{"id":12624,"date":"2023-03-29T14:54:02","date_gmt":"2023-03-29T18:54:02","guid":{"rendered":"https:\/\/panel.ptcbio.com\/it-p2025tc\/?page_id=12624"},"modified":"2023-03-29T14:54:02","modified_gmt":"2023-03-29T18:54:02","slug":"hereditary-transthyretin-amyloidosis","status":"publish","type":"page","link":"https:\/\/panel.ptcbio.com\/it-p2025tc\/therapeutic-areas\/hereditary-transthyretin-amyloidosis\/","title":{"rendered":"Hereditary Transthyretin Amyloidosis"},"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<div class=\"hero__bg\">\n\t\t\t<div id=\"particles-js\" class=\"hero__constellation hero__constellation--center\"><\/div>\n\t\t<\/div>\n\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=\"\">Hereditary Transthyretin Amyloidosis (hATTR)<\/h1><\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"hero__subtitle\">Rare and fatal disease where deposits of amyloids cause progressive damage to multiple organ systems<\/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\" 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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\">Hereditary Transthyretin Amyloidosis<\/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<figure class=\"wp-block-image alignright size-full is-resized is-style-lightbox\"><img loading=\"lazy\" decoding=\"async\" width=\"2560\" height=\"2047\" src=\"https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/12\/hATTR-Signs-and-Symptoms-scaled-1.jpg\" alt=\"hATTR Signs and Symptoms\" class=\"wp-image-16564\" style=\"width:758px\" srcset=\"https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/12\/hATTR-Signs-and-Symptoms-scaled-1.jpg 2560w, https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/12\/hATTR-Signs-and-Symptoms-scaled-1-300x240.jpg 300w, https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/12\/hATTR-Signs-and-Symptoms-scaled-1-1024x819.jpg 1024w, https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/12\/hATTR-Signs-and-Symptoms-scaled-1-768x614.jpg 768w, https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/12\/hATTR-Signs-and-Symptoms-scaled-1-1536x1228.jpg 1536w, https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/12\/hATTR-Signs-and-Symptoms-scaled-1-2048x1638.jpg 2048w, https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/12\/hATTR-Signs-and-Symptoms-scaled-1-400x320.jpg 400w, https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/12\/hATTR-Signs-and-Symptoms-scaled-1-1126x900.jpg 1126w\" sizes=\"auto, (max-width: 2560px) 100vw, 2560px\" \/><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=\"2560\" height=\"2047\" src=\"https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/12\/hATTR-Signs-and-Symptoms-scaled-1.jpg\" class=\"attachment-full size-full\" alt=\"hATTR Signs and Symptoms\" hidden=\"\" loading=\"eager\" srcset=\"https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/12\/hATTR-Signs-and-Symptoms-scaled-1.jpg 2560w, https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/12\/hATTR-Signs-and-Symptoms-scaled-1-300x240.jpg 300w, https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/12\/hATTR-Signs-and-Symptoms-scaled-1-1024x819.jpg 1024w, https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/12\/hATTR-Signs-and-Symptoms-scaled-1-768x614.jpg 768w, https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/12\/hATTR-Signs-and-Symptoms-scaled-1-1536x1228.jpg 1536w, https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/12\/hATTR-Signs-and-Symptoms-scaled-1-2048x1638.jpg 2048w, https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/12\/hATTR-Signs-and-Symptoms-scaled-1-400x320.jpg 400w, https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/12\/hATTR-Signs-and-Symptoms-scaled-1-1126x900.jpg 1126w\" sizes=\"(max-width: 2560px) 100vw, 2560px\" \/><\/button><\/figure>\n\n\n\n<h4 class=\"wp-block-heading\" id=\"h-what-is-hereditary-transthyretin-amyloidosis\">What is Hereditary Transthyretin Amyloidosis?<\/h4>\n\n\n\n<p class=\"core-block core-paragraph\">Hereditary transthyretin amyloidosis (hATTR) is a rare, progressive and fatal disease. In hATTR-PN, the clinical manifestations are multisystemic and debilitating, with significant psychological and familial impact<sup>1,2<\/sup>. The disease can be influenced by the TTR genotype, geographic location, or other genetic and environmental factors. Therefore, the symptomatology, age of onset of symptoms, and rate of progression vary among patients.\u200b\u200b<\/p>\n\n\n\n<p class=\"core-block core-paragraph\">The symptoms, which are nonspecific and resemble many conditions, include<sup>1,3<\/sup>:\u200b<\/p>\n\n\n\n<ul class=\"core-block core-list core-list--ul\" class=\"wp-block-list\">\n<li>Autonomic neuropathy\u200b<\/li>\n\n\n\n<li>Peripheral sensory-motor neuropathy\u200b<\/li>\n\n\n\n<li>Gastrointestinal changes\u200b<\/li>\n\n\n\n<li>Ocular manifestations\u200b<\/li>\n\n\n\n<li>Cardiomyopathy\u200b<\/li>\n\n\n\n<li>Impairment of renal function\u200b<\/li>\n\n\n\n<li>Bilateral carpal tunnel syndrome\u200b<\/li>\n<\/ul>\n\n\n\n<p class=\"core-block core-paragraph\">\u200b<\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"h-differential-diagnoses-3\">\u200bDifferential Diagnoses<sup>3<\/sup><\/h3>\n\n\n\n<p class=\"core-block core-paragraph\">The signs and symptoms of hATTR are diverse and variable, which contributes to patients receiving incorrect diagnoses during their journey and further delaying access to the correct treatment. Among these differential diagnoses, the most important are:\u200b<\/p>\n\n\n\n<ul class=\"core-block core-list core-list--ul\" class=\"wp-block-list\">\n<li>Toxic neuropathies<\/li>\n\n\n\n<li>Diabetic neuropathy\u200b<\/li>\n\n\n\n<li>Leprosy\u200b<\/li>\n\n\n\n<li>Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP)\u200b<\/li>\n\n\n\n<li>Charcot-Marie-Tooth disease\u200b<\/li>\n\n\n\n<li>Fabry disease\u200b<\/li>\n\n\n\n<li>Hereditary autonomic and sensory neuropathies\u200b<\/li>\n\n\n\n<li>AL or primary amyloidosis (light chain immunoglobulin fragments)\u200b<\/li>\n<\/ul>\n\n\n\n<h4 class=\"wp-block-heading\" id=\"h-how-common-is-hereditary-transthyretin-amyloidosis\">How common is Hereditary Transthyretin Amyloidosis?<\/h4>\n\n\n\n<p class=\"core-block core-paragraph\">There are about 50,000 people with hATTR-PN worldwide1. V30M mutations are predominantly found in Portugal, Spain, France, Japan, Sweden, and in countries with descendants from these regions, such as Brazil3, however, more than 150 pathogenic variants are already known worldwide<sup>4<\/sup>.<\/p>\n\n\n\n<figure class=\"wp-block-image aligncenter size-full is-resized is-style-lightbox\"><img loading=\"lazy\" decoding=\"async\" width=\"1248\" height=\"646\" src=\"https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/12\/hATTR-prevalence.jpg\" alt=\"hATTR prevalence\" class=\"wp-image-16565\" style=\"width:452px\" srcset=\"https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/12\/hATTR-prevalence.jpg 1248w, https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/12\/hATTR-prevalence-300x155.jpg 300w, https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/12\/hATTR-prevalence-1024x530.jpg 1024w, https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/12\/hATTR-prevalence-768x398.jpg 768w, https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/12\/hATTR-prevalence-400x207.jpg 400w\" sizes=\"auto, (max-width: 1248px) 100vw, 1248px\" \/><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=\"1248\" height=\"646\" src=\"https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/12\/hATTR-prevalence.jpg\" class=\"attachment-full size-full\" alt=\"hATTR prevalence\" hidden=\"\" loading=\"eager\" srcset=\"https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/12\/hATTR-prevalence.jpg 1248w, https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/12\/hATTR-prevalence-300x155.jpg 300w, https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/12\/hATTR-prevalence-1024x530.jpg 1024w, https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/12\/hATTR-prevalence-768x398.jpg 768w, https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/12\/hATTR-prevalence-400x207.jpg 400w\" sizes=\"(max-width: 1248px) 100vw, 1248px\" \/><\/button><\/figure>\n\n\n\n<h4 class=\"wp-block-heading\" id=\"h-how-is-ptc-working-to-treat-hereditary-transthyretin-amyloidosis\">How is PTC Working to Treat Hereditary Transthyretin Amyloidosis?<\/h4>\n\n\n\n<p class=\"core-block core-paragraph\">PTC has a therapy to treat patients with hereditary transthyretin amyloidosis with polyneuropathy available in the U.S., European Union and Brazil. PTC has in-licensed the therapy in Latin America.<\/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] Gertz MA. Hereditary ATTR amyloidosis: burden of illness and diagnostic challenges. Am J Manag Care. 2017 Jun;23(7 Suppl):S107-S112. Review. PubMed PMID: 28978215.\u200b<\/p>\n<p>[2] LOPES, Alice et al. Life Paths Of Patients With Transthyretin-related Familial Amyloid Polyneuropathy: A Descriptive Study. Journal Of Community Genetics, [s.l.], v. 9, n. 1, p.93-99, 19 out. 2017. Springer Nature. http:\/\/dx.doi.org\/10.1007\/s12687-017-0338-0. Dispon\u00edvel em: . \u200b<\/p>\n<p>[3] Pinto MV, et al. Brazilian consensus for diagnosis, management and treatment of hereditary transthyretin amyloidosis with peripheral neuropathy: second edition Arq. Neuropsiquiatr. 2023;81(3):308\u2013321. DOI: https:\/\/doi.org\/10.1055\/s-0043-1764412.\u200b<\/p>\n<p>[4] Ando Y. Guidelines and new directions in the therapy and monitoring of ATTRv amyloidosis, Amyloid, 2022. DOI: https:\/\/doi.org\/10.1080\/13506129.2022.2052838<\/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<h3 class=\"text-block__heading\">\n\t\t\t\t<span class=\"heading--3\"  data-ptc-fitty=\"\">Do you have questions?<\/span>\n\t\t\t<\/h3>\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 and 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 Hereditary Transthyretin Amyloidosis? Hereditary transthyretin amyloidosis (hATTR) is a rare, progressive and fatal disease. In hATTR-PN, the clinical manifestations are multisystemic and debilitating, with significant psychological and familial impact1,2. The disease can be influenced by the TTR genotype, geographic location, or other genetic and environmental factors. Therefore, the symptomatology, age of onset of&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-12624","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>Hereditary Transthyretin Amyloidosis - 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\/hereditary-transthyretin-amyloidosis\/\" \/>\n<meta property=\"og:locale\" content=\"it_IT\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Hereditary Transthyretin Amyloidosis\" \/>\n<meta property=\"og:description\" content=\"What is Hereditary Transthyretin Amyloidosis? Hereditary transthyretin amyloidosis (hATTR) is a rare, progressive and fatal disease. In hATTR-PN, the clinical manifestations are multisystemic and debilitating, with significant psychological and familial impact1,2. The disease can be influenced by the TTR genotype, geographic location, or other genetic and environmental factors. 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