{"id":4004,"date":"2018-04-25T00:03:37","date_gmt":"2018-04-25T00:03:37","guid":{"rendered":"https:\/\/www.progeriaresearch.org\/?page_id=4004"},"modified":"2023-09-21T12:15:37","modified_gmt":"2023-09-21T16:15:37","slug":"first-ever-demonstrate-survival-benefit-in-progeria","status":"publish","type":"page","link":"https:\/\/www.progeriaresearch.org\/pt\/first-ever-demonstrate-survival-benefit-in-progeria\/","title":{"rendered":"Primeira terapia a demonstrar benef\u00edcio de sobreviv\u00eancia na Progeria"},"content":{"rendered":"<p>24 de abril de 2018: Um novo estudo publicado no The Journal of the American Medical Association (JAMA) relata que o lonafarnib, um inibidor da farnesiltransferase (FTI), ajudou a estender a sobreviv\u00eancia em crian\u00e7as com Progeria. Autores do Boston Children&#039;s Hospital e da Brown University rastrearam mais de 250 crian\u00e7as de seis continentes para demonstrar uma liga\u00e7\u00e3o entre o tratamento com lonafarnib e a sobreviv\u00eancia estendida.<\/p>\n<p><a href=\"https:\/\/jamanetwork.com\/journals\/jama\/article-abstract\/2679278?redirect=true&amp;appId=scweb\" target=\"_blank\" rel=\"noopener noreferrer\"><b>Clique aqui para ler o resumo do estudo<\/b><\/a><\/p>\n<p><strong>PARA A M\u00cdDIA:\u00a0 <\/strong><\/p>\n<ul>\n<li><strong><a href=\"https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2018\/04\/PRF-JAMA-Announcement_Press-Release-1.pdf\">Comunicado de imprensa<\/a><\/strong><\/li>\n<li><strong><a href=\"https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2018\/04\/PRF-JAMA-Announcement-Infographic.pdf\">Infogr\u00e1fico<\/a><\/strong><strong>\u00a0<\/strong><\/li>\n<li><a href=\"https:\/\/www.dropbox.com\/sh\/uuq64xv0w0kfrcf\/AAACRWWU4ZKezFTksabaimzba?dl=0\" target=\"_blank\" rel=\"noopener noreferrer\"><strong>V\u00eddeo B-roll<\/strong><\/a><\/li>\n<\/ul>\n<p><strong>Os resultados de\u00a0<\/strong><a href=\"https:\/\/www.progeriaresearch.org\/pt\/clinical-trials\/\"><strong>o ensaio cl\u00ednico de medicamentos para crian\u00e7as<\/strong><\/a><strong>\u00a0com Progeria est\u00e3o dispon\u00edveis e \u00e9 oficial!<\/strong>\u00a0 \u00a0O estudo usando lonafarnib, um tipo de inibidor de farnesiltransferase (FTI) originalmente desenvolvido para tratar c\u00e2ncer, apoia a extens\u00e3o da expectativa de vida em crian\u00e7as com Progeria. No ensaio cl\u00ednico, 27 crian\u00e7as com Progeria receberam lonafarnib oral duas vezes ao dia como monoterapia. O bra\u00e7o de controle deste estudo consistiu em crian\u00e7as com Progeria com idade, sexo e continente de resid\u00eancia semelhantes aos pacientes tratados, que n\u00e3o faziam parte do ensaio cl\u00ednico e, portanto, n\u00e3o receberam lonafarnib. Os resultados demonstraram que o tratamento com lonafarnib sozinho, em compara\u00e7\u00e3o com nenhum tratamento, foi associado a uma taxa de mortalidade significativamente menor (3,7% vs. 33,3%) ap\u00f3s uma mediana de 2,2 anos de acompanhamento. Resultados do estudo, que foi\u00a0<strong>financiado e coordenado pela The Progeria Research Foundation<\/strong>, foram publicados em 24 de abril de 2018 em <em>Jornal da Associa\u00e7\u00e3o M\u00e9dica Americana.<\/em><\/p>\n<p><em>Gordon et. al.,Associa\u00e7\u00e3o do tratamento com lonafarnib versus nenhum tratamento com a taxa de mortalidade em pacientes com s\u00edndrome de Hutchinson-Gilford Progeria<\/em>, <em>JAMA, 24 de abril de 2018 Volume 319, N\u00famero 16<\/em><strong>Resultados trazem nova esperan\u00e7a e otimismo<br \/>\n<\/strong>As crian\u00e7as viajaram para o Hospital Infantil de Boston para receber testes m\u00e9dicos abrangentes e medicamentos do estudo. Todos receberam lonafarnib oral, um FTI fornecido pela Merck &amp; Co. \u201cEste estudo publicado no JAMA mostra evid\u00eancias de que podemos come\u00e7ar a frear o r\u00e1pido processo de envelhecimento de crian\u00e7as com Progeria. Esses resultados fornecem novas promessas e otimismo para a comunidade da Progeria\u201d, disse Leslie Gordon, MD, PhD, cofundadora e diretora m\u00e9dica da PRF e principal autora do estudo.<\/p>\n<p>\u201cNa PRF, estamos trabalhando incansavelmente para financiar novos avan\u00e7os cient\u00edficos para crian\u00e7as que vivem com Prog\u00e9ria. Este estudo nos mostra que os ensaios cl\u00ednicos conduzidos hoje s\u00e3o nossa melhor esperan\u00e7a de salvar essas crian\u00e7as no futuro. Com base nos resultados promissores deste estudo com lonafarnib, sentimos um senso de urg\u00eancia mais forte do que nunca. O marco de hoje marca um novo come\u00e7o para essas crian\u00e7as e suas fam\u00edlias. Cada dia e cada momento contam. O objetivo da PRF \u00e9 encontrar a cura para a Prog\u00e9ria e este estudo nos traz mais um passo em dire\u00e7\u00e3o a esse objetivo\u201d, disse Meryl Fink, presidente e diretora executiva da PRF<\/p>\n<p><strong>Cobertura da m\u00eddia<\/strong><\/p>\n<p>Aqui est\u00e1 uma amostra da cobertura da imprensa recebida:<\/p>\n<ul>\n<li><strong>MedPage Hoje<br \/>\n<\/strong><a href=\"https:\/\/www.medpagetoday.com\/blogs\/themethodsman\/72505\" target=\"_blank\" rel=\"noopener noreferrer\"><em>Medicamento pode tornar a progeria control\u00e1vel <\/em><\/a><\/li>\n<li><strong>Globo de Boston<\/strong><br \/>\n<a href=\"https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2018\/07\/The-Boston-Globe_A-pediatric-researcher-whose-son-died-of-progeria-sees-....pdf\" target=\"_blank\" rel=\"noopener noreferrer\"><em>Pesquisador pedi\u00e1trico cujo filho morreu de progeria v\u00ea promessa em novo tratamento<\/em> 24 de abril de 2018<\/a><\/li>\n<li><strong>Not\u00edcias Marrons<\/strong><br \/>\n<a href=\"https:\/\/news.brown.edu\/articles\/2018\/04\/progeria\" target=\"_blank\" rel=\"noopener noreferrer\"><em>Pesquisa sugere terapia medicamentosa promissora para doen\u00e7a do envelhecimento prematuro<\/em> 24 de abril de 2018<\/a><\/li>\n<\/ul>\n<p><em>\u201cEsta \u00e9 uma hist\u00f3ria inspiradora em muitos n\u00edveis. H\u00e1 o potencial de transformar as vidas de um grupo de crian\u00e7as muito especiais. Mas tamb\u00e9m \u00e9 exemplar do m\u00e9todo cient\u00edfico que deu certo, onde a ci\u00eancia b\u00e1sica rigorosa e uma aplica\u00e7\u00e3o inteligente de um medicamento existente se combinaram para produzir resultados que s\u00e3o avan\u00e7os genu\u00ednos.\u201d<\/em><\/p>\n<p><em>\u2013 F. Perry Wilson MD, MSCE, Faculdade de Medicina de Yale, MedPage Today<\/em><\/p>\n<p><strong>Como chegamos a esse dia maravilhoso?<\/strong><br \/>\nSeguindo o <a href=\"https:\/\/www.progeriaresearch.org\/pt\/identification-of-gene-gives-hope-to-children-with-progeria\/\" target=\"_blank\" rel=\"noopener noreferrer\">Descoberta da muta\u00e7\u00e3o gen\u00e9tica em 2003<\/a> que causa a Prog\u00e9ria, identificaram pesquisadores financiados pela PRF\u00a0<a href=\"https:\/\/www.progeriaresearch.org\/pt\/the-fti-drug\/\">FTIs<\/a>\u00a0como um potencial tratamento medicamentoso. A muta\u00e7\u00e3o causadora da Progeria leva \u00e0 produ\u00e7\u00e3o da prote\u00edna\u00a0<em>progerina<\/em>, que danifica a fun\u00e7\u00e3o celular. Parte do efeito t\u00f3xico da progerina no corpo \u00e9 causado por uma mol\u00e9cula chamada \u201cgrupo farnesil\u201d, que se liga \u00e0 prote\u00edna progerina e a ajuda a danificar as c\u00e9lulas do corpo. Os FTIs agem bloqueando a liga\u00e7\u00e3o do grupo farnesil \u00e0 progerina, reduzindo os danos que a progerina causa.<\/p>\n<p><strong>Para mais detalhes do estudo,\u00a0<a href=\"https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2018\/04\/PRF-JAMA-Announcement_Press-Release-1.pdf\">Clique aqui para o comunicado de imprensa<\/a> <\/strong><\/p>\n<p><em>\u201cDemonstrar a efic\u00e1cia dos tratamentos nessa pequena popula\u00e7\u00e3o de crian\u00e7as com essa doen\u00e7a rara e fatal \u00e9 um grande desafio. Portanto, estou particularmente encorajado por essas \u00faltimas descobertas.\u201d<\/em><\/p>\n<p><em>\u2013 Dr. Francis Collins, Diretor dos Institutos Nacionais de Sa\u00fade<\/em><\/p>\n<p><strong>Progeria associada ao processo normal de envelhecimento<\/strong><a href=\"https:\/\/web-beta.archive.org\/web\/20170128050324\/https:\/www.progeriaresearch.org\/whats_new_in_progeria_research.html#2\"><br \/>\n<\/a>Pesquisas mostram que a prote\u00edna causadora da Prog\u00e9ria\u00a0<em>progerina<\/em>\u00a0tamb\u00e9m \u00e9 produzido na popula\u00e7\u00e3o em geral e aumenta com a idade. Os pesquisadores planejam continuar explorando o efeito dos FTIs, o que pode ajudar os cientistas a aprender mais sobre a doen\u00e7a cardiovascular que afeta milh\u00f5es, bem como o processo normal de envelhecimento que afeta a todos n\u00f3s.<\/p>\n<p><strong>Obrigado a TODOS \u2013<\/strong>\u00a0<strong>N\u00e3o ter\u00edamos conseguido sem voc\u00ea!<\/strong><br \/>\nUm dos principais motivos pelos quais alcan\u00e7amos esses resultados inovadores \u00e9 por causa dos grandes apoiadores que forneceram financiamento e outro suporte, ajudando-nos a ficar um passo mais perto de atingir nosso objetivo final: uma cura para a Prog\u00e9ria.<\/p>","protected":false},"excerpt":{"rendered":"<p>April 24, 2018: A new study published in The Journal of the American Medical Association (JAMA) reports that lonafarnib, a farnesyltransferase inhibitor (FTI), helped extend survival in children with Progeria. Authors from Boston Children\u2019s Hospital and Brown University tracked more than 250 children from six continents to demonstrate a link between lonafarnib treatment and extended [&hellip;]<\/p>\n","protected":false},"author":2,"featured_media":0,"parent":0,"menu_order":0,"comment_status":"closed","ping_status":"closed","template":"","meta":{"_et_pb_use_builder":"","_et_pb_old_content":"","_et_gb_content_width":"","footnotes":"","_links_to":"","_links_to_target":""},"class_list":["post-4004","page","type-page","status-publish","hentry"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v27.8 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>First Ever | The Progeria Research Foundation<\/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:\/\/www.progeriaresearch.org\/pt\/first-ever-demonstrate-survival-benefit-in-progeria\/\" \/>\n<meta property=\"og:locale\" content=\"pt_PT\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"First Ever | The Progeria Research Foundation\" \/>\n<meta property=\"og:description\" content=\"April 24, 2018: A new study published in The Journal of the American Medical Association (JAMA) reports that lonafarnib, a farnesyltransferase inhibitor (FTI), helped extend survival in children with Progeria. 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