{"id":2948,"date":"2021-06-10T14:00:30","date_gmt":"2021-06-10T13:00:30","guid":{"rendered":"https:\/\/mft.nhs.uk\/rmch\/?p=2948"},"modified":"2025-10-23T13:39:32","modified_gmt":"2025-10-23T13:39:32","slug":"patient-at-royal-manchester-childrens-hospital-is-first-in-the-north-of-england-to-receive-life-saving-gene-therapy-drug","status":"publish","type":"post","link":"https:\/\/mft.nhs.uk\/rmch\/patient-at-royal-manchester-childrens-hospital-is-first-in-the-north-of-england-to-receive-life-saving-gene-therapy-drug\/","title":{"rendered":"Patient at Royal Manchester Children\u2019s Hospital is first in the North of England to receive life-saving gene-therapy drug"},"content":{"rendered":"<div class=\"wpb-content-wrapper\"><p>[vc_row][vc_column][vc_column_text]A life-saving drug that can improve mobility in babies and young children suffering from a rare genetic condition has been given to a patient for the first time in the North of England at Royal Manchester Children\u2019s Hospital.<\/p>\n<p>Zolgensma is a one-off gene therapy that treats Spinal Muscular Atrophy (SMA), a rare and often fatal genetic disease that causes paralysis, muscle weakness and progressive loss of movement.<\/p>\n<p>The latest data suggests that Zolgensma can provide improvement in motor function for young children with a specific type of SMA and prolong their lives.<\/p>\n<p>Zolgensma, which has a list price of \u00a31.79 million per dose, was made available on the NHS following a landmark deal struck with manufacturers Novartis Gene Therapies in March.<\/p>\n<p>Royal Manchester Children\u2019s Hospital (RMCH) is part of Manchester University NHS Foundation Trust, which is one of four centres approved for the use of Zolgensma for the treatment for Type-1 SMA. These are:<\/p>\n<ul>\n<li>Evelina London Children\u2019s Hospital (part of Guy\u2019s &amp; St Thomas\u2019 NHS Foundation Trust) and with agreement to develop a sub hub at Great Ormond Street Hospital for Children NHS Foundation Trust<\/li>\n<li>Manchester University NHS Foundation Trust<\/li>\n<li>Sheffield Children\u2019s NHS Foundation Trust<\/li>\n<li>University Hospitals Bristol and Weston NHS Foundation Trust<\/li>\n<\/ul>\n<p>The Trusts will be working as a national Multi-Disciplinary Team in collaboration with regional Neuromuscular Centres across the country to share expertise and experience for this new therapy.<\/p>\n<p>Clinical teams at RMCH will assess patients individually for their eligibility and, alongside the family, consider whether treatment with Zolgensma is the best approach.<\/p>\n<p>This recent treatment marks another significant milestone for gene therapies in the NHS and adds to the suite of Advanced Therapeutic Medicinal Products available at RMCH.<\/p>\n<p>Dr Gary McCullagh, Consultant Paediatric Neurologist at RMCH said: \u201cWe are very pleased to be able to offer this treatment for our young patients affected by this debilitating, life-limiting disease. Although rare, Spinal Muscular Atrophy is still the leading genetic cause of death among young children. This new treatment will not only save lives, but will also improve quality of life for some children.\u201d<\/p>\n<p>Jane Valente, Medical Director at RMCH said: \u201cThe provision of this promising new gene therapy will come as very welcome news to families across the country. Here at Royal Manchester Children\u2019s Hospital we look forward to providing this new service for our patients across the North West and beyond.\u201d<\/p>\n<p><img loading=\"lazy\" decoding=\"async\" class=\"aligncenter size-large wp-image-2949 wpsmartcrop-image\" src=\"https:\/\/mft.nhs.uk\/app\/uploads\/sites\/8\/2021\/06\/RMCH-Zolgensma-team-scaled-1024x768.jpg\" alt=\"\" width=\"1024\" height=\"768\" srcset=\"https:\/\/mft.nhs.uk\/app\/uploads\/sites\/8\/2021\/06\/RMCH-Zolgensma-team-scaled-1024x768.jpg 1024w, https:\/\/mft.nhs.uk\/app\/uploads\/sites\/8\/2021\/06\/RMCH-Zolgensma-team-scaled-300x225.jpg 300w, https:\/\/mft.nhs.uk\/app\/uploads\/sites\/8\/2021\/06\/RMCH-Zolgensma-team-scaled-768x576.jpg 768w, https:\/\/mft.nhs.uk\/app\/uploads\/sites\/8\/2021\/06\/RMCH-Zolgensma-team-scaled-1536x1152.jpg 1536w, https:\/\/mft.nhs.uk\/app\/uploads\/sites\/8\/2021\/06\/RMCH-Zolgensma-team-scaled-2048x1536.jpg 2048w, https:\/\/mft.nhs.uk\/app\/uploads\/sites\/8\/2021\/06\/RMCH-Zolgensma-team-scaled-60x45.jpg 60w, https:\/\/mft.nhs.uk\/app\/uploads\/sites\/8\/2021\/06\/RMCH-Zolgensma-team-scaled-680x510.jpg 680w\" sizes=\"auto, (max-width: 1024px) 100vw, 1024px\" data-smartcrop-focus=\"[50,50]\" \/>[\/vc_column_text][\/vc_column][\/vc_row]<\/p>\n<\/div>","protected":false},"excerpt":{"rendered":"<p>[vc_row][vc_column][vc_column_text]A life-saving drug that can improve mobility in babies and young children suffering from a rare genetic condition has been given to a patient for the first time in the North of England at Royal Manchester Children\u2019s Hospital. Zolgensma is a one-off gene therapy that treats Spinal Muscular Atrophy (SMA), a rare and often fatal [&hellip;]<\/p>\n","protected":false},"author":23,"featured_media":2949,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"footnotes":""},"categories":[8,136],"tags":[],"class_list":["post-2948","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-news","category-patient-stories"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v26.9 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>Patient at Royal Manchester Children\u2019s Hospital is first in the North of England to receive life-saving gene-therapy drug - Royal Manchester Children&#039;s Hospital<\/title>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/mft.nhs.uk\/rmch\/patient-at-royal-manchester-childrens-hospital-is-first-in-the-north-of-england-to-receive-life-saving-gene-therapy-drug\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Patient at Royal Manchester Children\u2019s Hospital is first in the North of England to receive life-saving gene-therapy drug - Royal Manchester Children&#039;s Hospital\" \/>\n<meta property=\"og:description\" content=\"[vc_row][vc_column][vc_column_text]A life-saving drug that can improve mobility in babies and young children suffering from a rare genetic condition has been given to a patient for the first time in the North of England at Royal Manchester Children\u2019s Hospital. 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