A toddler with a rare fatal genetic condition has become the first child in the UK to receive a lifesaving gene therapy treatment on the NHS.
19-month-old Teddi has been treated with a revolutionary gene therapy known by its brand name Libmeldy – which has a list price of £2.8 million, and was the most expensive drug in the world when the NHS negotiated a “significant confidential discount” last year to make the treatment available to patients.
Despite this discount though, it still remains the most expensive drug licensed in Europe – with Teddi being the first child to be given the treatment.
The Northumberland toddler has a rare and fatal genetic disease called metachromatic leukodystrophy (MLD), and was sadly diagnosed with the condition along with her three-year-old older sister Nala in April last year.
MLD causes severe damage to the affected child’s nervous system and organs, and devastatingly results in a life expectancy of between just five and eight years.
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Parents thank Manchester doctors as toddler with fatal disease is saved by world’s most expensive drug / Credit: NHS England
Teddi was treated by a specialist service being delivered at Royal Manchester Children’s Hospital (RMCH) – which is collaboration with Manchester’s Centre for Genomic Medicine at Saint Mary’s Hospital.
The centre in Manchester is one of just five European sites administering the treatment – which works by removing the child’s stem cells and replacing the faulty gene that causes MLD, before re-injecting the treated cells into the patient – and is the only site in the UK.
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NHS England explains that the most common form of MLD usually develops in babies younger than 30 months, and due to the development of a crucial enzyme that leads to a build-up of fats that then destroy the protective layers around the child’s nerves, it can lead to loss of sight, speech, and hearing, as well as difficulty moving, brain impairment, seizures, and eventually death in childhood.
Ally and Jake have two beautiful girls, Nala and Teddi. In April 2022, both girls were diagnosed with metachromatic leukodystrophy (MLD), a genetic disease resulting in a life expectancy of five to eight years.
Teddi was the first person in the UK to receive the Libmeldy treatment outside of a clinical trial – which began when she was 12 months old.
Treatment first began with the removal of stem cells at the end of June, which were then treated before the transplant took place in August, and Teddi was discharged back to her home in Northumberland in October to become “a happy and healthy toddler” now showing no signs of the devastating disease she was born with.
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Sadly though, Teddi’s older sister Nala was not eligible for the treatment, as the clinical guidance requires the gene treatment to be administered before the irreversible damage caused by the disease progresses too far, NHS England explained.
Teddi and older sister Nala were both diagnosed with MLD in April 2022 / Credit: NHS England
Teddi’s mum, Ally Shaw, has praised Manchester doctors for saving her little girl.
“In April last year, our world was turned upside down when not one, but both of our daughters were diagnosed with MLD,” Ally said.
“Being told our first daughter, Nala, wasn’t eligible for any treatment, would continue to lose all functions, and die extremely young was the most heart-breaking and hardest thing to come to terms with. However, amongst the pain, was hope for our younger daughter, Teddi. We were told that a new gene therapy treatment had, luckily, recently been made available on the NHS.
“We are extremely privileged that Teddi is the first child to receive this on the NHS and grateful that she has the opportunity to lead a long and hopefully normal life.
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“Without this treatment, we would be facing both our children being taken away.
“We would like to say a huge thank you to our specialists, doctors and nurses and all the staff at Royal Manchester Children’s Hospital who have been fantastic in caring not just for Teddi, but us as a family.”
19-month-old Teddi has been treated with a revolutionary gene therapy / Credit: NHS England
NHS England has called this “a huge moment of hope” for parents and their babies who are born with this devastating inherited disorder.
Speaking on the success of the treatment, NHS chief executive Amanda Pritchard said: “Thanks to advancements in gene therapies, and the commercial ability of the NHS to strike deals for cutting-edge drugs and then deliver them through our phenomenally skilled specialist staff, children born with this condition now have the opportunity to lead normal, healthy lives.”
Previous treatment options for MLD on the NHS were limited to managing symptoms and providing supportive care.
But now, the new Libmeldy treatment will be available to babies and young children with no clinical signs or symptoms, as well as those with early symptoms of the condition, so long as they can still walk independently and with no evidence of cognitive decline.
Featured Image – NHS England
Manchester
Science and Industry Museum reveals CGIs of iconic Power Hall that’s been closed for years
Daisy Jackson
One of the Science and Industry Museum’s most iconic attractions will reopen this summer – and the beloved museum has revealed a sneak peak of how things will look.
The museum has shared CGIs of the reimagined Power Hall: The Law Family Gallery, where visitors will be able to explore a lively working gallery full of engines and rail vehicles.
The popular part of the museum closed for urgent repairs in 2019 and has since been undergoing conservation work and a new look created by award-winning designers Studio MUTT.
The sounds, smells and sights of Manchester’s past and present industry will be brought to the Power Hall when it reopens.
Visitors will soon again be able to rediscover objects and learn the stories behind those who powered Manchester’s industry.
There’ll be three main themes within the Power Hall – Making More, which will explore how engines helped people make more, faster, with steam engines installed at factories and mills.
Then there’ll be Powering Lives, which will examine how engines power the electricity network we all plug into every day.
And also Connecting Places, which looks at how locomotives have connected communities around the world, starting at the site of the museum which sparked a transport revolution in 1830.
Science and Industry Museum reveals CGIs of iconic Power Hall that’s been closed for years
Kate Chatfield, Interpretation and Content Manager at the Science and Industry Museum, said: “Power Hall: The Law Family Gallery will be a must-see Manchester experience – a living gallery that showcases a unique collection of historic 19th and early 20th century working engines to tell the story of Manchester as an epicentre for the engine-driven ideas and industry that shaped the world as we know it today.
“Our most iconic objects will be available to explore like never before as we bring to life the people behind the power through stories of the engineers, makers and technicians who use their skills and senses to create and care for engines, both today and in the past.”
The work on the Power Hall is part of a multi-million-pound regeneration project across the Science and Industry Museum, which is conserving and reimagining these historic buildings.
The Power Hall has been future-proofed in the six years it’s been closed to the public, including urgent roof and timber repairs.
Further information about what to see and do and the Power Hall’s opening date will be announced in the coming weeks. Sign up to the museum’s mailing list to be among the first to hear more.
Power Hall: The Law Family Gallery project has been made possible with support from The Law Family Charitable Foundation, the Wolfson Foundation, The National Lottery Heritage Fund, The Headley Trust, Royal Commission for the Exhibition of 1851, Atmos International, The Beaverbrooks Charitable Trust, The Zochonis Charitable Trust and other donors who choose to remain anonymous.
Special thanks to the Department for Culture, Media and Sport for their significant contribution to the gallery, and to the Department for Energy Security and Net Zero for funding the decarbonisation of the Power Hall through the Public Sector Decarbonisation Scheme, delivered by Salix Finance.
Featured image: Supplied
Manchester
Greater Manchester cleans up at first-ever Festival of Women’s Football Awards
Danny Jones
This week marked the inaugural Festival of Women’s Football Awards (FWFA), which not only saw a whole host of female talent from up and down the English pyramid celebrated, but Greater Manchester in particular picked up a number of accolades.
Of course, we all know that 0161 is the true home of football, with Manchester’s rich sporting heritage known worldwide, but it’s always nice to see our region recognised for its continued contribution to the beautiful game.
That reputation goes double for the women’s game too, as the 2025 FWFA saw a trio of local clubs and a trio of different players winning across multiple categories.
Starting with the blue side of Manchester, Manchester City Women star Yui Hasegawa picked up Player of the Year from a strong shortlist of midfielders.
Joining the club back in the summer of 2022, Hasegawa famously scored on her debut and was named in the PFA Team of the Year that season.
Playing in 92 of the 96 games since her arrival, she is widely considered not just one of the best midfielders in the Women’s Super League (WSL) but the world.
As for the red half of the city, not only did Phallon Tullis-Joyce – who has shone since filling the very big boots of Mary Earps – pick up the award for the best shotstopper, but centre-back Maya Le Tissier was named both Defender and Young Player of the Year.
Latics Women only launched their squad this season but have already amassed a passionate following, hence the award, and also won the Lancashire Women’s County League Championship at the first time of asking.
Congratulations to all the Greater Mancunian footballing organisations and sports personalities recognised at this year’s Festival of Women’s Football Awards – here’s hoping for even more in 2026!
🏆 We were delighted to attend this evening’s Festival of Women’s Football Awards in London…
It was an honour to be shortlisted for eight national awards, and take home the ‘Best Fan Experience’ prize! 💙#wafc 🔵⚪️ pic.twitter.com/OoeN92Vxwv