{"id":4512,"date":"2025-09-19T13:20:49","date_gmt":"2025-09-19T10:20:49","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=4512"},"modified":"2025-09-19T17:03:29","modified_gmt":"2025-09-19T14:03:29","slug":"fda-grants-orphan-drug-designation-to-ns-051-ncnp-04-for-the-treatment-of-duchenne-muscular-dystrophy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/fr\/fda-grants-orphan-drug-designation-to-ns-051-ncnp-04-for-the-treatment-of-duchenne-muscular-dystrophy\/","title":{"rendered":"FDA accorde la d\u00e9signation de m\u00e9dicament orphelin au NS-051\/NCNP-04 pour le traitement de la dystrophie musculaire de Duchenne"},"content":{"rendered":"<p class=\"wp-block-paragraph\"><strong>Nippon Shinyaku a annonc\u00e9 que la Food and Drug Administration am\u00e9ricaine (FDA) a accord\u00e9 la d\u00e9signation de m\u00e9dicament orphelin au NS-051\/NCNP-04 qui est en cours de d\u00e9veloppement pour le traitement de la dystrophie musculaire de Duchenne (DMD).<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Aux \u00c9tats-Unis, la d\u00e9signation de m\u00e9dicament orphelin est accord\u00e9e aux traitements contre des maladies touchant moins de 200\u00a0000 patients, soutenant ainsi leur d\u00e9veloppement et leur \u00e9valuation. Cette d\u00e9signation conf\u00e8re au m\u00e9dicament NS-051\/NCNP-04 des droits de commercialisation exclusifs pendant sept ans. <a href=\"https:\/\/dmdwarrior.com\/fr\/fda-grants-rare-pediatric-disease-designation-to-nippon-shinyakus-exon-51-skipping-study-ns-051-ncnp-04\/\" target=\"_blank\" rel=\"noreferrer noopener\">Le NS-051\/NCNP-04 a d\u00e9j\u00e0 re\u00e7u la d\u00e9signation de maladie p\u00e9diatrique rare par le FDA en janvier 2025.<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">La DMD est une maladie progressive d&#039;atrophie musculaire caus\u00e9e par un d\u00e9ficit en dystrophine. Elle entra\u00eene une faiblesse des muscles squelettiques, cardiaques et respiratoires. De nombreux types de mutations g\u00e9n\u00e9tiques peuvent \u00eatre \u00e0 l&#039;origine de la DMD, et le NS-051\/NCNP-04 est en cours de d\u00e9veloppement pour traiter les patients pr\u00e9sentant des mutations g\u00e9n\u00e9tiques confirm\u00e9es et susceptibles d&#039;\u00eatre trait\u00e9s par un traitement par saut d&#039;exon 51. En savoir plus\u00a0: <a href=\"https:\/\/dmdwarrior.com\/fr\/mutations-and-deletions-amenable-to-exon-51-skipping-therapies-dmd\/\" target=\"_blank\" rel=\"noreferrer noopener\">Mutations et d\u00e9l\u00e9tions sensibles aux th\u00e9rapies par saut d&#039;exon 51<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">NS-051\/NCNP-04 est un oligonucl\u00e9otide antisens co-d\u00e9couvert par le Centre national de neurologie et de psychiatrie (NCNP, Kodaira City \u00e0 Tokyo ; pr\u00e9sident, Kazuyuki Nakagome) et Nippon Shinyaku. NS-051\/NCNP-04 saute une partie de l&#039;information g\u00e9n\u00e9tique du g\u00e8ne de la dystrophine et produit une prot\u00e9ine de dystrophine fonctionnelle avec une longueur de cha\u00eene l\u00e9g\u00e8rement plus courte, ce qui devrait avoir pour effet de supprimer la d\u00e9t\u00e9rioration de la fonction musculaire.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Chez Nippon Shinyaku, nous nous engageons \u00e0 d\u00e9velopper de nouvelles th\u00e9rapies innovantes pour les maladies incurables et rares avec un sens de la mission et travaillons activement pour apporter de nouvelles options de traitement aux patients atteints de DMD d\u00e8s que possible.<\/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\/\">Th\u00e9rapies par saut d&#039;exon 51 \u00e0 venir pour le traitement de la dystrophie musculaire de Duchenne<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Nippon Shinyaku announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to NS-051\/NCNP-04 which is being developed for the treatment of Duchenne Muscular Dystrophy (DMD). The Orphan Drug Designation in the U.S. is granted for treatments for diseases affecting fewer than 200,000 patients in the U.S., supporting their development and [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":4514,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[122,193,421,194,136,192,195],"class_list":["post-4512","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-exon-51","tag-exon-51-skipping","tag-exon-51-skipping-therapy","tag-exon-51-treatment","tag-nippon-shinyaku","tag-ns-051-ncnp-04","tag-orphan-drug"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/posts\/4512","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=4512"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/posts\/4512\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/media\/4514"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/media?parent=4512"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/categories?post=4512"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/tags?post=4512"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}