{"id":130975,"date":"2021-11-20T18:24:19","date_gmt":"2021-11-21T02:24:19","guid":{"rendered":"https:\/\/lifeboat.com\/blog\/2021\/11\/fda-approves-first-drug-for-genetic-cause-of-dwarfism"},"modified":"2021-11-20T18:24:19","modified_gmt":"2021-11-21T02:24:19","slug":"fda-approves-first-drug-for-genetic-cause-of-dwarfism","status":"publish","type":"post","link":"https:\/\/lifeboat.com\/blog\/2021\/11\/fda-approves-first-drug-for-genetic-cause-of-dwarfism","title":{"rendered":"FDA approves first drug for genetic cause of dwarfism"},"content":{"rendered":"<p><a class=\"aligncenter blog-photo\" href=\"https:\/\/lifeboat.com\/blog.images\/fda-approves-first-drug-for-genetic-cause-of-dwarfism2.jpg\"><\/a><\/p>\n<p>The Food and Drug Administration <a href=\"https:\/\/www.prnewswire.com\/news-releases\/fda-approves-first-drug-to-improve-growth-in-children-with-most-common-form-of-dwarfism-301429137.html\" target=\"_blank\" rel=\"noopener\">approved<\/a> the first treatment for the most common cause of dwarfism Friday, a drug that has proved to increase children\u2019s height but has been polarizing among adults with short stature.<\/p>\n<p>The treatment, developed by BioMarin Pharmaceutical, is a once-daily injection for children with achondroplasia, a rare genetic disorder that results in dwarfism and can lead to serious medical complications. In <a href=\"https:\/\/www.thelancet.com\/journals\/lancet\/article\/PIIS0140-6736(20)31541-5\/fulltext\" target=\"_blank\" rel=\"noopener\">a pivotal clinical trial<\/a>, patients who got the drug, called Voxzogo, grew 1.6 centimeters more over the course of a year than those who received placebo. That means patients who take Voxzogo throughout childhood are likely to reach heights similar to their peers who don\u2019t have achondroplasia, according to BioMarin.<\/p>\n<p>\u201cIt\u2019s the difference between being able to drive a car or not, reaching stuff in closets, being able to take care of your hygiene,\u201d said Jean-Jacques Bienaim\u00e9, BioMarin\u2019s CEO. \u201cIt would make a huge difference for those patients. There\u2019s no question about it.\u201d<\/p>\n","protected":false},"excerpt":{"rendered":"<p>The Food and Drug Administration approved the first treatment for the most common cause of dwarfism Friday, a drug that has proved to increase children\u2019s height but has been polarizing among adults with short stature. The treatment, developed by BioMarin Pharmaceutical, is a once-daily injection for children with achondroplasia, a rare genetic disorder that results [\u2026]<\/p>\n","protected":false},"author":513,"featured_media":0,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[11,412],"tags":[],"class_list":["post-130975","post","type-post","status-publish","format-standard","hentry","category-biotech-medical","category-genetics"],"_links":{"self":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/posts\/130975","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/users\/513"}],"replies":[{"embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/comments?post=130975"}],"version-history":[{"count":0,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/posts\/130975\/revisions"}],"wp:attachment":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/media?parent=130975"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/categories?post=130975"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/tags?post=130975"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}