{"id":10141,"date":"2022-12-30T17:48:10","date_gmt":"2022-12-30T17:48:10","guid":{"rendered":"https:\/\/panel.ptcbio.com\/it-p2025tc\/?page_id=10141"},"modified":"2022-12-30T17:48:10","modified_gmt":"2022-12-30T17:48:10","slug":"duchenne-muscular-dystrophy","status":"publish","type":"page","link":"https:\/\/panel.ptcbio.com\/it-p2025tc\/therapeutic-areas\/duchenne-muscular-dystrophy\/","title":{"rendered":"Duchenne muscular dystrophy"},"content":{"rendered":"<div class=\"hero-header__outer-container\">\n\n\t<div class=\"hero hero--styled color-theme--denim-blue hero--no-image hero--normal-spacing\">\n\t\t\t\t\t\t\t\t\t\t<div class=\"hero__inner hero__inner--center\">\n\t\t\t<div class=\"hero__content hero__content--center hero__content--no-image\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"u-mt-1 u-my-2\"><h1 class=\"hero__title hero__title--standard\" data-ptc-fitty=\"\">Duchenne Muscular Dystrophy (DMD)<\/h1><\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"hero__subtitle\">Rare and fatal genetic disorder resulting in progressive muscle weakness from early childhood<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t\t<\/div>\n\t\t\t<div class=\"hero__breadcrumbs\"><nav\n\tclass=\"breadcrumbs breadcrumbs--deep\"\n\taria-label=\"Breadcrumb\"\n>\n\t<ol class=\"breadcrumbs__list\" itemscope itemtype=\"https:\/\/schema.org\/BreadcrumbList\">\n\t\t\t\t\t\t\t\t\t<li class=\"breadcrumbs__crumb breadcrumbs__crumb--light\" itemscope itemprop=\"itemListElement\" itemtype=\"https:\/\/schema.org\/ListItem\">\n\t\t\t\t\t\t\t\t\t\t\t<a class=\"breadcrumbs__link breadcrumbs__link--home breadcrumbs__link--light\" itemprop=\"item\" href=\"https:\/\/panel.ptcbio.com\/it-p2025tc\/\">\n\t\t\t\t\t\t\t<span class=\"sr-only\" itemprop=\"name\">Homepage<\/span>\n\t\t\t\t\t\t<\/a>\n\t\t\t\t\t\t\t\t\t\t<meta itemprop=\"position\" content=\"1\" \/>\n\t\t\t\t<\/li>\n\t\t\t\t\t\t\t\t\t\t\t\t<li class=\"breadcrumbs__crumb breadcrumbs__crumb--light\" itemscope itemprop=\"itemListElement\" itemtype=\"https:\/\/schema.org\/ListItem\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<a\n\t\t\t\t\t\t\t\tclass=\"breadcrumbs__link\" itemprop=\"item\"\n\t\t\t\t\t\t\t\thref=\"https:\/\/panel.ptcbio.com\/it-p2025tc\/therapeutic-areas\/\"\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<span itemprop=\"name\">Therapeutic Areas<\/span>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/a>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<meta itemprop=\"position\" content=\"2\" \/>\n\t\t\t\t<\/li>\n\t\t\t\t\t\t\t\t\t\t\t\t<li class=\"breadcrumbs__crumb breadcrumbs__crumb--light\" itemscope itemprop=\"itemListElement\" itemtype=\"https:\/\/schema.org\/ListItem\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<span\n\t\t\t\t\t\t\t\tclass=\"breadcrumbs__current\" itemprop=\"item\"\n\t\t\t\t\t\t\t\taria-current=\"page\"\t\t\t\t\t\t\t>\n\t\t\t\t\t\t\t\t<span itemprop=\"name\">Duchenne muscular dystrophy<\/span>\n\t\t\t\t\t\t\t<\/span>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<meta itemprop=\"position\" content=\"3\" \/>\n\t\t\t\t<\/li>\n\t\t\t\t\t\t<\/ol>\n<\/nav>\n<\/div>\n\t<\/div>\n\n\n\n<h4 class=\"wp-block-heading\" id=\"h-what-is-duchenne-muscular-dystrophy\">What is Duchenne Muscular Dystrophy?<\/h4>\n\n\n\n<figure class=\"wp-block-image alignright size-full is-resized is-style-lightbox\"><img loading=\"lazy\" decoding=\"async\" width=\"630\" height=\"690\" src=\"https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/10\/Duchenne-Signs-Symptoms-graphic.jpg\" alt=\"Duchenne Muscular Dystrophy Signs and Symptoms Infographic\" class=\"wp-image-16408\" style=\"width:368px;height:auto\" srcset=\"https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/10\/Duchenne-Signs-Symptoms-graphic.jpg 630w, https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/10\/Duchenne-Signs-Symptoms-graphic-274x300.jpg 274w, https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/10\/Duchenne-Signs-Symptoms-graphic-400x438.jpg 400w\" sizes=\"auto, (max-width: 630px) 100vw, 630px\" \/><button class=\"modal-toggle\" type=\"button\" data-bs-toggle=\"modal\" data-bs-target=\"#mg-image-modal\" aria-label=\"View image in lightbox\"><img fetchpriority=\"high\" decoding=\"async\" width=\"630\" height=\"690\" src=\"https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/10\/Duchenne-Signs-Symptoms-graphic.jpg\" class=\"attachment-full size-full\" alt=\"Duchenne Muscular Dystrophy Signs and Symptoms Infographic\" hidden=\"\" loading=\"eager\" srcset=\"https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/10\/Duchenne-Signs-Symptoms-graphic.jpg 630w, https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/10\/Duchenne-Signs-Symptoms-graphic-274x300.jpg 274w, https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/10\/Duchenne-Signs-Symptoms-graphic-400x438.jpg 400w\" sizes=\"(max-width: 630px) 100vw, 630px\" \/><\/button><\/figure>\n\n\n\n<p class=\"core-block core-paragraph\">Muscular dystrophies involve progressive muscle wasting and weakness and are caused by a mutation in the DNA that results in either the absence of or very low levels of the dystrophin protein.<sup>1<\/sup> Duchenne muscular dystrophy (DMD) is the most common \u2013 and most severe \u2013 type of muscular dystrophy.<sup>2<\/sup><\/p>\n\n\n\n<p class=\"core-block core-paragraph\">Duchenne muscular dystrophy occurs when a mutation in the dystrophin gene prevents the cell from making a functional dystrophin protein.<sup>3,4<\/sup> Dystrophin is a muscle membrane associated protein and is critical to the structural and membrane stability of muscle fibers in the skeletal, diaphragm and heart muscle.<sup>3,4<\/sup> The absence of normally functioning dystrophin results in muscle fragility, such that muscle injury occurs when muscles contract or stretch during normal use.<sup>4,5<\/sup> As muscle damage progresses, connective tissue and fat replace muscle fibers, resulting in inexorable muscle weakness.<sup>4,5<\/sup> Patients with Duchenne muscular dystrophy typically lose the ability to walk by their early teens, require ventilation support in their late teens and, eventually, die due to heart and lung failure.<sup>6,7,8,9<\/sup> The average life expectancy for Duchenne muscular dystrophy patients is in their mid-twenties (mid-thirties with comprehensive health care intervention).<sup>10<\/sup><\/p>\n\n\n\n<h4 class=\"wp-block-heading\" id=\"h-how-common-is-duchenne-muscular-dystrophy-is-it-treatable\">How Common is Duchenne Muscular Dystrophy? Is it Treatable?<\/h4>\n\n\n\n<figure class=\"wp-block-image alignleft size-full is-style-lightbox\"><img loading=\"lazy\" decoding=\"async\" width=\"302\" height=\"156\" src=\"https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/10\/Duchenne.jpg\" alt=\"Duchenne Muscular Dystrophy prevalence infographic\" class=\"wp-image-16407\" srcset=\"https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/10\/Duchenne.jpg 302w, https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/10\/Duchenne-300x155.jpg 300w\" sizes=\"auto, (max-width: 302px) 100vw, 302px\" \/><button class=\"modal-toggle\" type=\"button\" data-bs-toggle=\"modal\" data-bs-target=\"#mg-image-modal\" aria-label=\"View image in lightbox\"><img decoding=\"async\" width=\"302\" height=\"156\" src=\"https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/10\/Duchenne.jpg\" class=\"attachment-full size-full\" alt=\"Duchenne Muscular Dystrophy prevalence infographic\" hidden=\"\" loading=\"eager\" srcset=\"https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/10\/Duchenne.jpg 302w, https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/10\/Duchenne-300x155.jpg 300w\" sizes=\"(max-width: 302px) 100vw, 302px\" \/><\/button><\/figure>\n\n\n\n<p class=\"core-block core-paragraph\">Duchenne muscular dystrophy occurs in approximately one in 5,000 male births and about 20,000 babies worldwide are born with it each year . Genetic tests are available to determine if a patient\u2019s Duchenne muscular dystrophy is caused by a nonsense mutation.<sup>10<\/sup> We estimate that a nonsense mutation is the cause of Duchenne muscular dystrophy in approximately 13% of patients.<sup>11<\/sup><\/p>\n\n\n\n<p class=\"core-block core-paragraph\">Improvements in the function, quality of life, and longevity of patients with Duchenne muscular dystrophy are often achieved through a multidisciplinary approach to symptom management.<sup>6,12,13<\/sup> With prolonged survival, individuals with DMD face a unique set of challenges related to psychosocial issues and transitions of care, including but not limited to mental health and independence, functionality, and quality of life in critical domains of living, such as education, employment and interpersonal relationships.<sup>6,12<\/sup><\/p>\n\n\n\n<h4 class=\"wp-block-heading\" id=\"h-how-is-ptc-working-to-treat-dmd\">How is PTC Working to Treat DMD?<\/h4>\n\n\n\n<p class=\"core-block core-paragraph\">PTC currently has two approved medicines for treating Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene in ambulatory patients aged 2 years and older: Translarna\u2122 (ataluren) and Emflaza\u00ae (deflazacort).<sup>6,12<\/sup><\/p>\n\n\n\n<p class=\"core-block core-paragraph\"><strong>How Can You Stay Informed About Duchenne Muscular Dystrophy?<\/strong><\/p>\n\n\n\n<p class=\"core-block core-paragraph\"><a href=\"https:\/\/panel.ptcbio.com\/it-p2025tc\/patients-caregivers-resources\/\" target=\"_blank\" rel=\"noreferrer noopener\">Resources for Patients and Caregivers<\/a><\/p>\n\n\n\n<p class=\"core-block core-paragraph\"><a href=\"https:\/\/panel.ptcbio.com\/it-p2025tc\/healthcare-providers-resources\/\" target=\"_blank\" rel=\"noreferrer noopener\">Resources for Healthcare Providers<\/a><\/p>\n\n\n\n<div  class=\"references-block color-theme--purple-steel\">\n\t<div class=\"references-block__wrapper\">\n\n\t\t\t\t\t<div class=\"references-block__title heading--6\">\n\t\t\t\tREFERENCES\t\t\t<\/div>\n\t\t\n\t\t<div class=\"references-block__content wysiwyg wysiwyg--pure\">\n\t\t\t<p>[1] Theadom A, et al. Neuroepimediology. 2014;43:259-268.<br \/>\n[2] National Institute of Health. What are the types and symptoms of muscular dystrophy (MD)? Available at: https:\/\/www.nichd.nih.gov\/health\/topics\/musculardys\/conditioninfo\/types. [Accessed March 2023]<br \/>\n[3] Goemans N, et al. Eur Neurol Rev. 2014;9:78-82.<br \/>\n[4] Amato AA and Brown RH Jr. Muscular Dystrophies and other muscle diseases. In: Kasper DL, Fauci AS, Hausser SL, et al., eds., Harrison\u2019s Principles of internal Medicine, 19th Ed.<br \/>\n[5] Blake DJ, et al. Physiol Rev. 2002;82:291-329.<br \/>\n[6] Birnkrant DJ, et al Lancet Neurol. 2018;17 445-455.<br \/>\n[7] National Institute of Health. About Duchenne Muscular Dystrophy. Available at: https:\/\/www.genome.gov\/Genetic-Disorders\/Duchenne-Muscular-Dystrophy. [Accessed March 2023]<br \/>\n[8] Mendell JR, Lloyd-Puryear M. Muscle Nerve. 2013;48:21\u201326.<br \/>\n[9] van Dommelen P, et al. Dev Med Child Neurol. 2020; doi: 10.1111\/dmcn.14623.<br \/>\n[10] van Ruiten HJ, et al. Arch Dis Child. 2014;99:1074\u20131077<br \/>\n[11] Bushby K, et al. Muscle Nerve. 2014 Oct; 50(4): 477\u2013487.<br \/>\n[12] Birnkrant DJ, et al. Lancet Neurol.2018;17:251\u2013267.<br \/>\n[13] Birnkrant DJ, et al. Lancet Neurol. 2018:17:347\u2013361.<br \/>\n[14] PTC Therapeutics. Translarna\u2122 (ataluren) Summary of Product Characteristics. June 2021<br \/>\n[15] PTC Therapeutics. Emflaza\u2122 (deflazacort) Prescribing Information. July 2022<\/p>\n\t\t<\/div>\n\t<\/div>\n<\/div>\n\n\n<div  class=\"text-block text-block--standard color-theme--sky-blue-partial\">\n\t\t<div class=\"text-block__wrapper\" >\n\n\t\t\n\t\t\t\t\t<h2 class=\"text-block__heading\">\n\t\t\t\t<span class=\"heading--2\"  data-ptc-fitty=\"\">Do you have questions?<\/span>\n\t\t\t<\/h2>\n\t\t\n\t\t<div class=\"text-block__content wysiwyg--decoration wysiwyg--color\">\n\t\t\t<p><span class=\"NormalTextRun SCXP77754502 BCX0\">Please reach out if you would like to speak with\u00a0<\/span><span class=\"NormalTextRun SCXP77754502 BCX0\">us.<\/span><\/p>\n\t\t<\/div>\n\n\t\t\n\t\t\t<div class=\"dual-cta__ctas dual-cta__ctas--same dual-cta__ctas--center\">\n\n\t\t\t\t\t<a href=\"mailto:PatientInfo@ptcbio.com\" target=\"\" class=\"dual-cta__button btn btn--primary\">Patients &amp; Families<\/a>\n\t\t\n\t\t\t\t\t<a href=\"mailto:MedInfo@ptcbio.com\" target=\"\" class=\"dual-cta__button btn btn--primary\">Healthcare Professionals<\/a>\n\t\t\n\t\t\n\t<\/div>\n\n\t<\/div>\n<\/div>\n\n","protected":false},"excerpt":{"rendered":"<p>What is Duchenne Muscular Dystrophy? Muscular dystrophies involve progressive muscle wasting and weakness and are caused by a mutation in the DNA that results in either the absence of or very low levels of the dystrophin protein.1 Duchenne muscular dystrophy (DMD) is the most common \u2013 and most severe \u2013 type of muscular dystrophy.2 Duchenne&hellip;<\/p>\n","protected":false},"author":2,"featured_media":0,"parent":9951,"menu_order":0,"comment_status":"closed","ping_status":"closed","template":"","meta":{"_acf_changed":false,"footnotes":""},"class_list":["post-10141","page","type-page","status-publish","hentry"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO Premium plugin v27.5 (Yoast SEO v27.5) - https:\/\/yoast.com\/product\/yoast-seo-premium-wordpress\/ -->\n<title>Duchenne muscular dystrophy - PTC Therapeutics (Italy)<\/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:\/\/panel.ptcbio.com\/it-p2025tc\/therapeutic-areas\/duchenne-muscular-dystrophy\/\" \/>\n<meta property=\"og:locale\" content=\"it_IT\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Duchenne muscular dystrophy\" \/>\n<meta property=\"og:description\" content=\"What is Duchenne Muscular Dystrophy? Muscular dystrophies involve progressive muscle wasting and weakness and are caused by a mutation in the DNA that results in either the absence of or very low levels of the dystrophin protein.1 Duchenne muscular dystrophy (DMD) is the most common \u2013 and most severe \u2013 type of muscular dystrophy.2 Duchenne&hellip;\" \/>\n<meta property=\"og:url\" content=\"https:\/\/panel.ptcbio.com\/it-p2025tc\/therapeutic-areas\/duchenne-muscular-dystrophy\/\" \/>\n<meta property=\"og:site_name\" content=\"PTC Therapeutics (Italy)\" \/>\n<meta property=\"og:image\" content=\"https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/10\/Duchenne-Signs-Symptoms-graphic.jpg\" \/>\n<meta name=\"twitter:card\" content=\"summary_large_image\" \/>\n<meta name=\"twitter:label1\" content=\"Tempo di lettura stimato\" \/>\n\t<meta name=\"twitter:data1\" content=\"3 minuti\" \/>\n<script type=\"application\/ld+json\" class=\"yoast-schema-graph\">{\"@context\":\"https:\\\/\\\/schema.org\",\"@graph\":[{\"@type\":\"WebPage\",\"@id\":\"https:\\\/\\\/panel.ptcbio.com\\\/it-p2025tc\\\/therapeutic-areas\\\/duchenne-muscular-dystrophy\\\/\",\"url\":\"https:\\\/\\\/panel.ptcbio.com\\\/it-p2025tc\\\/therapeutic-areas\\\/duchenne-muscular-dystrophy\\\/\",\"name\":\"Duchenne muscular dystrophy - PTC Therapeutics (Italy)\",\"isPartOf\":{\"@id\":\"https:\\\/\\\/panel.ptcbio.com\\\/it-p2025tc\\\/#website\"},\"primaryImageOfPage\":{\"@id\":\"https:\\\/\\\/panel.ptcbio.com\\\/it-p2025tc\\\/therapeutic-areas\\\/duchenne-muscular-dystrophy\\\/#primaryimage\"},\"image\":{\"@id\":\"https:\\\/\\\/panel.ptcbio.com\\\/it-p2025tc\\\/therapeutic-areas\\\/duchenne-muscular-dystrophy\\\/#primaryimage\"},\"thumbnailUrl\":\"https:\\\/\\\/panel.ptcbio.com\\\/it-p2025tc\\\/wp-content\\\/uploads\\\/sites\\\/21\\\/2024\\\/10\\\/Duchenne-Signs-Symptoms-graphic.jpg\",\"datePublished\":\"2022-12-30T17:48:10+00:00\",\"breadcrumb\":{\"@id\":\"https:\\\/\\\/panel.ptcbio.com\\\/it-p2025tc\\\/therapeutic-areas\\\/duchenne-muscular-dystrophy\\\/#breadcrumb\"},\"inLanguage\":\"it-IT\",\"potentialAction\":[{\"@type\":\"ReadAction\",\"target\":[\"https:\\\/\\\/panel.ptcbio.com\\\/it-p2025tc\\\/therapeutic-areas\\\/duchenne-muscular-dystrophy\\\/\"]}]},{\"@type\":\"ImageObject\",\"inLanguage\":\"it-IT\",\"@id\":\"https:\\\/\\\/panel.ptcbio.com\\\/it-p2025tc\\\/therapeutic-areas\\\/duchenne-muscular-dystrophy\\\/#primaryimage\",\"url\":\"https:\\\/\\\/panel.ptcbio.com\\\/it-p2025tc\\\/wp-content\\\/uploads\\\/sites\\\/21\\\/2024\\\/10\\\/Duchenne-Signs-Symptoms-graphic.jpg\",\"contentUrl\":\"https:\\\/\\\/panel.ptcbio.com\\\/it-p2025tc\\\/wp-content\\\/uploads\\\/sites\\\/21\\\/2024\\\/10\\\/Duchenne-Signs-Symptoms-graphic.jpg\",\"width\":630,\"height\":690,\"caption\":\"Duchenne Muscular Dystrophy Signs and Symptoms Infographic\"},{\"@type\":\"BreadcrumbList\",\"@id\":\"https:\\\/\\\/panel.ptcbio.com\\\/it-p2025tc\\\/therapeutic-areas\\\/duchenne-muscular-dystrophy\\\/#breadcrumb\",\"itemListElement\":[{\"@type\":\"ListItem\",\"position\":1,\"name\":\"Home\",\"item\":\"https:\\\/\\\/panel.ptcbio.com\\\/it-p2025tc\\\/\"},{\"@type\":\"ListItem\",\"position\":2,\"name\":\"Therapeutic Areas\",\"item\":\"https:\\\/\\\/panel.ptcbio.com\\\/it-p2025tc\\\/therapeutic-areas\\\/\"},{\"@type\":\"ListItem\",\"position\":3,\"name\":\"Duchenne muscular dystrophy\"}]},{\"@type\":\"WebSite\",\"@id\":\"https:\\\/\\\/panel.ptcbio.com\\\/it-p2025tc\\\/#website\",\"url\":\"https:\\\/\\\/panel.ptcbio.com\\\/it-p2025tc\\\/\",\"name\":\"PTC Therapeutics Italia\",\"description\":\"\",\"publisher\":{\"@id\":\"https:\\\/\\\/panel.ptcbio.com\\\/it-p2025tc\\\/#organization\"},\"potentialAction\":[{\"@type\":\"SearchAction\",\"target\":{\"@type\":\"EntryPoint\",\"urlTemplate\":\"https:\\\/\\\/panel.ptcbio.com\\\/it-p2025tc\\\/?s={search_term_string}\"},\"query-input\":{\"@type\":\"PropertyValueSpecification\",\"valueRequired\":true,\"valueName\":\"search_term_string\"}}],\"inLanguage\":\"it-IT\"},{\"@type\":\"Organization\",\"@id\":\"https:\\\/\\\/panel.ptcbio.com\\\/it-p2025tc\\\/#organization\",\"name\":\"PTC Therapeutics Italia\",\"url\":\"https:\\\/\\\/panel.ptcbio.com\\\/it-p2025tc\\\/\",\"logo\":{\"@type\":\"ImageObject\",\"inLanguage\":\"it-IT\",\"@id\":\"https:\\\/\\\/panel.ptcbio.com\\\/it-p2025tc\\\/#\\\/schema\\\/logo\\\/image\\\/\",\"url\":\"https:\\\/\\\/panel.ptcbio.com\\\/it-p2025tc\\\/wp-content\\\/uploads\\\/sites\\\/21\\\/2026\\\/04\\\/ptcLogo.png\",\"contentUrl\":\"https:\\\/\\\/panel.ptcbio.com\\\/it-p2025tc\\\/wp-content\\\/uploads\\\/sites\\\/21\\\/2026\\\/04\\\/ptcLogo.png\",\"width\":542,\"height\":542,\"caption\":\"PTC Therapeutics Italia\"},\"image\":{\"@id\":\"https:\\\/\\\/panel.ptcbio.com\\\/it-p2025tc\\\/#\\\/schema\\\/logo\\\/image\\\/\"}}]}<\/script>\n<!-- \/ Yoast SEO Premium plugin. -->","yoast_head_json":{"title":"Duchenne muscular dystrophy - PTC Therapeutics (Italy)","robots":{"index":"index","follow":"follow","max-snippet":"max-snippet:-1","max-image-preview":"max-image-preview:large","max-video-preview":"max-video-preview:-1"},"canonical":"https:\/\/panel.ptcbio.com\/it-p2025tc\/therapeutic-areas\/duchenne-muscular-dystrophy\/","og_locale":"it_IT","og_type":"article","og_title":"Duchenne muscular dystrophy","og_description":"What is Duchenne Muscular Dystrophy? Muscular dystrophies involve progressive muscle wasting and weakness and are caused by a mutation in the DNA that results in either the absence of or very low levels of the dystrophin protein.1 Duchenne muscular dystrophy (DMD) is the most common \u2013 and most severe \u2013 type of muscular dystrophy.2 Duchenne&hellip;","og_url":"https:\/\/panel.ptcbio.com\/it-p2025tc\/therapeutic-areas\/duchenne-muscular-dystrophy\/","og_site_name":"PTC Therapeutics (Italy)","og_image":[{"url":"https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/10\/Duchenne-Signs-Symptoms-graphic.jpg","type":"","width":"","height":""}],"twitter_card":"summary_large_image","twitter_misc":{"Tempo di lettura stimato":"3 minuti"},"schema":{"@context":"https:\/\/schema.org","@graph":[{"@type":"WebPage","@id":"https:\/\/panel.ptcbio.com\/it-p2025tc\/therapeutic-areas\/duchenne-muscular-dystrophy\/","url":"https:\/\/panel.ptcbio.com\/it-p2025tc\/therapeutic-areas\/duchenne-muscular-dystrophy\/","name":"Duchenne muscular dystrophy - PTC Therapeutics (Italy)","isPartOf":{"@id":"https:\/\/panel.ptcbio.com\/it-p2025tc\/#website"},"primaryImageOfPage":{"@id":"https:\/\/panel.ptcbio.com\/it-p2025tc\/therapeutic-areas\/duchenne-muscular-dystrophy\/#primaryimage"},"image":{"@id":"https:\/\/panel.ptcbio.com\/it-p2025tc\/therapeutic-areas\/duchenne-muscular-dystrophy\/#primaryimage"},"thumbnailUrl":"https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/10\/Duchenne-Signs-Symptoms-graphic.jpg","datePublished":"2022-12-30T17:48:10+00:00","breadcrumb":{"@id":"https:\/\/panel.ptcbio.com\/it-p2025tc\/therapeutic-areas\/duchenne-muscular-dystrophy\/#breadcrumb"},"inLanguage":"it-IT","potentialAction":[{"@type":"ReadAction","target":["https:\/\/panel.ptcbio.com\/it-p2025tc\/therapeutic-areas\/duchenne-muscular-dystrophy\/"]}]},{"@type":"ImageObject","inLanguage":"it-IT","@id":"https:\/\/panel.ptcbio.com\/it-p2025tc\/therapeutic-areas\/duchenne-muscular-dystrophy\/#primaryimage","url":"https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/10\/Duchenne-Signs-Symptoms-graphic.jpg","contentUrl":"https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/10\/Duchenne-Signs-Symptoms-graphic.jpg","width":630,"height":690,"caption":"Duchenne Muscular Dystrophy Signs and Symptoms Infographic"},{"@type":"BreadcrumbList","@id":"https:\/\/panel.ptcbio.com\/it-p2025tc\/therapeutic-areas\/duchenne-muscular-dystrophy\/#breadcrumb","itemListElement":[{"@type":"ListItem","position":1,"name":"Home","item":"https:\/\/panel.ptcbio.com\/it-p2025tc\/"},{"@type":"ListItem","position":2,"name":"Therapeutic Areas","item":"https:\/\/panel.ptcbio.com\/it-p2025tc\/therapeutic-areas\/"},{"@type":"ListItem","position":3,"name":"Duchenne muscular dystrophy"}]},{"@type":"WebSite","@id":"https:\/\/panel.ptcbio.com\/it-p2025tc\/#website","url":"https:\/\/panel.ptcbio.com\/it-p2025tc\/","name":"PTC Therapeutics Italia","description":"","publisher":{"@id":"https:\/\/panel.ptcbio.com\/it-p2025tc\/#organization"},"potentialAction":[{"@type":"SearchAction","target":{"@type":"EntryPoint","urlTemplate":"https:\/\/panel.ptcbio.com\/it-p2025tc\/?s={search_term_string}"},"query-input":{"@type":"PropertyValueSpecification","valueRequired":true,"valueName":"search_term_string"}}],"inLanguage":"it-IT"},{"@type":"Organization","@id":"https:\/\/panel.ptcbio.com\/it-p2025tc\/#organization","name":"PTC Therapeutics Italia","url":"https:\/\/panel.ptcbio.com\/it-p2025tc\/","logo":{"@type":"ImageObject","inLanguage":"it-IT","@id":"https:\/\/panel.ptcbio.com\/it-p2025tc\/#\/schema\/logo\/image\/","url":"https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2026\/04\/ptcLogo.png","contentUrl":"https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2026\/04\/ptcLogo.png","width":542,"height":542,"caption":"PTC Therapeutics Italia"},"image":{"@id":"https:\/\/panel.ptcbio.com\/it-p2025tc\/#\/schema\/logo\/image\/"}}]}},"_links":{"self":[{"href":"https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-json\/wp\/v2\/pages\/10141","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-json\/wp\/v2\/pages"}],"about":[{"href":"https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-json\/wp\/v2\/types\/page"}],"author":[{"embeddable":true,"href":"https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-json\/wp\/v2\/comments?post=10141"}],"version-history":[{"count":0,"href":"https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-json\/wp\/v2\/pages\/10141\/revisions"}],"up":[{"embeddable":true,"href":"https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-json\/wp\/v2\/pages\/9951"}],"wp:attachment":[{"href":"https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-json\/wp\/v2\/media?parent=10141"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}