{"id":306772,"date":"2025-12-09T10:51:12","date_gmt":"2025-12-09T10:51:12","guid":{"rendered":"https:\/\/www.newsbeep.com\/uk\/306772\/"},"modified":"2025-12-09T10:51:12","modified_gmt":"2025-12-09T10:51:12","slug":"pioneering-duchenne-drug-approved-for-use-on-nhs-scotland","status":"publish","type":"post","link":"https:\/\/www.newsbeep.com\/uk\/306772\/","title":{"rendered":"Pioneering Duchenne drug approved for use on NHS Scotland"},"content":{"rendered":"

\n DMD is a rare genetic condition<\/a> which causes progressive muscle weakness, eventually impacting the heart and lungs.\n<\/p>\n

\n It is typically diagnosed in boys between the ages of 3 and 6.\u00a0\n<\/p>\n

\n While there is no cure, advocates for people living with the condition believe that givinostat can slow the progression of the disease, offering families precious time with their children.\n<\/p>\n

\n The medicine, which was approved by the Medicines and Healthcare products Regulatory Agency (MHRA),<\/a> blocks histone deacetylase \u2013 which is an enzyme that is overactive in DMD and inhibits muscle repair.\n<\/p>\n

\n Emily Reuben OBE, the co-founder and chief executive of Duchenne UK<\/a>, said that the approval was a \u2018significant breakthrough\u2019 for people living in Scotland.\n<\/p>\n

\n She remarked: \u201cIn the last year, we\u2019ve gone from having extremely limited treatment options available to now having two approved medicines, offering hope to hundreds of families, and in the case of givinostat, the promise of slowing down the progression of this devastating disease.\u201d\n<\/p>\n

\n Reuben added that the drug must now be approved by NHS<\/a> England and made available across the UK.\n<\/p>\n

\n She added: \u201cWe also continue to explore how patients who\u2019ve already lost their ability to walk can be given the opportunity to potentially benefit from this new treatment.\u201d\n<\/p>\n

\n Families in Scotland formed a united front to demand action in early 2025. They met Health Secretary Neil Gray<\/a>, who pledged to speak with NHS officials to ensure the rollout of the drug.\n<\/p>\n

\n While health boards cited logistical hurdles, they eventually committed to early access programmes and began contacting DMD families to arrange treatment.\n<\/p>\n

\n Reflecting on the drug\u2019s approval, Andy Fletcher, Chief Executive of Muscular Dystrophy UK<\/a> noted: \u201cThis is a landmark moment for families in Scotland. People with muscle wasting conditions, like Duchenne muscular dystrophy, don\u2019t have time to waste.\u00a0\n<\/p>\n

\n \u201cAccessing treatments as early as possible can make a significant difference and with givinostat now accepted for NHS use, families gain access to a treatment that may slow progression and help maintain quality of life.\u00a0\n<\/p>\n

\n \u201cTo see two treatments accepted in Scotland within a year is extraordinary progress for the DMD community. Our focus now is on ensuring families across the rest of the UK can benefit too.\u201d\n<\/p>\n

\n The campaign was shortlisted<\/a> at the Herald Scottish Politician of the Year Awards, with judges focusing on their efforts to place pressure on the Scottish Government<\/a> to provide givinostat to all eligible children.\n<\/p>\n

\n Read more:\n<\/p>\n

\n Katie Endacott, CEO at Action Duchenne<\/a> added: \u201cWe welcome the news that givinostat will now be available through the NHS in Scotland for ambulant boys living with Duchenne.\u00a0\n<\/p>\n

\n \u201cThis decision marks a vital step forward in access to treatments that have the potential to slow the progression of muscle degeneration.”<\/p>\n","protected":false},"excerpt":{"rendered":"DMD is a rare genetic condition which causes progressive muscle weakness, eventually impacting the heart and lungs. It…\n","protected":false},"author":2,"featured_media":306773,"comment_status":"","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[43],"tags":[102,2960,56,54,55],"class_list":{"0":"post-306772","1":"post","2":"type-post","3":"status-publish","4":"format-standard","5":"has-post-thumbnail","7":"category-healthcare","8":"tag-health","9":"tag-healthcare","10":"tag-uk","11":"tag-united-kingdom","12":"tag-unitedkingdom"},"_links":{"self":[{"href":"https:\/\/www.newsbeep.com\/uk\/wp-json\/wp\/v2\/posts\/306772","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.newsbeep.com\/uk\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.newsbeep.com\/uk\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/uk\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/uk\/wp-json\/wp\/v2\/comments?post=306772"}],"version-history":[{"count":0,"href":"https:\/\/www.newsbeep.com\/uk\/wp-json\/wp\/v2\/posts\/306772\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/uk\/wp-json\/wp\/v2\/media\/306773"}],"wp:attachment":[{"href":"https:\/\/www.newsbeep.com\/uk\/wp-json\/wp\/v2\/media?parent=306772"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.newsbeep.com\/uk\/wp-json\/wp\/v2\/categories?post=306772"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.newsbeep.com\/uk\/wp-json\/wp\/v2\/tags?post=306772"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}