{"id":10977,"date":"2023-01-05T21:34:16","date_gmt":"2023-01-05T21:34:16","guid":{"rendered":"https:\/\/panel.ptcbio.com\/it-p2025tc\/?page_id=10977"},"modified":"2023-01-05T21:34:16","modified_gmt":"2023-01-05T21:34:16","slug":"familial-partial-lipodystrophy","status":"publish","type":"page","link":"https:\/\/panel.ptcbio.com\/it-p2025tc\/therapeutic-areas\/familial-partial-lipodystrophy\/","title":{"rendered":"Familial partial lipodystrophy"},"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=\"\">Familial partial lipodystrophy (FPL)<\/h1><\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"hero__subtitle\">Group of diseases characterized by an abnormal distribution of fat around the body<\/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\" 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itemprop=\"name\">Familial partial lipodystrophy<\/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\/FPL-Signs-and-Symptoms-scaled-1.jpg\" alt=\"FPL Signs and Symptoms\" class=\"wp-image-16554\" style=\"width:758px\" srcset=\"https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/12\/FPL-Signs-and-Symptoms-scaled-1.jpg 2560w, https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/12\/FPL-Signs-and-Symptoms-scaled-1-300x240.jpg 300w, https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/12\/FPL-Signs-and-Symptoms-scaled-1-1024x819.jpg 1024w, https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/12\/FPL-Signs-and-Symptoms-scaled-1-768x614.jpg 768w, https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/12\/FPL-Signs-and-Symptoms-scaled-1-1536x1228.jpg 1536w, https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/12\/FPL-Signs-and-Symptoms-scaled-1-2048x1638.jpg 2048w, https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/12\/FPL-Signs-and-Symptoms-scaled-1-400x320.jpg 400w, https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/12\/FPL-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\/FPL-Signs-and-Symptoms-scaled-1.jpg\" class=\"attachment-full size-full\" alt=\"FPL Signs and Symptoms\" hidden=\"\" loading=\"eager\" srcset=\"https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/12\/FPL-Signs-and-Symptoms-scaled-1.jpg 2560w, https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/12\/FPL-Signs-and-Symptoms-scaled-1-300x240.jpg 300w, https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/12\/FPL-Signs-and-Symptoms-scaled-1-1024x819.jpg 1024w, https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/12\/FPL-Signs-and-Symptoms-scaled-1-768x614.jpg 768w, https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/12\/FPL-Signs-and-Symptoms-scaled-1-1536x1228.jpg 1536w, https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/12\/FPL-Signs-and-Symptoms-scaled-1-2048x1638.jpg 2048w, https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/12\/FPL-Signs-and-Symptoms-scaled-1-400x320.jpg 400w, https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/12\/FPL-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-familial-partial-lipodystrophy-fpl\">What is Familial Partial Lipodystrophy (FPL)?<\/h4>\n\n\n\n<p class=\"core-block core-paragraph\">Lipodystrophies, including familial partial lipodystrophies, are disorders characterized by a variable loss of adipose tissue (fat), without evidence of weight loss due to lack of food intake or increased energy expenditure, and metabolic complications<sup>1<\/sup>. \u200b<\/p>\n\n\n\n<p class=\"core-block core-paragraph\">\u200bThe signs and symptoms of FPL include<sup>2<\/sup>:\u200b<\/p>\n\n\n\n<ul class=\"core-block core-list core-list--ul\" class=\"wp-block-list\">\n<li>Selective and progressive loss of body fat (adipose tissue) in various areas of the body\u200b<\/li>\n\n\n\n<li>Reduction of subcutaneous fat in the arms and legs\u200b<\/li>\n\n\n\n<li>Onset during puberty\u200b<\/li>\n\n\n\n<li>Excessive accumulation of fat in other areas of the body, mainly the neck, face, and intra-abdominal region\u200b<\/li>\n\n\n\n<li>Hypertriglyceridemia\u200b<\/li>\n\n\n\n<li>Insulin resistance\u200b<\/li>\n\n\n\n<li>Diabetes\u200b<\/li>\n\n\n\n<li>Extent of adipose tissue loss: determines the severity of associated metabolic complications\u200b<\/li>\n\n\n\n<li>Other metabolic complications\u200b<\/li>\n\n\n\n<li>FPL is a highly burdensome disease, leading to significant metabolic complications that are not managed by current therapies, and it is a distressing condition for the patient.<\/li>\n<\/ul>\n\n\n\n<h4 class=\"wp-block-heading\" id=\"h-how-common-is-fpl\">How common is FPL?<\/h4>\n\n\n\n<p class=\"core-block core-paragraph\">The global prevalence of Lipodystrophy is 3.07 cases per million inhabitants, with 2.84 cases per million being Partial Lipodystrophy<sup>3<\/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=\"2017\" height=\"646\" src=\"https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/12\/FPL-prevalence.jpg\" alt=\"FPL prevalence\" class=\"wp-image-16555\" style=\"width:576px;height:auto\" srcset=\"https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/12\/FPL-prevalence.jpg 2017w, https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/12\/FPL-prevalence-300x96.jpg 300w, https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/12\/FPL-prevalence-1024x328.jpg 1024w, https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/12\/FPL-prevalence-768x246.jpg 768w, https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/12\/FPL-prevalence-1536x492.jpg 1536w, https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/12\/FPL-prevalence-400x128.jpg 400w\" sizes=\"auto, (max-width: 2017px) 100vw, 2017px\" \/><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=\"2017\" height=\"646\" src=\"https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/12\/FPL-prevalence.jpg\" class=\"attachment-full size-full\" alt=\"FPL prevalence\" hidden=\"\" loading=\"eager\" srcset=\"https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/12\/FPL-prevalence.jpg 2017w, https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/12\/FPL-prevalence-300x96.jpg 300w, https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/12\/FPL-prevalence-1024x328.jpg 1024w, https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/12\/FPL-prevalence-768x246.jpg 768w, https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/12\/FPL-prevalence-1536x492.jpg 1536w, https:\/\/panel.ptcbio.com\/it-p2025tc\/wp-content\/uploads\/sites\/21\/2024\/12\/FPL-prevalence-400x128.jpg 400w\" sizes=\"(max-width: 2017px) 100vw, 2017px\" \/><\/button><\/figure>\n\n\n\n<h4 class=\"wp-block-heading\" id=\"h-how-is-ptc-working-to-treat-familial-partial-lipodystrophy\">How is PTC working to treat Familial Partial Lipodystrophy?<\/h4>\n\n\n\n<p class=\"core-block core-paragraph\">PTC has a therapy for FPL that has been in-licensed in Latin America from Akcea Therapeutics, Inc., a wholly owned subsidiary of Ionis Pharmaceuticals, Inc.<\/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] Brown RJ, Araujo-Vilar D, Cheung PT, et al. The Diagnosis and Management of Lipodystrophy Syndromes: A Multi-Society Practice Guideline. J Clin Endocrinol Metab. 2016;101(12):4500-4511. doi:10.1210\/jc.2016-2466\u200b<\/p>\n<p>[2] Zammouri J, Vatier C, Capel E, et al. Molecular and Cellular Bases of Lipodystrophy Syndromes. Front Endocrinol (Lausanne). 2022;12:803189. Published 2022 Jan 3. doi:10.3389\/fendo.2021.803189\u200b<\/p>\n<p>[3] Chiquette E, Oral EA, Garg A, Ara\u00fajo-Vilar D, Dhankhar P. Estimating the prevalence of generalized and partial lipodystrophy: findings and challenges. Diabetes Metab Syndr Obes. 2017;10:375-383. Published 2017 Sep 13. doi:10.2147\/DMSO.S130810<\/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 &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 Familial Partial Lipodystrophy (FPL)? Lipodystrophies, including familial partial lipodystrophies, are disorders characterized by a variable loss of adipose tissue (fat), without evidence of weight loss due to lack of food intake or increased energy expenditure, and metabolic complications1. \u200b \u200bThe signs and symptoms of FPL include2:\u200b How common is FPL? The global prevalence&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-10977","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>Familial partial lipodystrophy - 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\/familial-partial-lipodystrophy\/\" \/>\n<meta property=\"og:locale\" content=\"it_IT\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Familial partial lipodystrophy\" \/>\n<meta property=\"og:description\" content=\"What is Familial Partial Lipodystrophy (FPL)? Lipodystrophies, including familial partial lipodystrophies, are disorders characterized by a variable loss of adipose tissue (fat), without evidence of weight loss due to lack of food intake or increased energy expenditure, and metabolic complications1. \u200b \u200bThe signs and symptoms of FPL include2:\u200b How common is FPL? 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Lipodystrophies, including familial partial lipodystrophies, are disorders characterized by a variable loss of adipose tissue (fat), without evidence of weight loss due to lack of food intake or increased energy expenditure, and metabolic complications1. \u200b \u200bThe signs and symptoms of FPL include2:\u200b How common is FPL? 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