{"id":3227,"date":"2020-04-15T10:44:29","date_gmt":"2020-04-15T15:44:29","guid":{"rendered":"https:\/\/restruct-lgmd-2021.pantheonsite.io\/?p=3227"},"modified":"2024-02-09T14:24:39","modified_gmt":"2024-02-09T14:24:39","slug":"lgmd-researcher-katherine-mathews","status":"publish","type":"post","link":"https:\/\/www.lgmd-info.org\/pt\/researcher-interview\/2020\/04\/15\/lgmd-researcher-katherine-mathews\/","title":{"rendered":"INVESTIGADORA LGMD: Katherine Mathews"},"content":{"rendered":"

Entrevista de destaque da LGMD sobre pesquisa<\/h2>\n
\"\"<\/figure>\n

INVESTIGADOR DA LGMD:\u00a0 <\/strong>\u00a0Katherine D. Mathews<\/p>\n

Filia\u00e7\u00e3o:\u00a0 <\/strong>Universidade de Iowa, Departamentos de Pediatria e Neurologia<\/p>\n

Fun\u00e7\u00e3o ou cargo:<\/strong> \u00a0\u00a0<\/p>\n

Professor, Departamentos de Pediatria e Neurologia<\/p>\n

Vice-presidente de Investiga\u00e7\u00e3o Cl\u00ednica, Departamento de Pediatria<\/p>\n

Que educa\u00e7\u00e3o e forma\u00e7\u00e3o teve para chegar \u00e0 sua posi\u00e7\u00e3o atual?<\/strong><\/p>\n

Fiz o internato em pediatria, neurologia e neurologia infantil e depois fiz uma bolsa de estudos em gen\u00e9tica. A minha investiga\u00e7\u00e3o cient\u00edfica b\u00e1sica centrou-se na base gen\u00e9tica da distrofia muscular. Mais tarde, na minha carreira, houve uma transi\u00e7\u00e3o l\u00f3gica para um enfoque cl\u00ednico na distrofia muscular.<\/p>\n

O que o levou a seguir uma carreira de investiga\u00e7\u00e3o e a estudar a distrofia muscular em particular?<\/strong><\/p>\n

O interesse pela investiga\u00e7\u00e3o surgiu do desejo de contribuir para melhorar os cuidados a um n\u00edvel mais alargado do que apenas para os doentes que vejo na cl\u00ednica. Quando comecei a minha carreira, n\u00e3o conhec\u00edamos a base gen\u00e9tica ou molecular da maioria das distrofias musculares, pelo que n\u00e3o t\u00ednhamos uma base l\u00f3gica para desenvolver tratamentos. Inicialmente, o acaso e a casualidade levaram-me a concentrar-me na investiga\u00e7\u00e3o das distrofias musculares, mas quanto mais compreendia as doen\u00e7as e conhecia os doentes, mais empenhado me tornava neste grupo de doen\u00e7as.\u00a0<\/p>\n

Que temas est\u00e3o a estudar?<\/strong><\/p>\n

\u00a0Tenho um foco particular nos aspectos cl\u00ednicos das distroglicanopatias, que englobam tanto a LGMD como a distrofia muscular cong\u00e9nita. Tamb\u00e9m fa\u00e7o parte de um esfor\u00e7o de colabora\u00e7\u00e3o apoiado pelo CDC para compreender a epidemiologia de todas as distrofias musculares. A nossa equipa faz parte do cons\u00f3rcio GRASP da LGMD e est\u00e1 envolvida noutros estudos colaborativos de hist\u00f3ria natural. Finalmente, estou envolvido numa s\u00e9rie de ensaios cl\u00ednicos patrocinados pela ind\u00fastria que investigam potenciais tratamentos para doen\u00e7as neuromusculares.<\/p>\n

Como \u00e9 que o seu trabalho vai ajudar os doentes? \u00c9 de natureza mais cient\u00edfica ou poder\u00e1 tornar-se um tratamento para a LGMD?<\/strong>s ou MD<\/strong>s em geral?<\/strong><\/p>\n

O meu trabalho sobre as distroglicanopatias dever\u00e1 ajudar a fornecer a base para a conce\u00e7\u00e3o de futuros ensaios cl\u00ednicos e para a compreens\u00e3o da hist\u00f3ria natural destas doen\u00e7as, de modo a prestar melhores cuidados aos doentes actuais. Do mesmo modo, o trabalho de epidemiologia \u00e9 importante para compreender a hist\u00f3ria natural dos diferentes tipos de distrofia muscular e para as empresas que est\u00e3o a considerar a conce\u00e7\u00e3o de ensaios de tratamento - precisam de saber quantos doentes existem para determinar como conceber um ensaio que seja vi\u00e1vel e realista. Espero que os esfor\u00e7os das nossas equipas contribuam para melhorar a gest\u00e3o dos doentes com distrofias musculares.\u00a0<\/p>\n

O que gostaria que os doentes e outras pessoas interessadas na LGMD soubessem sobre a investiga\u00e7\u00e3o (os seus pr\u00f3prios projectos e a \u00e1rea em geral)?<\/strong><\/p>\n

Os doentes precisam de compreender que mesmo responder a perguntas simples sobre os seres humanos \u00e9 muitas vezes um trabalho moroso. Isto \u00e9 particularmente verdade no caso das doen\u00e7as raras. Os seres humanos s\u00e3o complicados e h\u00e1 muitas coisas que contribuem para a varia\u00e7\u00e3o dos resultados, tanto factores gen\u00e9ticos como factores n\u00e3o gen\u00e9ticos. Em parte devido a esta variabilidade, os estudos de tratamento t\u00eam de ser concebidos com muito cuidado. \u00c9 prov\u00e1vel que alguns ensaios de tratamento n\u00e3o demonstrem benef\u00edcios. O \u00fanico ensaio de tratamento que \u00e9 um verdadeiro fracasso \u00e9 aquele que n\u00e3o consegue responder \u00e0 quest\u00e3o do benef\u00edcio e da seguran\u00e7a. Um ensaio de tratamento que demonstre que uma determinada terap\u00eautica n\u00e3o melhora os resultados \u00e9 um ensaio bem sucedido - permite-nos avan\u00e7ar para uma abordagem diferente ao tratamento.<\/p>\n

O que o inspira a continuar a trabalhar neste dom\u00ednio?<\/strong><\/p>\n

Sinto-me muito feliz por interagir com os doentes, as fam\u00edlias e os colegas que conhe\u00e7o atrav\u00e9s deste trabalho. Eles s\u00e3o a principal for\u00e7a motriz. Em segundo lugar, a acelera\u00e7\u00e3o da promessa de tratamentos significativos \u00e9 entusiasmante!<\/p>\n

Como \u00e9 que os doentes o podem encorajar e ajudar no seu trabalho?<\/strong><\/p>\n

\u00a0Participar na investiga\u00e7\u00e3o na medida das suas possibilidades. Ser um doente ou um sujeito de investiga\u00e7\u00e3o bem informado. Apoie o financiamento cont\u00ednuo neste dom\u00ednio (NIH, MDA, funda\u00e7\u00f5es de doen\u00e7as espec\u00edficas, investigadores individuais). Fale com os seus senadores e deputados federais sobre a import\u00e2ncia do apoio cont\u00ednuo dos NIH \u00e0 investiga\u00e7\u00e3o em geral, \u00e0s doen\u00e7as neuromusculares e \u00e0 sua doen\u00e7a em particular. Conte a sua hist\u00f3ria.\u00a0<\/p>\n<\/div><\/div><\/div><\/div><\/div>","protected":false},"excerpt":{"rendered":"","protected":false},"author":1,"featured_media":4376,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"footnotes":""},"categories":[91,92],"tags":[],"class_list":["post-3227","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-researcher-interview","category-researcher"],"acf":[],"yoast_head":"\nLGMD RESEARCHER: Katherine Mathews - LGMD Awareness 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.lgmd-info.org\/pt\/researcher-interview\/2020\/04\/15\/lgmd-researcher-katherine-mathews\/\" \/>\n<meta property=\"og:locale\" content=\"pt_PT\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" 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