{"id":2547,"date":"2025-01-23T14:50:06","date_gmt":"2025-01-23T11:50:06","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=2547"},"modified":"2025-11-03T20:31:05","modified_gmt":"2025-11-03T17:31:05","slug":"fda-grants-rare-pediatric-disease-designation-to-nippon-shinyakus-exon-51-skipping-study-ns-051-ncnp-04","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/fr\/fda-grants-rare-pediatric-disease-designation-to-nippon-shinyakus-exon-51-skipping-study-ns-051-ncnp-04\/","title":{"rendered":"FDA accorde la d\u00e9signation de maladie p\u00e9diatrique rare \u00e0 l&#039;\u00e9tude de saut d&#039;exon 51 de Nippon Shinyaku (NS-051\/NCNP-04)"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Nippon Shinyaku et le Centre national de neurologie et de psychiatrie ont d\u00e9couvert conjointement l&#039;oligonucl\u00e9otide antisens NS-051\/NCNP-04. En omettant une partie du mat\u00e9riel g\u00e9n\u00e9tique du g\u00e8ne de la dystrophine, NS-051\/NCNP-04 cr\u00e9e une prot\u00e9ine dystrophine fonctionnelle avec une longueur de cha\u00eene l\u00e9g\u00e8rement plus courte, ce qui devrait emp\u00eacher le d\u00e9clin de la fonction musculaire.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Dot\u00e9e d&#039;un sens aigu du but, Nippon Shinyaku a activement d\u00e9velopp\u00e9 des m\u00e9dicaments pour gu\u00e9rir des maladies rares et incurables afin d&#039;introduire rapidement des solutions pour les patients atteints de DMD.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>En savoir plus<\/strong>:\u00a0<a href=\"https:\/\/dmdwarrior.com\/fr\/dmd-therapies\/\" target=\"_blank\" rel=\"noreferrer noopener\">Essais cliniques sur la dystrophie musculaire de Duchenne (Liste de toutes les recherches)<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Nippon Shinyaku and the National Center of Neurology and Psychiatry jointly discovered the antisense oligonucleotide NS-051\/NCNP-04. By omitting a portion of the dystrophin gene&#8217;s genetic material, NS-051\/NCNP-04 creates a functioning dystrophin protein with a little shorter chain length, which should prevent the decline in muscle function. With a strong sense of purpose, Nippon Shinyaku has [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":2550,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[193,194,88,136,192],"class_list":["post-2547","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-exon-51-skipping","tag-exon-51-treatment","tag-fda","tag-nippon-shinyaku","tag-ns-051-ncnp-04"],"subtitle":"Nippon Shinyaku a d\u00e9clar\u00e9 que le NS-051\/NCNP-04, un m\u00e9dicament en cours de d\u00e9veloppement pour traiter la dystrophie musculaire de Duchenne (DMD), a re\u00e7u la d\u00e9signation de maladie p\u00e9diatrique rare par la Food and Drug Administration (FDA).","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/posts\/2547","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=2547"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/posts\/2547\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/media\/2550"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/media?parent=2547"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/categories?post=2547"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/tags?post=2547"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}