{"id":307,"date":"2024-11-19T00:00:00","date_gmt":"2024-11-19T00:00:00","guid":{"rendered":"https:\/\/farmacialino.pt\/online\/a-fda-concedeu-a-designacao-de-medicamento-orfao-odd-ao-mdl-101-da-modalis-para-tratar-lama2-cmd\/"},"modified":"2024-12-11T12:48:22","modified_gmt":"2024-12-11T12:48:22","slug":"a-fda-concedeu-a-designacao-de-medicamento-orfao-odd-ao-mdl-101-da-modalis-para-tratar-lama2-cmd","status":"publish","type":"post","link":"https:\/\/farmacialino.pt\/online\/a-fda-concedeu-a-designacao-de-medicamento-orfao-odd-ao-mdl-101-da-modalis-para-tratar-lama2-cmd\/","title":{"rendered":"A FDA concedeu a designa\u00e7\u00e3o de medicamento \u00f3rf\u00e3o (ODD) ao MDL-101 da Modalis para tratar LAMA2-CMD."},"content":{"rendered":"<p>A distrofia <a href=\"https:\/\/farmacialino.pt\/online\/product\/baclofen\/\" data-internallinksmanager029f6b8e52c=\"42\" title=\"Baclofen\">muscular<\/a> cong\u00eanita tipo 1A (LAMA2-CMD) \u00e9 causada pela aus\u00eancia da prote\u00edna LAMA2, levando a uma forma grave e precoce de distrofia muscular. A <a href=\"https:\/\/www.fda.gov\/\" data-internallinksmanager029f6b8e52c=\"8\" title=\"fda\">FDA<\/a> dos EUA concedeu a designa\u00e7\u00e3o de medicamento \u00f3rf\u00e3o para o tratamento experimental MDL-101 da Modalis Therapeutics, que visa tratar LAMA2-CMD. O MDL-101 \u00e9 uma terapia de edi\u00e7\u00e3o epigen\u00e9tica que ativa o gene LAMA1 para compensar a falta de fun\u00e7\u00e3o do LAMA2 nos m\u00fasculos dos pacientes. Esta abordagem pode oferecer um tratamento de longa dura\u00e7\u00e3o para pessoas com LAMA2-CMD.<\/p>\n<p>A designa\u00e7\u00e3o de medicamento \u00f3rf\u00e3o ajuda a desenvolver tratamentos para doen\u00e7as que afetam menos de 200.000 pessoas nos EUA. Com esta designa\u00e7\u00e3o, a Modalis recebe benef\u00edcios como isen\u00e7\u00e3o de taxas de aplica\u00e7\u00e3o, redu\u00e7\u00f5es fiscais e prioridade no desenvolvimento do tratamento no pa\u00eds. Se aprovado, o MDL-101 ter\u00e1 sete anos de exclusividade de vendas nos EUA.<\/p>\n<p>Modalis usa tecnologia de edi\u00e7\u00e3o gen\u00e9tica de precis\u00e3o para desenvolver terapias sem danificar o DNA. Essa tecnologia permite ajustar a express\u00e3o gen\u00e9tica sem causar quebras no DNA.<\/p>\n","protected":false},"excerpt":{"rendered":"<p>A distrofia muscular cong\u00eanita tipo 1A (LAMA2-CMD) \u00e9 causada pela aus\u00eancia da prote\u00edna LAMA2, levando a uma forma grave e [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":0,"comment_status":"closed","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"site-sidebar-layout":"default","site-content-layout":"","ast-site-content-layout":"default","site-content-style":"default","site-sidebar-style":"default","ast-global-header-display":"","ast-banner-title-visibility":"","ast-main-header-display":"","ast-hfb-above-header-display":"","ast-hfb-below-header-display":"","ast-hfb-mobile-header-display":"","site-post-title":"","ast-breadcrumbs-content":"","ast-featured-img":"","footer-sml-layout":"","ast-disable-related-posts":"","theme-transparent-header-meta":"","adv-header-id-meta":"","stick-header-meta":"","header-above-stick-meta":"","header-main-stick-meta":"","header-below-stick-meta":"","astra-migrate-meta-layouts":"default","ast-page-background-enabled":"default","ast-page-background-meta":{"desktop":{"background-color":"var(--ast-global-color-4)","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""},"tablet":{"background-color":"","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""},"mobile":{"background-color":"","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""}},"ast-content-background-meta":{"desktop":{"background-color":"var(--ast-global-color-5)","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""},"tablet":{"background-color":"var(--ast-global-color-5)","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""},"mobile":{"background-color":"var(--ast-global-color-5)","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""}},"footnotes":""},"categories":[289],"tags":[],"class_list":["post-307","post","type-post","status-publish","format-standard","hentry","category-noticias"],"_links":{"self":[{"href":"https:\/\/farmacialino.pt\/online\/wp-json\/wp\/v2\/posts\/307","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/farmacialino.pt\/online\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/farmacialino.pt\/online\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/farmacialino.pt\/online\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/farmacialino.pt\/online\/wp-json\/wp\/v2\/comments?post=307"}],"version-history":[{"count":1,"href":"https:\/\/farmacialino.pt\/online\/wp-json\/wp\/v2\/posts\/307\/revisions"}],"predecessor-version":[{"id":340,"href":"https:\/\/farmacialino.pt\/online\/wp-json\/wp\/v2\/posts\/307\/revisions\/340"}],"wp:attachment":[{"href":"https:\/\/farmacialino.pt\/online\/wp-json\/wp\/v2\/media?parent=307"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/farmacialino.pt\/online\/wp-json\/wp\/v2\/categories?post=307"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/farmacialino.pt\/online\/wp-json\/wp\/v2\/tags?post=307"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}