{"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\/fr\/researcher-interview\/2020\/04\/15\/lgmd-researcher-katherine-mathews\/","title":{"rendered":"Chercheur du LGMD : Katherine Mathews"},"content":{"rendered":"

LGMD Spotlight Interview sur la recherche<\/h2>\n
\"\"<\/figure>\n

CHERCHEUR DE LGMD :\u00a0 <\/strong>\u00a0Katherine D. Mathews<\/p>\n

Affiliation :\u00a0 <\/strong>Universit\u00e9 de l'Iowa, d\u00e9partements de p\u00e9diatrie et de neurologie<\/p>\n

R\u00f4le ou fonction :<\/strong> \u00a0\u00a0<\/p>\n

Professeur, D\u00e9partements de p\u00e9diatrie et de neurologie<\/p>\n

Vice-pr\u00e9sidence de l'investigation clinique, d\u00e9partement de p\u00e9diatrie<\/p>\n

Quels sont les \u00e9tudes et la formation qui vous ont permis d'acc\u00e9der \u00e0 votre poste actuel ?<\/strong><\/p>\n

J'ai effectu\u00e9 un internat en p\u00e9diatrie, en neurologie et en neurologie infantile, puis j'ai obtenu une bourse de recherche en g\u00e9n\u00e9tique. Mes recherches en sciences fondamentales ont port\u00e9 sur les bases g\u00e9n\u00e9tiques de la dystrophie musculaire. Plus tard dans ma carri\u00e8re, j'ai logiquement opt\u00e9 pour une approche clinique de la dystrophie musculaire.<\/p>\n

Qu'est-ce qui vous a amen\u00e9 \u00e0 suivre une carri\u00e8re dans la recherche et \u00e0 \u00e9tudier la dystrophie musculaire en particulier ?<\/strong><\/p>\n

L'int\u00e9r\u00eat pour la recherche est n\u00e9 du d\u00e9sir de contribuer \u00e0 l'am\u00e9lioration des soins \u00e0 un niveau plus large que les seuls patients que je vois en clinique. Lorsque j'ai commenc\u00e9 ma carri\u00e8re, nous ne connaissions pas les bases g\u00e9n\u00e9tiques ou mol\u00e9culaires de la plupart des dystrophies musculaires et ne disposions donc d'aucune base logique pour d\u00e9velopper des traitements. Au d\u00e9part, c'est le hasard et la chance qui m'ont amen\u00e9 \u00e0 me concentrer sur la recherche sur les dystrophies musculaires, mais plus je comprenais les maladies et apprenais \u00e0 conna\u00eetre les patients, plus je m'engageais en faveur de ce groupe de troubles.\u00a0<\/p>\n

Quels sont les sujets que vous \u00e9tudiez ?<\/strong><\/p>\n

\u00a0Je m'int\u00e9resse particuli\u00e8rement aux aspects cliniques des dystroglycanopathies, qui englobent \u00e0 la fois la LGMD et la dystrophie musculaire cong\u00e9nitale. Je participe \u00e9galement \u00e0 un effort de collaboration soutenu par le CDC pour comprendre l'\u00e9pid\u00e9miologie de toutes les dystrophies musculaires. Notre \u00e9quipe fait partie du consortium LGMD GRASP et participe \u00e0 d'autres \u00e9tudes collaboratives sur l'histoire naturelle. Enfin, je participe \u00e0 un certain nombre d'essais cliniques parrain\u00e9s par l'industrie et portant sur des traitements potentiels des maladies neuromusculaires.<\/p>\n

Comment vos travaux aideront-ils les patients ? S'agit-il d'un travail de nature plus scientifique ou pourrait-il devenir un traitement pour la LGMD ?<\/strong>ou MD<\/strong>en g\u00e9n\u00e9ral ?<\/strong><\/p>\n

Mes travaux sur les dystroglycanopathies devraient contribuer \u00e0 jeter les bases de la conception des futurs essais cliniques et de la compr\u00e9hension de l'histoire naturelle de ces maladies afin de mieux soigner les patients aujourd'hui. De m\u00eame, le travail \u00e9pid\u00e9miologique est important pour comprendre l'histoire naturelle des diff\u00e9rents types de dystrophie musculaire et pour les entreprises qui envisagent de concevoir des essais de traitement - elles ont besoin de savoir combien de patients il y a pour d\u00e9terminer comment concevoir un essai qui soit faisable et r\u00e9aliste. J'esp\u00e8re que les efforts de nos \u00e9quipes contribueront \u00e0 am\u00e9liorer la prise en charge des patients atteints de dystrophies musculaires.\u00a0<\/p>\n

Qu'aimeriez-vous que les patients et les autres personnes int\u00e9ress\u00e9es par la LGMD sachent \u00e0 propos de la recherche (vos propres projets et le domaine en g\u00e9n\u00e9ral) ?<\/strong><\/p>\n

Les patients doivent comprendre que m\u00eame r\u00e9pondre \u00e0 des questions simples sur des \u00eatres humains est souvent un travail de longue haleine. C'est particuli\u00e8rement vrai pour les maladies rares. Les \u00eatres humains sont complexes et de nombreux facteurs, g\u00e9n\u00e9tiques ou non, contribuent \u00e0 la variation des r\u00e9sultats. C'est en partie \u00e0 cause de cette variabilit\u00e9 que les \u00e9tudes de traitement doivent \u00eatre con\u00e7ues avec le plus grand soin. Il est probable que certains essais th\u00e9rapeutiques n'apporteront aucun b\u00e9n\u00e9fice. le seul essai th\u00e9rapeutique qui soit un v\u00e9ritable \u00e9chec est celui qui ne r\u00e9pond pas \u00e0 la question du b\u00e9n\u00e9fice et de la s\u00e9curit\u00e9. un essai th\u00e9rapeutique qui d\u00e9montre qu'une certaine th\u00e9rapie n'am\u00e9liore pas les r\u00e9sultats est un essai r\u00e9ussi - il nous permet de passer \u00e0 une approche diff\u00e9rente du traitement.<\/p>\n

Qu'est-ce qui vous incite \u00e0 continuer \u00e0 travailler dans ce domaine ?<\/strong><\/p>\n

J'ai beaucoup de chance de pouvoir interagir avec les patients, les familles et les coll\u00e8gues que je rencontre dans le cadre de mon travail. Ils constituent une force motrice essentielle. Deuxi\u00e8mement, l'acc\u00e9l\u00e9ration de la promesse de traitements significatifs est passionnante !<\/p>\n

Comment les patients peuvent-ils vous encourager et vous aider dans votre travail ?<\/strong><\/p>\n

\u00a0Participez \u00e0 la recherche dans la mesure de vos possibilit\u00e9s. Soyez un patient ou un sujet de recherche bien inform\u00e9. Soutenez le financement continu dans ce domaine (NIH, MDA, fondations sp\u00e9cifiques \u00e0 la maladie, chercheurs individuels). Parlez \u00e0 vos s\u00e9nateurs et repr\u00e9sentants f\u00e9d\u00e9raux de l'importance d'un soutien continu des NIH \u00e0 la recherche en g\u00e9n\u00e9ral, aux maladies neuromusculaires et \u00e0 votre maladie en particulier. Racontez votre histoire.\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\/fr\/researcher-interview\/2020\/04\/15\/lgmd-researcher-katherine-mathews\/\" \/>\n<meta property=\"og:locale\" content=\"fr_FR\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" 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