{"id":4536,"date":"2025-08-08T12:52:19","date_gmt":"2025-08-08T16:52:19","guid":{"rendered":"https:\/\/www.med.unc.edu\/pediatrics\/muenzer-mps-center\/?p=4536"},"modified":"2025-12-23T16:51:57","modified_gmt":"2025-12-23T21:51:57","slug":"fda-delays-put-lives-at-risk-for-ultrarare-disease","status":"publish","type":"post","link":"https:\/\/www.med.unc.edu\/pediatrics\/muenzer-mps-center\/2025\/08\/fda-delays-put-lives-at-risk-for-ultrarare-disease\/","title":{"rendered":"FDA Delays Put Lives at Risk for Ultrarare Disease"},"content":{"rendered":"<figure id=\"attachment_3052\" class=\"thumbnail wp-caption alignleft\" style=\"width: 255px\"><img loading=\"lazy\" decoding=\"async\" class=\"size-medium wp-image-3052\" src=\"https:\/\/www.med.unc.edu\/pediatrics\/muenzer-mps-center\/wp-content\/uploads\/sites\/1312\/2024\/07\/dr-muenzer-2-245x300.jpg\" alt=\"Dr. Muenzer\" width=\"245\" height=\"300\" srcset=\"https:\/\/www.med.unc.edu\/pediatrics\/muenzer-mps-center\/wp-content\/uploads\/sites\/1312\/2024\/07\/dr-muenzer-2-245x300.jpg 245w, https:\/\/www.med.unc.edu\/pediatrics\/muenzer-mps-center\/wp-content\/uploads\/sites\/1312\/2024\/07\/dr-muenzer-2-768x942.jpg 768w, https:\/\/www.med.unc.edu\/pediatrics\/muenzer-mps-center\/wp-content\/uploads\/sites\/1312\/2024\/07\/dr-muenzer-2-600x736.jpg 600w, https:\/\/www.med.unc.edu\/pediatrics\/muenzer-mps-center\/wp-content\/uploads\/sites\/1312\/2024\/07\/dr-muenzer-2.jpg 800w\" sizes=\"auto, (max-width: 245px) 100vw, 245px\" \/><figcaption class=\"caption wp-caption-text\">Dr. Muenzer<\/figcaption><\/figure>\n<p>In this <em>BioCentury<\/em>\u00a0op-ed, Dr. Joseph Muenzer joins other advocates in calling for a fit-for-purpose regulatory pathway for ultrarare diseases. Standard placebo-controlled clinical trials are often not feasible for these conditions, especially those affecting the brain where symptom onset and progression vary greatly between patients. These challenges can add years to the approval process, leaving patients without treatment while irreversible damage occurs.<\/p>\n<p>Read the full article:\u00a0<a href=\"https:\/\/www.biocentury.com\/article\/656694\/fda-delays-mean-more-deaths-ultrarare-diseases-need-a-fit-for-purpose-pathway\" target=\"_blank\" rel=\"noopener\">FDA delays mean more deaths: Ultrarare diseases need a fit-for-purpose pathway<\/a>.<\/p>\n","protected":false},"excerpt":{"rendered":"<p>In this BioCentury\u00a0op-ed, Dr. Joseph Muenzer joins other advocates in calling for a fit-for-purpose regulatory pathway for ultrarare diseases. Standard placebo-controlled clinical trials are often not feasible for these conditions, especially those affecting the brain where symptom onset and progression vary greatly between patients. These challenges can add years to the approval process, leaving patients &hellip; <a href=\"https:\/\/www.med.unc.edu\/pediatrics\/muenzer-mps-center\/2025\/08\/fda-delays-put-lives-at-risk-for-ultrarare-disease\/\" aria-label=\"Read more about FDA Delays Put Lives at Risk for Ultrarare Disease\">Read more<\/a><\/p>\n","protected":false},"author":119071,"featured_media":4596,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"layout":"","cellInformation":"","apiCallInformation":"","footnotes":"","_links_to":"","_links_to_target":""},"categories":[1],"tags":[],"class_list":["post-4536","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-news","odd"],"acf":[],"featured_image":"https:\/\/www.med.unc.edu\/pediatrics\/muenzer-mps-center\/wp-content\/uploads\/sites\/1312\/2025\/12\/311a4f2c-72f2-44e7-817c-f61a4d7935f8.jpg","featured_image_medium":"https:\/\/www.med.unc.edu\/pediatrics\/muenzer-mps-center\/wp-content\/uploads\/sites\/1312\/2025\/12\/311a4f2c-72f2-44e7-817c-f61a4d7935f8-300x300.jpg","featured_image_medium_large":"https:\/\/www.med.unc.edu\/pediatrics\/muenzer-mps-center\/wp-content\/uploads\/sites\/1312\/2025\/12\/311a4f2c-72f2-44e7-817c-f61a4d7935f8-768x768.jpg","featured_image_large":"https:\/\/www.med.unc.edu\/pediatrics\/muenzer-mps-center\/wp-content\/uploads\/sites\/1312\/2025\/12\/311a4f2c-72f2-44e7-817c-f61a4d7935f8-1024x1024.jpg","featured_image_thumbnail":"https:\/\/www.med.unc.edu\/pediatrics\/muenzer-mps-center\/wp-content\/uploads\/sites\/1312\/2025\/12\/311a4f2c-72f2-44e7-817c-f61a4d7935f8-150x150.jpg","featured_image_alt":"Dr. Joseph Muenzer","category_details":[{"name":"News","link":"https:\/\/www.med.unc.edu\/pediatrics\/muenzer-mps-center\/category\/news\/"}],"tag_details":[],"_links_to":[],"_links_to_target":[],"_links":{"self":[{"href":"https:\/\/www.med.unc.edu\/pediatrics\/muenzer-mps-center\/wp-json\/wp\/v2\/posts\/4536","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.med.unc.edu\/pediatrics\/muenzer-mps-center\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.med.unc.edu\/pediatrics\/muenzer-mps-center\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.med.unc.edu\/pediatrics\/muenzer-mps-center\/wp-json\/wp\/v2\/users\/119071"}],"replies":[{"embeddable":true,"href":"https:\/\/www.med.unc.edu\/pediatrics\/muenzer-mps-center\/wp-json\/wp\/v2\/comments?post=4536"}],"version-history":[{"count":4,"href":"https:\/\/www.med.unc.edu\/pediatrics\/muenzer-mps-center\/wp-json\/wp\/v2\/posts\/4536\/revisions"}],"predecessor-version":[{"id":4602,"href":"https:\/\/www.med.unc.edu\/pediatrics\/muenzer-mps-center\/wp-json\/wp\/v2\/posts\/4536\/revisions\/4602"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.med.unc.edu\/pediatrics\/muenzer-mps-center\/wp-json\/wp\/v2\/media\/4596"}],"wp:attachment":[{"href":"https:\/\/www.med.unc.edu\/pediatrics\/muenzer-mps-center\/wp-json\/wp\/v2\/media?parent=4536"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.med.unc.edu\/pediatrics\/muenzer-mps-center\/wp-json\/wp\/v2\/categories?post=4536"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.med.unc.edu\/pediatrics\/muenzer-mps-center\/wp-json\/wp\/v2\/tags?post=4536"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}