{"id":4274,"date":"2025-08-04T17:03:02","date_gmt":"2025-08-04T14:03:02","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=4274"},"modified":"2025-08-04T17:13:24","modified_gmt":"2025-08-04T14:13:24","slug":"dyne-therapeutics-announces-fda-breakthrough-therapy-designation-for-dyne-251-in-duchenne-muscular-dystrophy-dmd","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/fr\/dyne-therapeutics-announces-fda-breakthrough-therapy-designation-for-dyne-251-in-duchenne-muscular-dystrophy-dmd\/","title":{"rendered":"Dyne Therapeutics annonce la d\u00e9signation de th\u00e9rapie r\u00e9volutionnaire FDA pour DYNE-251 dans la dystrophie musculaire de Duchenne (DMD)"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Dyne Therapeutics a annonc\u00e9 que la Food and Drug Administration (FDA) am\u00e9ricaine (FDA) a accord\u00e9 la d\u00e9signation de th\u00e9rapie innovante \u00e0 DYNE-251 pour le traitement des patients atteints de dystrophie musculaire de Duchenne (DMD) sensibles au saut d&#039;exon 51. Cette d\u00e9signation s&#039;appuie sur les donn\u00e9es de l&#039;essai clinique DELIVER en cours.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">DYNE-251\u00a0: D\u00e9signation de th\u00e9rapie r\u00e9volutionnaire<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u00ab Cette d\u00e9signation de th\u00e9rapie r\u00e9volutionnaire pour DYNE-251 t\u00e9moigne de son potentiel en tant que th\u00e9rapie de nouvelle g\u00e9n\u00e9ration con\u00e7ue pour apporter une am\u00e9lioration fonctionnelle significative aux personnes atteintes de DMD chez qui le saut de l&#039;exon 51 peut entra\u00eener la production de dystrophine quasi-int\u00e9grale \u00bb, a d\u00e9clar\u00e9 Doug Kerr, MD, Ph.D., directeur m\u00e9dical de Dyne. \u00ab Comme nous l&#039;avons d\u00e9j\u00e0 annonc\u00e9, DYNE-251 a d\u00e9montr\u00e9 une am\u00e9lioration fonctionnelle soutenue sur dix-huit mois, \u00e9valu\u00e9e par des mesures cl\u00e9s telles que le temps de lever et le 95e centile de la vitesse de foul\u00e9e. Le niveau d&#039;expression de dystrophine quasi-int\u00e9grale observ\u00e9 marque une avanc\u00e9e significative vers des b\u00e9n\u00e9fices potentiels significatifs pour les patients. \u00bb<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Apprendre encore plus<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/fr\/exon-51-skipping-therapies-dmd\/\" target=\"_blank\" rel=\"noreferrer noopener\">Th\u00e9rapies par saut d&#039;exon 51 \u00e0 venir pour le traitement de la dystrophie musculaire de Duchenne<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>En savoir plus<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/fr\/dyne-therapeutics-receives-european-medicines-agency-ema-orphan-drug-designation-for-dyne-251-in-duchenne-muscular-dystrophy\/\">Dyne Therapeutics re\u00e7oit la d\u00e9signation de m\u00e9dicament orphelin de l&#039;Agence europ\u00e9enne des m\u00e9dicaments (EMA) pour DYNE-251 dans la dystrophie musculaire de Duchenne<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Dyne Therapeutics announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to DYNE-251 for the treatment of patients with Duchenne muscular dystrophy (DMD), amenable to exon 51 skipping. The designation is based on data from the ongoing DELIVER clinical trial. DYNE-251: Breakthrough Therapy Designation \u201cThis Breakthrough Therapy Designation for DYNE-251 [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":4275,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[208,145,122,193,194,88],"class_list":["post-4274","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-dyne-therapeutics","tag-dyne-251","tag-exon-51","tag-exon-51-skipping","tag-exon-51-treatment","tag-fda"],"subtitle":"To treat Duchenne muscular dystrophy (DMD) patients who can skip exon 51, Dyne Therapeutics announced that the FDA has given Breakthrough Therapy Designation to DYNE-251. From the ongoing DELIVER clinical trial, the designation was made.","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/posts\/4274","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/comments?post=4274"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/posts\/4274\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/media\/4275"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/media?parent=4274"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/categories?post=4274"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/tags?post=4274"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}