{"id":116987,"date":"2025-11-05T16:04:10","date_gmt":"2025-11-05T16:04:10","guid":{"rendered":"https:\/\/www.newsbeep.com\/il\/116987\/"},"modified":"2025-11-05T16:04:10","modified_gmt":"2025-11-05T16:04:10","slug":"fda-approves-treatment-for-rare-disease-offering-hope-to-florence-family","status":"publish","type":"post","link":"https:\/\/www.newsbeep.com\/il\/116987\/","title":{"rendered":"FDA approves treatment for rare disease, offering hope to Florence family"},"content":{"rendered":"<p>FLORENCE, S.C. (WPDE) \u2014 This week, the Food and Drug Administration (FDA) approved the first-ever treatment for people living with an extremely rare mitochondrial disease.<\/p>\n<p>Thymidine kinase 2 deficiency, or TK2D, is a rare, progressive, and life-threatening disorder that causes severe muscle weakness.<\/p>\n<p>Six-year-old Jeremiah of Florence was diagnosed when he was just eight months old. The disease makes it difficult for him to walk, eat, and even breathe on his own.<\/p>\n<p>But this week, for the first time, there\u2019s new hope.<\/p>\n<p>On Monday, a biopharmaceutical company announced FDA approval of Kygevvi, a powdered medication designed to treat both adults and children who develop symptoms before the age of 12.<\/p>\n<p>\u201cI honestly just cried,&#8221; Aneesa, Jeremiah&#8217;s mother, said. &#8220;It was one of those moments where I didn\u2019t quite have the words, and I literally just cried. I was extremely thankful, just speechless to be quite honest.\u201d<\/p>\n<p>Until now, there had been no FDA-approved treatments for TK2D.<\/p>\n<p>MORE: <a href=\"https:\/\/wpde.com\/news\/local\/mark-kruea-elected-mayor-of-myrtle-beach-brenda-bethune-mayoral-race-march-12-running-winner-city-council-honored-looking-retiring-april\" target=\"_blank\" rel=\"nofollow noopener\">Mark Kruea unofficially wins Myrtle Beach mayoral race<\/a><\/p>\n<p>Jeremiah has been part of a clinical trial for the past several years, helping lead the way to this breakthrough.<\/p>\n<p>\u201cHopefully this treatment increases access to those who may not have been a part of the clinical trial so let\u2019s just say somebody was born and of course diagnosed with tk2d instead of them having to find a clinical trial they now have access to the research medication that Jeremiah and a few others were on so it improves the access to medication in order to help those with tk2d to thrive,\u201d Aneesa said.<\/p>\n<p>According to Rare Disease Advisor, only 107 cases of TK2D had been reported worldwide as of 2018.<\/p>\n<p>Clinical studies show patients receiving the newly approved drug experienced an 86% reduction in the overall risk of death from the start of treatment.<\/p>\n<p>Jeremiah will continue taking Kygevvi three times a day, his mom says she\u2019s already seen progress.<\/p>\n<p>To learn more about Jeremiah&#8217;s journey and this new breakthrough, search \u201ctk2d&#8221; on our website.<\/p>\n","protected":false},"excerpt":{"rendered":"FLORENCE, S.C. (WPDE) \u2014 This week, the Food and Drug Administration (FDA) approved the first-ever treatment for people&hellip;\n","protected":false},"author":2,"featured_media":116988,"comment_status":"","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[33],"tags":[163,85,46,482],"class_list":{"0":"post-116987","1":"post","2":"type-post","3":"status-publish","4":"format-standard","5":"has-post-thumbnail","7":"category-medication","8":"tag-health","9":"tag-il","10":"tag-israel","11":"tag-medication"},"_links":{"self":[{"href":"https:\/\/www.newsbeep.com\/il\/wp-json\/wp\/v2\/posts\/116987","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.newsbeep.com\/il\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.newsbeep.com\/il\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/il\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/il\/wp-json\/wp\/v2\/comments?post=116987"}],"version-history":[{"count":0,"href":"https:\/\/www.newsbeep.com\/il\/wp-json\/wp\/v2\/posts\/116987\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/il\/wp-json\/wp\/v2\/media\/116988"}],"wp:attachment":[{"href":"https:\/\/www.newsbeep.com\/il\/wp-json\/wp\/v2\/media?parent=116987"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.newsbeep.com\/il\/wp-json\/wp\/v2\/categories?post=116987"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.newsbeep.com\/il\/wp-json\/wp\/v2\/tags?post=116987"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}