\/plugins\/seo\/ -->\n<title>Nova terapija za Duchenne mi\u0161i\u0107nu distrofiju - Hrvatski savez za rijetke bolesti<\/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:\/\/rijetke-bolesti.com\/en\/nova-terapija-za-duchenne-misicnu-distrofiju\/\" \/>\n<meta property=\"og:locale\" content=\"en_GB\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Nova terapija za Duchenne mi\u0161i\u0107nu distrofiju - Hrvatski savez za rijetke bolesti\" \/>\n<meta property=\"og:description\" content=\"Povjerenstvo za humane lijekove (CHMP) Europske agencije za lijekove (EMA) donijelo je pozitivno mi\u0161ljenje kojim se preporu\u010duje izdavanje uvjetnog odobrenja za stavljanje u promet lijeka givinostat za lije\u010denje pokretnih bolesnika s Duchenneovom mi\u0161i\u0107nom distrofijom (DMD) starijih od \u0161est godina. Europska komisija kona\u010dnu bi odluku o...\" \/>\n<meta property=\"og:url\" content=\"https:\/\/rijetke-bolesti.com\/en\/nova-terapija-za-duchenne-misicnu-distrofiju\/\" \/>\n<meta property=\"og:site_name\" content=\"Hrvatski savez za rijetke bolesti\" \/>\n<meta property=\"article:published_time\" content=\"2025-05-06T09:43:45+00:00\" \/>\n<meta property=\"article:modified_time\" content=\"2025-10-17T11:06:00+00:00\" \/>\n<meta property=\"og:image\" content=\"https:\/\/rijetke-bolesti.com\/wp-content\/uploads\/2019\/03\/Duvyzat.png\" \/>\n\t<meta property=\"og:image:width\" content=\"391\" \/>\n\t<meta property=\"og:image:height\" content=\"592\" \/>\n\t<meta property=\"og:image:type\" content=\"image\/png\" \/>\n<meta name=\"author\" content=\"sara\" \/>\n<meta name=\"twitter:card\" content=\"summary_large_image\" \/>\n<meta name=\"twitter:label1\" content=\"Written by\" \/>\n\t<meta name=\"twitter:data1\" content=\"sara\" \/>\n\t<meta name=\"twitter:label2\" content=\"Estimated reading time\" \/>\n\t<meta name=\"twitter:data2\" content=\"2 minutes\" \/>\n<script type=\"application\/ld+json\" class=\"yoast-schema-graph\">{\"@context\":\"https:\/\/schema.org\",\"@graph\":[{\"@type\":\"WebPage\",\"@id\":\"https:\/\/rijetke-bolesti.com\/nova-terapija-za-duchenne-misicnu-distrofiju\/\",\"url\":\"https:\/\/rijetke-bolesti.com\/nova-terapija-za-duchenne-misicnu-distrofiju\/\",\"name\":\"Nova terapija za Duchenne mi\u0161i\u0107nu distrofiju - 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