{"id":9951,"date":"2022-12-27T17:55:41","date_gmt":"2022-12-27T17:55:41","guid":{"rendered":"https:\/\/panel.ptcbio.com\/it-p2025tc\/?page_id=9951"},"modified":"2022-12-27T17:55:41","modified_gmt":"2022-12-27T17:55:41","slug":"therapeutic-areas","status":"publish","type":"page","link":"https:\/\/panel.ptcbio.com\/it-p2025tc\/therapeutic-areas\/","title":{"rendered":"Therapeutic Areas"},"content":{"rendered":"
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Therapeutic Areas<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t

Diseases we\u2019re on a mission to change<\/h1><\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t
\n\t\t\t\t\t\t\t\t\t\"Patient\t\t\t\t\t\t\t\t<\/figure>\n\t\t\t\t\t<\/div>\n\t\t\t<\/div>\n\t\t\t
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  1. \n\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\tHomepage<\/span>\n\t\t\t\t\t\t<\/a>\n\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t<\/li>\n\t\t\t\t\t\t\t\t\t\t\t\t
  2. \n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\tTherapeutic Areas<\/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\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

    We are deeply committed to patients living with rare diseases who have little-to-no treatment options, and this passion fuels and inspires us. <\/strong>Our commercial and development efforts are focused in two therapeutic areas – neurology and metabolic conditions – in which we have deep experience and a record of success.<\/p>\n\n\n\n

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    \n\t<\/span>\n\tMetabolism<\/span>\n\t<\/span>\n<\/h2>\n\t\t<\/div>\n\t\n\t\n\t
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    \n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\tPhenylketonuria (PKU)\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\t\t\t\n\t\t\t\t\t\t\t\tMetabolic condition caused by mutations to phenylalanine hydroxylase that can lead to cognitive disabilities and seizures\t\t\t\t\t\t\t<\/span>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t<\/a>\n\t\t\t<\/div>\n\t\t\t\t\t\t\t\t
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    \n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\tFamilial chylomicronemia syndrome (FCS)\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\t\t\t\n\t\t\t\t\t\t\t\tSerious disease that prevents the body from breaking down fats\t\t\t\t\t\t\t<\/span>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t<\/a>\n\t\t\t<\/div>\n\t\t\t\t\t\t\t\t
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    \n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\tFamilial partial lipodystrophy (FPL)\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\t\t\t\n\t\t\t\t\t\t\t\tGroup of diseases characterized by an abnormal distribution of fat around the body\t\t\t\t\t\t\t<\/span>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t<\/a>\n\t\t\t<\/div>\n\t\t\t<\/div>\n\n<\/div>\n\n\n\n\n
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    \n\t<\/span>\n\tNeurology<\/span>\n\t<\/span>\n<\/h2>\n\t\t<\/div>\n\t\n\t\n\t
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    \n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\tAromatic l-amino acid decarboxylase (AADC) deficiency\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\t\t\t\n\t\t\t\t\t\t\t\tPatients with AADC deficiency lack dopamine, an essential neurotransmitter that controls movement\t\t\t\t\t\t\t<\/span>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t<\/a>\n\t\t\t<\/div>\n\t\t\t\t\t\t\t\t
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    \n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\tDuchenne muscular dystrophy (DMD)\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\t\t\t\n\t\t\t\t\t\t\t\tRare and fatal genetic disorder resulting in progressive muscle weakness from early childhood\t\t\t\t\t\t\t<\/span>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t<\/a>\n\t\t\t<\/div>\n\t\t\t\t\t\t\t\t
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    \n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\tFriedreich's ataxia (FA)\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\t\t\t\n\t\t\t\t\t\t\t\tRare, inherited, progressive disease resulting from mitochondrial dysfunction\t\t\t\t\t\t\t<\/span>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t<\/a>\n\t\t\t<\/div>\n\t\t\t\t\t\t\t\t
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    \n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\tHereditary transthyretin amyloidosis (hATTR)\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\t\t\t\n\t\t\t\t\t\t\t\tRare and fatal disease where deposits of amyloids cause progressive damage to multiple organ systems\t\t\t\t\t\t\t<\/span>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t<\/a>\n\t\t\t<\/div>\n\t\t\t\t\t\t\t\t
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    \n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\tHuntington's disease (HD)\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\t\t\t\n\t\t\t\t\t\t\t\tProgressive brain disorder that causes uncontrolled movements and cognitive loss\t\t\t\t\t\t\t<\/span>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t<\/a>\n\t\t\t<\/div>\n\t\t\t\t\t\t\t\t
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    \n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\tSpinal muscular atrophy (SMA)\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\t\t\t\n\t\t\t\t\t\t\t\tHereditary disease that progressively destroys motor neurons\t\t\t\t\t\t\t<\/span>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t<\/a>\n\t\t\t<\/div>\n\t\t\t<\/div>\n\n<\/div>\n\n\n\n\n
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    \n\t\t\t\tClinical Trials at PTC<\/span>\n\t\t\t<\/h3>\n\t\t\n\t\t
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    Interested in\u00a0<\/span><\/span>enrolling in a\u00a0<\/span><\/span>clinical trial?<\/span><\/span>\u200b<\/span><\/p>\n\t\t<\/div>\n\n\t\t\n\t\t\t

    \n\n\t\t\t\t\tLearn more<\/a>\n\t\t\n\t\t\n\t\t\n\t<\/div>\n\n\t<\/div>\n<\/div>\n\n\n\n\n\n\n\n\n\n\n\n","protected":false},"excerpt":{"rendered":"

    We are deeply committed to patients living with rare diseases who have little-to-no treatment options, and this passion fuels and inspires us. Our commercial and development efforts are focused in two therapeutic areas – neurology and metabolic conditions – in which we have deep experience and a record of success.<\/p>\n","protected":false},"author":1,"featured_media":0,"parent":0,"menu_order":0,"comment_status":"closed","ping_status":"closed","template":"","meta":{"_acf_changed":false,"footnotes":""},"class_list":["post-9951","page","type-page","status-publish","hentry"],"acf":[],"yoast_head":"\nTherapeutic Areas - 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\/\" \/>\n<meta property=\"og:locale\" content=\"it_IT\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Therapeutic Areas\" \/>\n<meta property=\"og:description\" content=\"We are deeply committed to patients living with rare diseases who have little-to-no treatment options, and this passion fuels and inspires us. Our commercial and development efforts are focused in two therapeutic areas – neurology and metabolic conditions – in which we have deep experience and a record of success.\" \/>\n<meta property=\"og:url\" content=\"https:\/\/panel.ptcbio.com\/it-p2025tc\/therapeutic-areas\/\" \/>\n<meta property=\"og:site_name\" content=\"PTC Therapeutics (Italy)\" \/>\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=\"1 minuto\" \/>\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\\\/\",\"url\":\"https:\\\/\\\/panel.ptcbio.com\\\/it-p2025tc\\\/therapeutic-areas\\\/\",\"name\":\"Therapeutic Areas - 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