{"id":259115,"date":"2025-12-31T02:45:16","date_gmt":"2025-12-31T02:45:16","guid":{"rendered":"https:\/\/www.europesays.com\/ie\/259115\/"},"modified":"2025-12-31T02:45:16","modified_gmt":"2025-12-31T02:45:16","slug":"colchester-boy-given-1-8m-drug-has-new-lease-of-life","status":"publish","type":"post","link":"https:\/\/www.europesays.com\/ie\/259115\/","title":{"rendered":"Colchester boy given \u00a31.8m drug has new lease of life"},"content":{"rendered":"

\"ContributedContributed<\/p>\n

Edward was one of the first children in England to be given the gene therapy Zolgensma through the NHS<\/p>\n

A five-year-old boy who received the world’s most expensive drug as a baby has made “incredible progress” and can walk independently, his mother said.<\/p>\n

Edward, from Colchester, has spinal muscular atrophy (SMA) which means he lacks a protein vital for muscle development.<\/p>\n

He was one of the first children in England to be given the gene therapy Zolgensma,<\/a> which costs \u00a31.79m for the one-off treatment, through the NHS in 2021.<\/p>\n

Mother Megan said Edward was her “pride and joy” and he had achieved milestones she never thought possible.<\/p>\n

\"ContributedContributed<\/p>\n

Edward is full of life and a real character, according to his mum<\/p>\n

About 65 babies are born with SMA<\/a> in England each year. It causes muscle weakness and affects movement and breathing, meaning most babies do not live past the age of two without intervention.<\/p>\n

Megan said Edward had gone from being lethargic as a baby to a cheeky, playful boy, who was “full of life” and “a real character”.<\/p>\n

He might need to use a wheelchair for the rest of his life, but she added: “It does not matter, as long as he is happy. We are so proud of him.”<\/p>\n

\"ContributedContributed<\/p>\n

Edward can now walk 20 to 30 steps independently<\/p>\n

“Edward had to have a double hip replacement in October and he’s only just getting back on his feet, but in general he is doing so well.<\/p>\n

“He is learning to swim, he can float on his own, which is really hard for children with SMA because they don’t have natural buoyancy.<\/p>\n

“This summer, he jumped off a boat into the sea and he went on a jet ski. He’s a very sweet, loveable little guy.”<\/p>\n

Edward has just started school, where he has made lots of friends, and “does everything an ordinary five-year-old boy does”.<\/p>\n

“We just didn’t think that was possible. We didn’t know what quality of life he would have,” she said.<\/p>\n

Various doctors and medical professionals visited him whenever he was in hospital, even when they were not treating him, because they were amazed by his progress, she added.<\/p>\n

“They want to see first-hand what gene therapy has done for him.”<\/p>\n

\"Megan<\/p>\n

Edward received the gene therapy four years ago and it has transformed his life<\/p>\n

The family moved to London so Edward can have physiotherapy up to five times a week.<\/p>\n

Ms Willis gave up her job in event management to care full-time for Edward. <\/p>\n

It was not certain he would receive the drug on the NHS, so she started a fundraising campaign, and has used the money to pay for specialist physio and equipment, to which she credits his progress.<\/p>\n

“We raised \u00a3170,000 over five years but that money has nearly gone. It’s been put to a lot of good use,” she said.<\/p>\n

“It has saved us as a family, not having to worry about the money. We’re fundraising again now because all of the progress he has made has been due to private care.”<\/p>\n

\"ContributedContributed<\/p>\n

Edward has made huge progress due to the private physiotherapy the family has been able to fund via a fundraising page<\/p>\n

Zolgensma is thought to be the most expensive drug in the world, though NHS England said it had negotiated an undisclosed discount on its \u00a31.79m list price.<\/p>\n

Edward, who was diagnosed at two months old, was receiving another drug called Spinraza, which involves regular spinal regular injections for life, compared to a one-off injection of Zolgensma.<\/p>\n

Because it is such a new drug, long-term outcomes are not known, but Megan said she believed this generation of babies with SMA would be the first to reach adulthood.<\/p>\n

\"ContributedContributed<\/p>\n

Edward started school this year and had made lots of friends, his mum said<\/p>\n

Prof James Palmer, medical director for specialised commissioning at NHS England, said: “It is a huge pleasure to see the remarkable benefits that this innovative gene therapy has provided for Edward since he was treated four years ago.<\/p>\n

“Edward is one of more than 150 children with SMA to benefit from this one-shot treatment which has had a huge impact on their lives, and I’m optimistic that many more conditions like SMA will also become treatable over the coming years as medical advances continue at pace.”<\/p>\n","protected":false},"excerpt":{"rendered":"Contributed Edward was one of the first children in England to be given the gene therapy Zolgensma through…\n","protected":false},"author":2,"featured_media":259116,"comment_status":"","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[74],"tags":[18,19,17,82],"class_list":{"0":"post-259115","1":"post","2":"type-post","3":"status-publish","4":"format-standard","5":"has-post-thumbnail","7":"category-technology","8":"tag-eire","9":"tag-ie","10":"tag-ireland","11":"tag-technology"},"share_on_mastodon":{"url":"https:\/\/pubeurope.com\/@ie\/115811891975374888","error":""},"_links":{"self":[{"href":"https:\/\/www.europesays.com\/ie\/wp-json\/wp\/v2\/posts\/259115","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.europesays.com\/ie\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.europesays.com\/ie\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.europesays.com\/ie\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/www.europesays.com\/ie\/wp-json\/wp\/v2\/comments?post=259115"}],"version-history":[{"count":0,"href":"https:\/\/www.europesays.com\/ie\/wp-json\/wp\/v2\/posts\/259115\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.europesays.com\/ie\/wp-json\/wp\/v2\/media\/259116"}],"wp:attachment":[{"href":"https:\/\/www.europesays.com\/ie\/wp-json\/wp\/v2\/media?parent=259115"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.europesays.com\/ie\/wp-json\/wp\/v2\/categories?post=259115"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.europesays.com\/ie\/wp-json\/wp\/v2\/tags?post=259115"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}