{"id":3505,"date":"2025-06-13T20:45:29","date_gmt":"2025-06-13T20:45:29","guid":{"rendered":"https:\/\/life4hsp.com\/?p=3505"},"modified":"2025-06-23T11:05:38","modified_gmt":"2025-06-23T09:05:38","slug":"approbation-du-premier-essai-dune-nouvelle-therapie-genique-pour-la-hsp-spg47","status":"publish","type":"post","link":"https:\/\/life4hsp.com\/fr\/approbation-du-premier-essai-dune-nouvelle-therapie-genique-pour-la-hsp-spg47\/","title":{"rendered":"Approbation du premier essai d'une nouvelle th\u00e9rapie g\u00e9nique pour la HSP (SPG47)"},"content":{"rendered":"<p><span data-olk-copy-source=\"MessageBody\">Il y a des nouvelles encourageantes pour les personnes atteintes de parapl\u00e9gie spastique h\u00e9r\u00e9ditaire : l'autorit\u00e9 sanitaire am\u00e9ricaine FDA a donn\u00e9 son accord pour un premier essai d'une nouvelle th\u00e9rapie g\u00e9nique pour une forme rare de parapl\u00e9gie spastique h\u00e9r\u00e9ditaire, \u00e0 savoir le SPG47.<\/span><\/p>\n<h3><b>Qu'est-ce que le SPG47 ?<\/b><\/h3>\n<p>Le SPG47 est une forme rare et h\u00e9r\u00e9ditaire de HSP dans laquelle les enfants pr\u00e9sentent tr\u00e8s t\u00f4t des sympt\u00f4mes tels qu'une raideur musculaire, des difficult\u00e9s \u00e0 marcher, des retards de d\u00e9veloppement et des troubles de la parole. La cause est une mutation dans le g\u00e8ne SPG47, emp\u00eachant un important prot\u00e9ine dans les cellules c\u00e9r\u00e9brales de fonctionner correctement. Il n'existe actuellement aucun traitement.<\/p>\n<h3><b>Que va-t-il se passer maintenant ?<\/b><\/h3>\n<p>Une entreprise de biotechnologie, <a href=\"https:\/\/blackfin.bio\/\">BlackfinBio<\/a>est d\u00e9sormais autoris\u00e9e \u00e0 lancer une premi\u00e8re \u00e9tude sur des enfants atteints de SPG47. Cela se fera dans un h\u00f4pital sp\u00e9cialis\u00e9 \u00e0 Boston (\u00c9tats-Unis). L\u2019objectif est de v\u00e9rifier si le traitement est s\u00fbr.<\/p>\n<p>Le traitement consiste en une injection unique dans le liquide c\u00e9phalorachidien avec un fragment d\u2019ADN \u00ab sain \u00bb. Ce g\u00e8ne sain devrait permettre aux cellules de l\u2019enfant de produire \u00e0 nouveau la bonne prot\u00e9ine.<\/p>\n<h3><b>Pourquoi est-ce important ?<\/b><\/h3>\n<ul>\n<li>Il s\u2019agit du tout premier traitement contre le SPG47 test\u00e9 chez l\u2019humain.<\/li>\n<li>La th\u00e9rapie vise la cause m\u00eame de la maladie, pas seulement les sympt\u00f4mes.<\/li>\n<li>L\u2019approbation de la FDA repr\u00e9sente une avanc\u00e9e majeure vers une future prise en charge.<\/li>\n<\/ul>\n<h3><b>Que cela signifie-t-il pour les familles ?<\/b><\/h3>\n<p>M\u00eame si la recherche est encore au stade pr\u00e9coce, elle redonne espoir aux enfants atteints de SPG47 et \u00e0 leurs familles. Des progr\u00e8s sont en cours, et des scientifiques et m\u00e9decins s\u2019engagent s\u00e9rieusement \u00e0 trouver des solutions aux formes rares de HSP. <a href=\"https:\/\/www.sheffield.ac.uk\/news\/fda-approves-trial-advance-pioneering-treatment-hereditary-spastic-paraplegia-hsp\">cliquez ici<\/a> sur la recherche<\/p>","protected":false},"excerpt":{"rendered":"<p>Er is hoopvol nieuws voor mensen met HSP: de Amerikaanse gezondheidsautoriteit FDA heeft toestemming gegeven voor een eerste proef met een nieuwe gentherapie voor een zeldzame vorm van erfelijke spastische paraplegie, namelijk SPG47. Wat is SPG47? SPG47 is een zeldzame, erfelijke vorm van HSP waarbij kinderen al op jonge leeftijd klachten krijgen zoals spierstijfheid, moeite [&hellip;]<\/p>\n","protected":false},"author":5,"featured_media":3506,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"inline_featured_image":false,"footnotes":""},"categories":[13],"tags":[],"class_list":["post-3505","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-onderzoek"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v27.9 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>Gentherapie voor SPG47<\/title>\n<meta name=\"description\" content=\"de Amerikaanse gezondheidsautoriteit FDA heeft toestemming gegeven voor een eerste proef met een nieuwe gentherapie voor SPG47.\" \/>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" 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