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
Travel warning issued as Manchester braces for busiest day of the year so far
Daisy Jackson
A ‘plan ahead’ travel warning has been issued for this weekend as the Manchester Marathon brings the busiest day of the year so far on the tram network.
Tens of thousands of people will hit the streets to take part in the race itself, with thousands more cheering them on from the sidelines.
This year’s Manchester Marathon route takes 36,000 runners through Sale, Stretford and Timperley before finishing up at a new finish line on Oxford Road – and there’ll be some big changes to your usual travel routes to accommodate this.
TfGM has said that Bee Network trams will be very busy before, during and after the 26.2 mile race, especially in Manchester and Trafford, with a number of road closures in place too.
Taking place on Sunday 27 April, the Manchester Marathon will coincide with Manchester’s popular St George’s Day parade which starts and ends across town around Oldham Road and Great Ancoats Street.
People are being encouraged to plan their journeys carefully this weekend, as it’s expected to be the busiest day of the year on the Bee Network trams.
The Manchester Marathon last year set a new personal best for the tram network, with 175,000 tram journeys racked up – the highest ever recorded on a single Sunday.
Trams will be the best choice to get around the Manchester Marathon course with key stops along the route including Sale, Timperley and Chorlton.
Many road closures will also be in place but at different times of day, so make sure you check HERE for the latest.
This will impact bus services, which will be on diversion from 8am until the end of service on Sunday, with traffic and congestion expected on surrounding roads.
Runners heading to the start line will only be able to access the athletes’ village from Old Trafford and Trafford Bar tram stops up until 9am. Participants arriving after 9am should get off at Wharfside or Exchange Quay instead.
Metrolink is promising to deploy as many double trams as possible across the network, with additional services between Piccadilly and Timperley.
As for other modes of transport, those who wish to drive should make us of Park and Ride facilities where possible; and those on the train should note that Salford Central remains closed with bus replacements in place.
Danny Vaughan, Chief Network Director at TfGM said: “The Manchester Marathon is a highlight of the Manchester city centre calendar and our job is to make sure everyone can get to and from the event as smoothly as possible.
“We want everyone to have a great day – whether they are taking on the run themselves or cheering on a friend or loved one – and would urge people to plan their journey in advance, leave plenty of time and check the Bee Network app or website for the most up-to-date information.
“The start and finish lines will be especially busy so if you are coming to watch you may be better off catching the tram to Sale, Timperley or Chorlton, grabbing a coffee and claiming afront row seat to cheer runners on.
“Using public transport is the best way to avoid congestion and delays – and with Tap and Go contactless payments it’s never been easier or more seamless to get around Greater Manchester by bus and tram.”
Passengers can now travel seamlessly between bus and tram and pay for their journey by tapping on when boarding a bus without the need for a ticket, making it the quickest and easiest way to pay for travel.
People travelling on Bee Network buses on Marathon Day can simply tap in and pay £5 for the day on as many buses as they need and just £4.90 on the tram. You can use all Bee Network buses as much as you want all day from just £5.40*.
Kirsty Wilde, Operations Director, from Manchester Marathon organisers A.S.O. UK, said: “The TfGM tram service is an invaluable part of our event, which allows thousands of participants and supporters to navigate around the city. With a record-breaking 36,000 participants set to take part on Sunday 27th April – and more exciting activities taking place over the weekend as a whole – we’re anticipating a rise in tram journeys, which will subsequently bring economic benefits to local vendors, the hospitality sector, and retail businesses.
“We’d like to thank Transport for Greater Manchester for its continued hard work helping to facilitate what will be the largest event yet, and would also like to say a big thank you to the residents and businesses across Greater Manchester for their understanding and support.”
Kobbie Mainoo shouts out more of his favourite food spots in Manchester
Danny Jones
Man United star Kobbie Mainoo is back putting people onto his favourite places to eat in Manchester, and judging by his picks, he doesn’t just know football – he knows his food spots too.
The Stockport-born footballer turned 20 years old this month, and as well as celebrating leaving his teens with that impressive goal in the comeback against Lyon, the Manchester United midfielder was met with a birthday cake by his club, along with a fun little game of 20 questions.
Sharing the short but insightful Q&A, in which the number 37 dove into his favourite parts of matchdays, the best birthday gift he’s ever received, as well as the tried and tested dream dinner guest lineup.
Naming a very sporty trio of Mike Tyson, Michael Jordan and Jon Jones, the only logical next step was to ask him where his favourite places to eat around Manchester were, to which he answered…
That’s right, spotlighting a personal favourite of ours, the first Manc venue on his list was Pho Cue, the authentic family-run Vietnamese spot known for great value, top-quality street food such as their big hitter bánh mìs, as well as big queues every lunchtime.
If you don’t know already, then get to know.
As you can see, the second place he mentioned with the booked-up-till-kingdom come Onda Pasta Bar, which went viral for its ‘Tiramisu drawer’ last year and opened their first proper restaurant back in June.
Since then, it’s fair to say it’s more popular than ever and you’re lucky to get a table; if you, however, you’ll be treated to some of the most luxurious and indulgent pasta not just in Manchester but probably anywhere in the country, if you ask us.
These latest shout-outs come after Mainoo famously put Salford’s culinary kings, Burgerism, on the nationwide map after name-dropping them in his British GQ interview in November 2024.
You can watch the full thing HERE, but you already know the biggest takeaway from that chat, quite literally: the boy knows his burgers.
Kobbie is actually just one of several famous faces to have proclaimed Burgerism as the best in the business, at least in Greater Manchester of late.
Not only have British influencers Ollie Ball and Jacob Pasquill now become seemingly regular presenters on their social media, but online content creator turned boxer and WWE star Logan Paul also visited the beloved burger joint and reviewed it during a recent visit to the UK.
We’ll confess we’re more likely to take the local lad’s word on board over the bloke who used to make Vines, but it’s just yet more proof that the Manc food scene is top tier.
Come on, just look at the state of these selections:
Not a bad little spread there, Kobb… (Credit: The Manc Eats)
We’re not sure how much of this stuff Mainoo is allowed on the diet sheet, but we can at least confirm that Kobbie knows his Manchester food, and as far as favourites go, we might just stand a chance of becoming best friends yet.
Happy birthday to a young man with a very bright future and hopefully a very active metabolism.