{"id":327650,"date":"2021-10-20T02:00:00","date_gmt":"2021-10-20T00:00:00","guid":{"rendered":"https:\/\/medizinonline.com\/single-agent-gene-therapy-shows-transformative-therapeutic-benefit\/"},"modified":"2021-10-20T02:00:00","modified_gmt":"2021-10-20T00:00:00","slug":"single-agent-gene-therapy-shows-transformative-therapeutic-benefit","status":"publish","type":"post","link":"https:\/\/medizinonline.com\/en\/single-agent-gene-therapy-shows-transformative-therapeutic-benefit\/","title":{"rendered":"Single-agent gene therapy shows transformative therapeutic benefit"},"content":{"rendered":"<p><strong>New trial data demonstrate age-appropriate development and achievement of motor milestones in presymptomatic children with SMA using single-agent gene therapy, without any respiratory or nutritional support or serious treatment-related adverse events.<\/strong><\/p>\n<p> <!--more--> <\/p>\n<p>If left untreated, spinal muscular atrophy (SMA) type 1 often leads to death or the need for permanent ventilation by the age of two. The SPR1NT study included children with two SMN2 copies presymptomatic. They received the only single-agent causal gene therapy to date, onasemnogen abeparvovec (Zolgensma\u00ae). The results demonstrate that all children were alive, free of ventilatory and nutritional support, and met the primary end point of sitting independently for \u226530 seconds. 11 of the 14 patients (78.6%) reached this milestone within the WHO-defined period of expected normal development. In addition, 11 of the 14 patients were subsequently able to stand independently and 9 of 14 were even able to walk independently. Most patients reached these milestones within the normal period of age-appropriate development.<\/p>\n<p>The STR1VE-EU study evaluated the efficacy and safety of a single, one-time i.v. infusion of Zolgensma\u00ae in patients with SMA type 1 and a biallelic deletion in the SMN1 gene or point mutations and one or two copies of the SMN2 gene. Of the 33 patients included, nine (27%) required nutritional support at baseline, another nine (27%) required ventilatory support at baseline, and five (15%) required both. It was found that with therapy, the majority of symptomatic patients with SMA type 1 (82%), including those with a more severe course of SMA, achieved milestones of motor development not previously observed in the natural history of SMA type 1. Sixteen of these children (49%) were even able to sit independently for \u226530 seconds. The exploratory endpoint, &#8220;ability to develop in an age-appropriate manner&#8221; in symptomatic patients, including swallowing, oral feeding, and age-appropriate weight maintenance, was also evaluated. Twenty-three patients belonged to the &#8216;ability to thrive&#8217; population, of whom seven patients (30%) met the criteria at 18 months of age.<\/p>\n<p><em>Source: New data on Zolgensma\u00ae show age-appropriate progression in presymptomatic patients and rapid, clinically meaningful efficacy in symptomatic patients, including those with severe SMA at baseline, Jun. 23, 2021, Novartis<\/em><\/p>\n<p>&nbsp;<\/p>\n<p><em>InFo NEUROLOGY &amp; PSYCHIATRY 2021; 19(4): 33.<\/em><\/p>\n","protected":false},"excerpt":{"rendered":"<p>New trial data demonstrate age-appropriate development and achievement of motor milestones in presymptomatic children with SMA using single-agent gene therapy, without any respiratory or nutritional support or serious treatment-related adverse&hellip;<\/p>\n","protected":false},"author":7,"featured_media":111076,"comment_status":"closed","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"pmpro_default_level":"","cat_1_feature_home_top":false,"cat_2_editor_pick":false,"csco_eyebrow_text":"Spinal muscular atrophy","footnotes":""},"category":[11396,11530,11363,11548,11503],"tags":[17065,17067],"powerkit_post_featured":[],"class_list":["post-327650","post","type-post","status-publish","format-standard","has-post-thumbnail","category-genetics","category-market-medicine","category-neurology","category-rx-en","category-studies","tag-sma-en","tag-spinal-muscular-atrophy","pmpro-has-access"],"acf":[],"publishpress_future_action":{"enabled":false,"date":"2026-06-25 12:30:53","action":"change-status","newStatus":"draft","terms":[],"taxonomy":"category","extraData":[]},"publishpress_future_workflow_manual_trigger":{"enabledWorkflows":[]},"wpml_current_locale":"en_US","wpml_translations":{"fr_FR":{"locale":"fr_FR","id":327654,"slug":"la-therapie-genique-a-dose-unique-montre-des-avantages-therapeutiques-transformateurs","post_title":"La th\u00e9rapie g\u00e9nique \u00e0 dose unique montre des avantages th\u00e9rapeutiques transformateurs","href":"https:\/\/medizinonline.com\/fr\/la-therapie-genique-a-dose-unique-montre-des-avantages-therapeutiques-transformateurs\/"},"it_IT":{"locale":"it_IT","id":327657,"slug":"la-terapia-genica-una-tantum-mostra-benefici-terapeutici-trasformativi","post_title":"La terapia genica una tantum mostra benefici terapeutici trasformativi","href":"https:\/\/medizinonline.com\/it\/la-terapia-genica-una-tantum-mostra-benefici-terapeutici-trasformativi\/"},"pt_PT":{"locale":"pt_PT","id":327658,"slug":"a-terapia-genetica-unica-mostra-beneficios-terapeuticos-transformadores","post_title":"A terapia gen\u00e9tica \u00fanica mostra benef\u00edcios terap\u00eauticos transformadores","href":"https:\/\/medizinonline.com\/pt-pt\/a-terapia-genetica-unica-mostra-beneficios-terapeuticos-transformadores\/"},"es_ES":{"locale":"es_ES","id":327637,"slug":"la-terapia-genica-unica-muestra-beneficios-terapeuticos-transformadores","post_title":"La terapia g\u00e9nica \u00fanica muestra beneficios terap\u00e9uticos transformadores","href":"https:\/\/medizinonline.com\/es\/la-terapia-genica-unica-muestra-beneficios-terapeuticos-transformadores\/"}},"_links":{"self":[{"href":"https:\/\/medizinonline.com\/en\/wp-json\/wp\/v2\/posts\/327650","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/medizinonline.com\/en\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/medizinonline.com\/en\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/medizinonline.com\/en\/wp-json\/wp\/v2\/users\/7"}],"replies":[{"embeddable":true,"href":"https:\/\/medizinonline.com\/en\/wp-json\/wp\/v2\/comments?post=327650"}],"version-history":[{"count":0,"href":"https:\/\/medizinonline.com\/en\/wp-json\/wp\/v2\/posts\/327650\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/medizinonline.com\/en\/wp-json\/wp\/v2\/media\/111076"}],"wp:attachment":[{"href":"https:\/\/medizinonline.com\/en\/wp-json\/wp\/v2\/media?parent=327650"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/medizinonline.com\/en\/wp-json\/wp\/v2\/category?post=327650"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/medizinonline.com\/en\/wp-json\/wp\/v2\/tags?post=327650"},{"taxonomy":"powerkit_post_featured","embeddable":true,"href":"https:\/\/medizinonline.com\/en\/wp-json\/wp\/v2\/powerkit_post_featured?post=327650"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}