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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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
Manchester’s Club de Padel claps back at ’embarrassing’ copycat ASOS product
Daisy Jackson
A local Manchester business has sarcastically pointed out the glaring similarities between a new t-shirt on ASOS and one of its own designs.
Club de Padel has a range of retro-inspired merch with UN:IK, a small independent streetwear brand also based here in Manchester.
Its collection of t-shirts, hoodies, tracksuits, hats and bags often feature a design of an illustrated padel ball (which to the untrained eye is basically a tennis ball) in motion.
But this week a t-shirt appeared on ASOS that also featured an illustrated padel ball in motion, just in a slightly different colour palette.
The ASOS version, which has now been removed from sale, also undercut Club de Padel’s price by £6.
Since Club de Padel took the ’embarrassing’ copycat product public on their Instagram page, ASOS has launched an investigation and taken it off their website.
The sports venture, based at Deansgate Square and the only padel club in Manchester, wrote: “Love the new designs ASOS, kinda reminds us of something though…
The ASOS t-shirt that Club de Padel has said copies their merch. Credit: ASOSClub de Padel’s t-shirt created by Manchester brand UN:IK. Credit: Club de Padel
“Ah. Well that’s embarrassing… We’ve got a new line coming soon with UN:IK Clothing you’re just gonna love ASOS.
“In the meantime we made this for you @asos, save your design team a job.”
An ASOS spokesperson said: “We take intellectual property issues very seriously and have removed this product from sale while we investigate further.”
Of the Club de Padel and UN:IK collaboration, aka the real deal, UN:IK says it has ‘served up a staple for us, retro inspired leisure pieces with Manchester’s first ever padel club’.
Club de Padel in Manchester has gone public to poke fun at ASOS
They wrote: “Inspired by owners after a trip to Spain, Club de Padel has hit the city with a wave and the lifestyle brand we have created with them reinforces the fastest growing sport across Europe.
“Our classic vintage washes and oversized fits, working with independent designs as always on this local independent partnership. Don’t be afraid to the sport, we’ll guarantee you love it as much as these clothes.”
Manchester Remembers release unbelievable new kits featuring iconic Manc landmarks for 2024 charity match
Danny Jones
The Manchester Remembers Foundation have just released the official kit designs for this year’s upcoming charity football match and they’re utterly amazing.
The 2024 Manchester Remembers charity game is fast approaching, with this year’s edition set to take place in July and the excitement is really starting to build – especially with kits as eye-catching as these.
Roping in help from local media and production company Badger and Coombes, as well as getting Lionesses legend Sue Smith involved in the kit launch, the brand new Legends and Celebs kits have finally been revealed in extremely fine fashion.
Lovingly made once again by Oldham-based kit manufacturers, Hope and Glory, and with Salfordian fashion brand Marc Darcy Menswear serving as the main sponsor, the new shirt designs feature incredibly detailed drawings of recognisable locations around Manchester. Just look at them:
— The McrRemembers Foundation™️⚽️🐝 (@McrRemembers) April 19, 2024
The 2024 Manchester Remembers kits have officially been unveiled.
As you can see, iconic Manc landmarks like Old Trafford and the Etihad Stadium take pride of place in the semi-opaque sublimated pattern on both the white and black versions of the kit.
Other noticeable buildings include Manchester Cathedral, the Central Library, Manchester Central (formerly the GMEX) and even a nice little nod to the Metrolink and Bee Network, just to name a few.
The incredibly detailed design pays homage to the city we all know and love and is a big step up from the already very well-received kits from last year’s charity match.
Once again, all the proceeds from the shirt sales go straight back into the Manchester Remembers Foundation and its partnered charities, which raise money for the families of those sadly lost in the 2017 Manchester Arena attack.
Celebs stripLegends kitYou can pre-order the shirts ahead of their release in May HERE and they’re very affordable compared to most footy kits these days.
With a whole host of exciting names taking part in the 2024 edition of the charitable community-driven event, including ex-Manchester United stars Antonia Valencia and Danny Simpson, local celebs like soap star Adam Thomas, YouTuber ‘Angry Ginge’ and many more, it’s going to be a momentous day.
This year marks just the third time the game has been held but the amazing people attached to this non-profit, not to mention the thousands that have turned up to see the wholesome fixture, have already helped raise over £117k for the associated charities. Unbelievable stuff.
Returning to Oldham Athletic‘s Boundary Park for the second year running, tickets for the game on Sunday, 28 July are already available and priced at only a fiver for under 16s and just £10 for adults, it’s one of the most uplifting and affordable afternoons of live sport you’ll find the region.
If you’ve not heard about this truly inspiring annual match until now, you can find out more HERE.