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
Popular night-time indie shopping market returns to Manchester tomorrow
Emily Sergeant
A unique indie shopping market is returning to Manchester, and you’ll be able to shop from 50 local small businesses all under one roof.
In case you hadn’t heard, the hugely-popular Night Market UK is back by popular demand, and it will be stopping off in our city centre for one night only tomorrow as part of the current leg of its tour across the country.
Returning to Manchester, but this time at a new location down at Fairfield Social Club in the Green Quarter, the unique night-time shopping experience will be showcasing more than 50 local small businesses – with everything from bespoke fashion items and beautifully-crafted homeware, to candles, artwork, silverware, and more on offer.
Brutal Fashion, Lost in Music, Urban Botany, and Dapper Alice are just a handful of the indie traders you’ll be able to shop from.
With event organisers promising there’ll be “a stall for everyone” to browse on the night, some of the other traders in attendance, include artisan producers, artists and bakers, as well as Fairfield Social Club’s resident street food purveyors Isit Kitchen, and pizza pros Killa Carbs.
DJ Clara B will also be taking over the club’s sound system too, and will be blasting tunes to set the scene all night long.
From bespoke fashion items and silverware courtesy of Brutal Fashion, to dazzling musical art prints from Lost in Music, and sculptural candles from Urban Botany to beautifully crafted homeware by Dapper Alice, there will be a stall for everyone on the night.
A popular night-time indie shopping market is returning to Manchester tomorrow / Credit: The Night Market UK | Kunal Mahesh Tewari (via The Night Market UK on Facebook)
The Night Market UK will open at Fairfield Social Club from 6pm tomorrow (Friday 26 July), and will run right through until 11pm.
Tickets are now on sale from just £4 each, and can be purchased in hourly entry timeslots – which organisers say “keeps the atmosphere just right” and ensures there’s enough space for everyone to be able to stay as little or as long as they’d like.
You can buy on the door or grab your tickets in advance here.
Featured Image – Supplied
Manchester
Beloved Manc butty shop Bada Bing set for a sensational return to the city centre
Danny Jones
Sandwich lovers rejoice because one of the finest butty shops to ever grace Manchester is returning: that’s right, Bada Bing is bada-back!
The Sopranos-inspired deli and sandwich shop that took its name from one of the central locations featured in the iconic US drama (yes, the strip club), was a huge success when it first opened in Manchester a few short years ago, so it was a huge blow when it closed in February 2022.
Starting out by serving sandwiches out of a window at The B Lounge pub on Paton Street near Piccadilly, before moving to a small kitchen on Radium Street and eventually setting up their stall inside Ancoats General Store, they would regularly have lines around the block every lunchtime.
These Italian-American sarnies were so popular you’d often struggle to get your claws on one – and believe us, they really are a two-handed task – as they’d sell out on what felt like most days. But now, whether you were a regular or someone who missed out, there is hope once again:
Announcing their sensational return to a new site over in the Northern Quarter, which will now mark the fourth premises they’ve popped up at, Bada Bing is back with a bang and, as you can, they dropped the news with one of the best reveal videos we’ve ever seen.
If you know, you know…
Set to take over 125 Oldham Street, owners Sam Gormally and Meg Lingenfelter haven’t yet graced us with an opening date but the new unit should hopefully be open sooner rather than later.
The duo, who previously worked at fellow NQ favourite Another Heart to Feed, came up with the concept during lockdown and it didn’t take long for the idea to take off, nor for them to earn their spot amongst the very best sandwich places in Manchester.
Seriously, these things were so big and unwieldy (in the best way possible) that they even used to come with eating instructions: both hands and the trademark Tony Soprano hunch recommended, though the slightly stained wife-beater, boxer shorts and open dressing gown look is optional.
From slices of provolone cheese, all the thinly sliced Italian meats you could think of and the closest thing to actual ‘gabagool‘ as you’ll find in Greater Manchester, the menu was fitting of being served up to the iconic characters that once sat outside Satriale’s and a big approving grin from the man himself.
Simply put, we cannot wait and we will certainly keep you posted when we find out exactly when Bada Bing confirms their official reopening date.