{"id":226768,"date":"2025-12-09T01:38:05","date_gmt":"2025-12-09T07:38:05","guid":{"rendered":"https:\/\/lifeboat.com\/blog\/2025\/12\/gene-therapy-improves-movement-in-kids-with-spinal-muscular-atrophy"},"modified":"2025-12-09T01:38:05","modified_gmt":"2025-12-09T07:38:05","slug":"gene-therapy-improves-movement-in-kids-with-spinal-muscular-atrophy","status":"publish","type":"post","link":"https:\/\/lifeboat.com\/blog\/2025\/12\/gene-therapy-improves-movement-in-kids-with-spinal-muscular-atrophy","title":{"rendered":"Gene therapy improves movement in kids with spinal muscular atrophy"},"content":{"rendered":"<p><a class=\"aligncenter blog-photo\" href=\"https:\/\/lifeboat.com\/blog.images\/gene-therapy-improves-movement-in-kids-with-spinal-muscular-atrophy.jpg\"><\/a><\/p>\n<p>A single-dose gene replacement therapy is found to improve movement ability in children over 2 years of age and teenagers with spinal muscular atrophy, according to research <a href=\"https:\/\/www.nature.com\/articles\/s41591-025-04103-w\" target=\"_blank\">published<\/a> in <i>Nature Medicine<\/i>. The results of this phase 3 clinical trial, involving 126 children and adolescents, could support an alternative to lifelong, repeat-dose treatments for people living with spinal atrophy beyond the age of 2 years.<\/p>\n<p>Spinal muscular atrophy is a rare genetic condition that causes muscle weakness and loss of movement over time. It develops because the body cannot make enough of a protein, called <a href=\"https:\/\/medicalxpress.com\/news\/2023-06-world-beautiful-disease-expensive.html?utm_source=embeddings&utm_medium=related&utm_campaign=internal\" rel=\"related\">survival motor neuron<\/a>, needed for healthy nerve cells.<\/p>\n<p>Onasemnogene abeparvovec is a gene therapy that restores production of this missing protein in a single treatment. However, it is currently approved in the U.S. and Europe only as a single intravenous treatment for children under 2 years of age. Therefore, those older than 2 years of age can receive treatments only to slow the disease, and these must be taken regularly, either by injection or orally.<\/p>\n","protected":false},"excerpt":{"rendered":"<p>A single-dose gene replacement therapy is found to improve movement ability in children over 2 years of age and teenagers with spinal muscular atrophy, according to research published in Nature Medicine. The results of this phase 3 clinical trial, involving 126 children and adolescents, could support an alternative to lifelong, repeat-dose treatments for people living [\u2026]<\/p>\n","protected":false},"author":662,"featured_media":0,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[11,412,47],"tags":[],"class_list":["post-226768","post","type-post","status-publish","format-standard","hentry","category-biotech-medical","category-genetics","category-neuroscience"],"_links":{"self":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/posts\/226768","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\/662"}],"replies":[{"embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/comments?post=226768"}],"version-history":[{"count":0,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/posts\/226768\/revisions"}],"wp:attachment":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/media?parent=226768"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/categories?post=226768"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/tags?post=226768"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}