{"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\/pt\/fda-grants-orphan-drug-designation-to-ns-051-ncnp-04-for-the-treatment-of-duchenne-muscular-dystrophy\/","title":{"rendered":"FDA concede designa\u00e7\u00e3o de medicamento \u00f3rf\u00e3o ao NS-051\/NCNP-04 para o tratamento da distrofia muscular de Duchenne"},"content":{"rendered":"<p class=\"wp-block-paragraph\"><strong>A Nippon Shinyaku anunciou que a Food and Drug Administration (FDA) dos EUA concedeu a designa\u00e7\u00e3o de medicamento \u00f3rf\u00e3o ao NS-051\/NCNP-04, que est\u00e1 sendo desenvolvido para o tratamento da distrofia muscular de Duchenne (DMD).<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">A Designa\u00e7\u00e3o de Medicamento \u00d3rf\u00e3o nos EUA \u00e9 concedida para tratamentos de doen\u00e7as que afetam menos de 200.000 pacientes nos EUA, apoiando seu desenvolvimento e avalia\u00e7\u00e3o. A Designa\u00e7\u00e3o de Medicamento \u00d3rf\u00e3o concede ao NS-051\/NCNP-04 sete anos de direitos exclusivos de comercializa\u00e7\u00e3o. <a href=\"https:\/\/dmdwarrior.com\/pt\/fda-grants-rare-pediatric-disease-designation-to-nippon-shinyakus-exon-51-skipping-study-ns-051-ncnp-04\/\" target=\"_blank\" rel=\"noreferrer noopener\">NS-051\/NCNP-04 j\u00e1 recebeu a designa\u00e7\u00e3o de doen\u00e7a pedi\u00e1trica rara pelo FDA em janeiro de 2025.<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">A DMD \u00e9 uma doen\u00e7a progressiva que atrofia muscular causada pela defici\u00eancia da prote\u00edna distrofina. Ela leva \u00e0 fraqueza dos m\u00fasculos esquel\u00e9ticos, card\u00edacos e respirat\u00f3rios. Existem muitos tipos de muta\u00e7\u00f5es gen\u00e9ticas que podem causar DMD, e a NS-051\/NCNP-04 est\u00e1 sendo desenvolvida para tratar pacientes com muta\u00e7\u00f5es gen\u00e9ticas confirmadas pass\u00edveis de terapia de skipping do exon 51. Saiba mais: <a href=\"https:\/\/dmdwarrior.com\/pt\/mutations-and-deletions-amenable-to-exon-51-skipping-therapies-dmd\/\" target=\"_blank\" rel=\"noreferrer noopener\">Muta\u00e7\u00f5es e dele\u00e7\u00f5es pass\u00edveis de terapias de salto do exon 51<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">NS-051\/NCNP-04 \u00e9 um oligonucleot\u00eddeo antisense descoberto em conjunto pelo Centro Nacional de Neurologia e Psiquiatria (NCNP, Cidade de Kodaira em T\u00f3quio; Presidente, Kazuyuki Nakagome) e Nippon Shinyaku. NS-051\/NCNP-04 ignora parte da informa\u00e7\u00e3o gen\u00e9tica do gene da distrofina e produz uma prote\u00edna distrofina funcional com um comprimento de cadeia ligeiramente menor, o que deve ter o efeito de suprimir a deteriora\u00e7\u00e3o da fun\u00e7\u00e3o muscular.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Na Nippon Shinyaku, estamos comprometidos em desenvolver novas terapias inovadoras para doen\u00e7as intrat\u00e1veis e raras com um senso de miss\u00e3o e trabalhando ativamente para trazer novas op\u00e7\u00f5es de tratamento para pacientes com DMD o mais r\u00e1pido poss\u00edvel.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Saber mais<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/pt\/exon-51-skipping-therapies-dmd\/\">Pr\u00f3ximas terapias de salto do Exon 51 para o tratamento da distrofia muscular 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\/pt\/wp-json\/wp\/v2\/posts\/4512","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/comments?post=4512"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/posts\/4512\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/media\/4514"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/media?parent=4512"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/categories?post=4512"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/tags?post=4512"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}