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
Beloved plant-based independent street food business Herbivorous is set to close imminently
Danny Jones
We hate writing this, but we have news of yet another local hospitality closure as beloved Manchester-born and pioneering plant-based brand Herbivorous has confirmed they’ll soon be calling it a day.
If you can hear us sighing through the screen, apologies – we’re just getting really fed up of saying farewell to our favourites, so god knows how rubbish those involved feel.
As you can see, the team behind the multi-location vegan street food specialists began by writing: “It’s a long one so stay with me… It seems you can’t open social media at the moment without reading about another hospitality business closing down.
“Throughout 8 years of Herbivorous years we’ve come up against many challenges from Covid 5 years ago to watching all the big vegan brands from London and beyond come to Manchester and largely disappear again to the closure of Hatch our busiest site, but with continually increasing costs its becoming really difficult to maintain a thriving business.”
Herbivorous started out life back in 2016 as a cult hit at the old outdoor food, drink and entertainment hub centring around a ‘container village’, the first of its kind in the city and soon to reopen under new owners and a new name.
Since then, co-founders Robyn and Damian have gone on to expand not just to one permanent site over in Withington but with outposts in Sheffield and York, having gained a strong reputation with stalls and pop-ups all over.
Their hospitality story might not have the ending we personally would hope for, but it’s been a brilliant one for so many foodie fans – us lot included.
Nevertheless, they go on to add in the emotional statement that external pressures such as a recent bereavement and their own ever-growing family have also contributed to the decision which, despite being an understandably hard one, “feel like the right time.”
“We are so incredibly proud of how far we’ve come from spending long days slinging duck wraps from our green vintage horse box at festivals to three Herbi locations across the North of England”, they continue, “and with that we’ve also met so many fantastic people!”
“Whether you worked for us at a few festivals over [the] summer or for years at our restaurant, we just wanted to say and massive thank you! The memories will stay with us forever and, of course, how can we not mention our fabulous customers!
“Thank you all so much for choosing Herbivorous over the years. Those of you who had our food at Festivals and then found us at one of our permanent locations, those of you who came back week after week to Hatch, Spark, Withington and beyond.”
They signed off by detailing their final business days, with their Sheffield Kommune spot having already shut for refurbishment; meanwhile, their spot in SPARK York will be wrapping up on Saturday, 19 April.
As for their flagship brick-and-mortar venue here in Greater Manchester, Herbivorous Withington will be shutting on Friday, 25 April.
Once again, there’s nothing we can say other than thank you for years of delicious food, that we’re guttted to see you go, and that everyone at The Manc Group wishes you the best whatever comes next.
AJ Tracey is playing a VERY intimate gig in Manchester next month
Thomas Melia
London rapper and respected grime artist AJ Tracey is ‘live and direct’ once again, announcing a whole host of UK dates, one of which is a rather intimate gig right here in Manchester.
It’s official: one of UK rap’s leading gents is getting back on the road and he’s showing a ‘Little More Love’ to lots of cities, including Manchester.
In a post on the UK star’s socials, AJ Tracey trades his music persona for that of a sports pundit as he announces the dates for his upcoming tour while impersonating an F1 commentator.
Anyone looking to attend Tracey’s ‘Not Even A Tour’ can catch the star up and down the country on his over 20-date tour with a stop in Manchester as soon as this month.
The rapper has reached phenomenal heights since dropping the unforgettable ‘Ladbroke Grove’ in 2019 and is heading out across the nation to make sure everyone knows about it.
Get ready to spit bars left and right as this rapper’s discography is stacked with serious tunes like ‘Dinner Guest’, ‘West Ten’, ‘Thiago Silva’ and more.
More recently, he collaborated with Walsall’s finest Jorja Smith for a flirty garage beat also known as ‘Crush’ with the video being the two artists talking it out in a good old fashioned caff.
The latest collaboration with Smith marks the start of a new era for Mr. Live and direct as it fell in line with the announcement of his third studio album.
Titled Don’t Die Before You’re Dead, we’re sure he’ll be “building a vibe” wherever he goes and probably previewing a few exclusive tracks from his upcoming project too.
This gig may come as quite a surprise for any hardcore fans of the ‘Ladbroke Grove’ star as the venue in which he’s performing is definitely intimate only, holding just over 250 guests.
This rap giant is bringing his unbelievable beats to none other than the legendary small-cap city centre live music venue, The Deaf Institute, at the end of this month.
It’s safe to say you won’t get many more chances, if any, to see him in such a tight-knit space again.
AJ Tracey is bringing ‘Not Even A Tour’ to Deaf Institute in Manchester on 30 April, with tickets on sale Friday 4 April from 12 noon.