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.
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.
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.
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.
“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
The bizarre explanation behind Manchester’s new mini ‘green space’ – that’s completely closed off
Danny Jones
One thing you always hear people who live in central Manchester calling out for is green space, but when a new patch of grass does arrive, you don’t exactly expect it to be completely inaccessible.
Just a quick FYI to those of you passionate about supporting what little nature there is left in our city centre: brace yourselves, as this one is rather annoying…
For anyone who’s walked down Whitworth Street West past the once lively Locks, or even got off at Deansgate–Castlefield tram stop and come down the glass stairs closest to the Albion Bridge, you may have noticed a rogue patch of grass surrounded by black fencing next to the City Road Inn pub.
It could be considered a nice little lawn were it not completely fenced off, but the question is, why has it popped up there? Well, local lad Curtis Rothwell has recently debunked the whole frustrating mess for us in a helpful video.
Yes, as explained by the Pre-Construction Manager who works for local utilities contractor Zenit Infrastructure Solutions Limited, the somewhat laughable but disheartening gated green space on Whitworth Street is essentially a feeble attempt at adhering to permission laws.
As detailed by Rothwell, the plot bought for a sizeable sum last year (only the original developers didn’t have the funding to actually pull off their initial plans) is now set to become student accommodation.
Currently, real estate firm Tri7 – which has now been fully folded into the parent group known as Fusion, as previous part-owners already – still has a hold on the land for now.
However, after being ordered by Manchester City Council to tidy up the area in the meantime, as they await planning permission to be fully approved, they decided to lay these new rolls of turf there.
Ok, so while the notion of the Council ordering a property company to make urban spaces look pretty and the idea of introducing more grassy zones into the city sound good on paper, as you can see, the real-life result doesn’t quite match up.
Not only does this feel like a bare minimum effort to appease the local authorities ahead of erecting yet another skyscraper, but more importantly, it feels like sheer nonchalant neglect and/or plain dismissal of those Manc residents crying out for more grass, nature and a break from the concrete jungle.
The fact that the rare green space is totally blocked by tall, almost authoritarian-feeling fences and sealed off by a big, black gate comes across like a casual two fingers to both the Council and those living in the city centre.
Speaking to The Manc, Curt commented: “It’s positive the council can take charge of how our empty spaces look after demolitions. In this case, maybe the demolition was carried out too early, which happens, but when it does, greenery is the best look they can give to local people.”
You can see the latest update on the planning permission HERE, and for now, if you’re looking for green spaces in Manchester, you’ll find them more frequently beyond the outskirts and heading into the boroughs; this one is strictly off limits. Find a whole bunch of ones that aren’t down below:
Featured Images — The Manc Group/Curtis Rothwell (via LinkedIn)
Manchester
There’s a tiny Japanese restaurant inside a Manchester cafe serving matcha afternoon tea
Daisy Jackson
There’s a tiny little Japanese restaurant space in Manchester tucked inside a coffee shop – and they’ve just started serving matcha afternoon teas.
Niwa Yakitori is a brilliant addition to the city centre – by day, it’s sharing the space at North Star Piccadilly and operating as a matcha cafe.
And by night, it transforms the space into a cosy Japanese restaurant, complete with paper lanterns, bamboo plants, and neon signs.
While the word-of-mouth restaurant space is a real rising star thanks to its menu of freshly barbecued chicken and seafood, as well as delicious flights of sake, it’s the new matcha afternoon tea that we popped in for this time around.
Each table is presented with a tiered tower of treats infused with the popular Japanese green tea, and this is some world-class baking that’s going on at Niwa Yakitori.
Let’s break it down, tier by tier, to give you an idea of what you can get.
Up first is a Japanese egg mayo sandwich, made with fluffy shokupan bread and Kewpie mayo, then sprinkled in Furikake for a little bit of salt and texture.
The Japanese egg mayo sandoStrawberry sando with matcha creamThe half-and-half cookieMatcha basque cheesecakeScones with matcha cream and yuzu jamJapanese afternoon tea in Manchester
Also joining the sandwich line-up are strawberry sandos (yes, the ones that M&S had a go at this summer), made with a matcha cream and more of that pillow-soft shokupan.
Moving on up, and what afternoon tea would be complete without some scones? These ones come with a matcha cream and a yuzu and chilli jam. Divine.
Then the very top tier has a gang of miniature treats, including an adorable teddy bear-shaped matcha chocolate truffle; a matcha muffin; and a matcha-infused madeleine.
A real star is the chunky cookie, a clever half-half yin-yang of matcha and white chocolate, vs vanilla and dark chocolate.
Niwa Yakitori is a tiny Japanese restaurant in ManchesterJapanese afternoon tea in ManchesterMatcha and hojita at Niwa Yakitori
And they show off their baking prowess with a slice of matcha basque cheesecake, with three layers and a scoop of strawberry ice cream on top.
All washed down with ceremonial-grade matcha or hojicha lattes, which can be made table-side in a traditional tea ceremony.
The team at Niwa Yakitori are hoping this afternoon tea will fill crucial tables in their tiny space at a time where hospitality is being crippled with rising costs.
So please, for the love of all that is matcha, get this lovely Manchester Japanese spot on your list to visit.