{"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\/es\/researcher-interview\/2020\/04\/15\/lgmd-researcher-katherine-mathews\/","title":{"rendered":"INVESTIGADORA LGMD: Katherine Mathews"},"content":{"rendered":"<div class=\"fusion-fullwidth fullwidth-box fusion-builder-row-1 fusion-flex-container nonhundred-percent-fullwidth non-hundred-percent-height-scrolling\" style=\"--awb-border-radius-top-left:0px;--awb-border-radius-top-right:0px;--awb-border-radius-bottom-right:0px;--awb-border-radius-bottom-left:0px;--awb-flex-wrap:wrap;\" ><div class=\"fusion-builder-row fusion-row fusion-flex-align-items-flex-start fusion-flex-content-wrap\" style=\"max-width:calc( 1400px + 0px );margin-left: calc(-0px \/ 2 );margin-right: calc(-0px \/ 2 );\"><div class=\"fusion-layout-column fusion_builder_column fusion-builder-column-0 fusion_builder_column_1_1 1_1 fusion-flex-column\" style=\"--awb-bg-size:cover;--awb-width-large:100%;--awb-margin-top-large:0px;--awb-spacing-right-large:0px;--awb-margin-bottom-large:0px;--awb-spacing-left-large:0px;--awb-width-medium:100%;--awb-spacing-right-medium:0px;--awb-spacing-left-medium:0px;--awb-width-small:100%;--awb-spacing-right-small:0px;--awb-spacing-left-small:0px;\"><div class=\"fusion-column-wrapper fusion-flex-justify-content-flex-start fusion-content-layout-column\"><div class=\"fusion-text fusion-text-1\"><h2 class=\"wp-block-heading\">Entrevista sobre investigaci\u00f3n en el punto de mira de la LGMD<\/h2>\n<figure class=\"wp-block-image size-large\"><img decoding=\"async\" class=\"lazyload wp-image-3228\" src=\"data:image\/svg+xml,%3Csvg%20xmlns%3D%27http%3A%2F%2Fwww.w3.org%2F2000%2Fsvg%27%20width%3D%27960%27%20height%3D%27720%27%20viewBox%3D%270%200%20960%20720%27%3E%3Crect%20width%3D%27960%27%20height%3D%27720%27%20fill-opacity%3D%220%22%2F%3E%3C%2Fsvg%3E\" data-orig-src=\"https:\/\/www.lgmd-info.org\/wp-content\/uploads\/2020\/04\/Matthews-Katherine.png\" alt=\"\" \/><\/figure>\n<p><strong>INVESTIGADOR DE LGMD:\u00a0 <\/strong>\u00a0Katherine D. Mathews<\/p>\n<p><strong>Afiliaci\u00f3n:\u00a0 <\/strong>Universidad de Iowa, Departamentos de Pediatr\u00eda y Neurolog\u00eda<\/p>\n<p><strong>Funci\u00f3n o cargo:<\/strong> \u00a0\u00a0<\/p>\n<p>Profesor, Departamentos de Pediatr\u00eda y Neurolog\u00eda<\/p>\n<p>Vicepresidente de Investigaci\u00f3n C\u00ednica, Departamento de Pediatr\u00eda<\/p>\n<p><strong>\u00bfQu\u00e9 educaci\u00f3n y formaci\u00f3n ha recibido para llegar a su puesto actual?<\/strong><\/p>\n<p>Realic\u00e9 la residencia en pediatr\u00eda, neurolog\u00eda y neurolog\u00eda infantil, y despu\u00e9s una beca de investigaci\u00f3n en gen\u00e9tica. Mi investigaci\u00f3n en ciencias b\u00e1sicas se centr\u00f3 en las bases gen\u00e9ticas de la distrofia muscular. M\u00e1s adelante en mi carrera, se produjo una transici\u00f3n l\u00f3gica hacia un enfoque cl\u00ednico de la distrofia muscular.<\/p>\n<p><strong>\u00bfQu\u00e9 le llev\u00f3 a dedicarse a la investigaci\u00f3n y, en particular, al estudio de la distrofia muscular?<\/strong><\/p>\n<p>El inter\u00e9s por la investigaci\u00f3n surgi\u00f3 del deseo de contribuir a mejorar la atenci\u00f3n a un nivel m\u00e1s amplio que el de los pacientes que veo en la cl\u00ednica.  Cuando empec\u00e9 mi carrera, no conoc\u00edamos las bases gen\u00e9ticas o moleculares de la mayor\u00eda de las distrofias musculares, por lo que no ten\u00edamos una base l\u00f3gica para desarrollar tratamientos. Al principio, la casualidad y el azar me llevaron a centrarme en la investigaci\u00f3n de las distrofias musculares, pero cuanto m\u00e1s comprend\u00eda las enfermedades y m\u00e1s conoc\u00eda a los pacientes, m\u00e1s me compromet\u00eda con este grupo de trastornos.\u00a0<\/p>\n<p><strong>\u00bfQu\u00e9 temas est\u00e1 estudiando?<\/strong><\/p>\n<p>\u00a0Me centro especialmente en los aspectos cl\u00ednicos de las distroglicanopat\u00edas, que engloban tanto la LGMD como la distrofia muscular cong\u00e9nita.  Tambi\u00e9n formo parte de un esfuerzo de colaboraci\u00f3n apoyado por los CDC para comprender la epidemiolog\u00eda de todas las distrofias musculares.  Nuestro equipo forma parte del consorcio LGMD GRASP y participa en otros estudios colaborativos de historia natural.  Por \u00faltimo, participo en varios ensayos cl\u00ednicos patrocinados por la industria que investigan posibles tratamientos para enfermedades neuromusculares.<\/p>\n<p><strong>\u00bfC\u00f3mo ayudar\u00e1 su trabajo a los pacientes? \u00bfTiene un car\u00e1cter m\u00e1s cient\u00edfico o podr\u00eda convertirse en un tratamiento para la LGMD?<\/strong><strong>s o MD<\/strong><strong>s en general?<\/strong><\/p>\n<p>Mi trabajo sobre las distroglicanopat\u00edas deber\u00eda ayudar a sentar las bases para dise\u00f1ar futuros ensayos cl\u00ednicos y comprender la historia natural de estas enfermedades para atender mejor a los pacientes en la actualidad.  Del mismo modo, el trabajo epidemiol\u00f3gico es importante para comprender la historia natural de los distintos tipos de distrofia muscular y para las empresas que se plantean dise\u00f1ar ensayos de tratamiento: necesitan saber cu\u00e1ntos pacientes hay para determinar c\u00f3mo dise\u00f1ar un ensayo que sea factible y realista.  Espero que los esfuerzos de nuestros equipos contribuyan a mejorar el tratamiento de los pacientes con distrofias musculares.\u00a0<\/p>\n<p><strong>\u00bfQu\u00e9 le gustar\u00eda que los pacientes y otras personas interesadas en la LGMD supieran sobre la investigaci\u00f3n (sus propios proyectos y sobre el campo en general)?<\/strong><\/p>\n<p>Los pacientes deben comprender que incluso responder a preguntas sencillas sobre seres humanos suele ser un trabajo lento. Esto es especialmente cierto en el caso de las enfermedades raras.  Los seres humanos son complicados y hay muchas cosas que contribuyen a la variaci\u00f3n de los resultados, tanto factores gen\u00e9ticos como no gen\u00e9ticos. En parte debido a esta variabilidad, los estudios de tratamiento deben dise\u00f1arse con mucho cuidado.  Es probable que algunos ensayos de tratamiento no muestren beneficios. el \u00fanico ensayo de tratamiento que es un verdadero fracaso es el que no logra responder a la pregunta sobre el beneficio y la seguridad. un ensayo de tratamiento que demuestra que una determinada terapia no mejora los resultados es un ensayo exitoso: nos permite pasar a un enfoque diferente del tratamiento.<\/p>\n<p><strong>\u00bfQu\u00e9 le inspira a seguir trabajando en este campo?<\/strong><\/p>\n<p>Me siento muy afortunada de interactuar con los pacientes, las familias y los colegas que conozco gracias a este trabajo.  Ellos son la principal fuerza motriz.  En segundo lugar, \u00a1la promesa acelerada de tratamientos significativos es apasionante!<\/p>\n<p><strong>\u00bfC\u00f3mo pueden los pacientes animarle y ayudarle en su trabajo?<\/strong><\/p>\n<p>\u00a0Participe en la investigaci\u00f3n en la medida de sus posibilidades.  Sea un paciente o sujeto de investigaci\u00f3n bien informado.  Apoye la financiaci\u00f3n continua en este campo (NIH, MDA, fundaciones espec\u00edficas de la enfermedad, investigadores individuales).  Hable con sus senadores y representantes federales sobre la importancia del apoyo continuado de los NIH a la investigaci\u00f3n en general, en enfermedades neuromusculares y en su enfermedad en particular.  Cuente su historia.\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":"<!-- This site is optimized with the Yoast SEO plugin v23.9 - https:\/\/yoast.com\/wordpress\/plugins\/seo\/ -->\n<title>LGMD 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\/es\/researcher-interview\/2020\/04\/15\/lgmd-researcher-katherine-mathews\/\" \/>\n<meta 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