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 AO Arena scoops up Take That gigs as Keane’s Co-op Live gig is cancelled in ongoing venue saga
Daisy Jackson
Manchester’s existing arena, the AO Arena, has stepped in to host more concerts that have been postponed by Co-op Live, with yet another act being rescheduled too.
First it was announced earlier today that A Boogie Wit Da Hoodie would be moving to the AO Arena, and now Take That’s five-night run that was set for the Co-op Live this May has also had a venue change.
Their two June dates at the new arena are said to be unaffected.
The Co-op Live‘s official opening has been hit with several delays, with events like Peter Kay’s double bill, Olivia Rodrigo and The Black Keys all already postponed over the last week or so.
Issuing a statement after rescuing multiple Take That shows, AO Arena general manager Jen Mitchell, said: “It wouldn’t be a Take That tour without a stop-off in Manchester, so it’s great to welcome the band and their fans back to AO Arena for five nights, in the heart of this wonderful city.”
The venue has naturally welcomed the sudden flood of extra names being added to its already strong lineup for 2024, doing its best to minimise inconvenience for gig-goers who have made travel arrangements, booked hotels etc.
As for the group themselves, they also responded to their tens of thousands of fans expecting to see them from this weekend onwards.
Confessing that they’ve had to make ‘the difficult decision’ to switch venues and although ‘it’s not one [they’ve taken lightly], they wanted to give fans as much notice as possible’, adding: “We thank you for your understanding and we’ll see you all soon”.
Meanwhile, Noughties favourites Keane have sadly had to shelve their Manchester show for the foreseeable following the venue’s continuing ‘technical issues’.
Announcing the news on their social pages on Thursday evening, they admitted that they were ‘absolutely gutted’ but that the decision was ‘entirely beyond our control’.
We’re absolutely gutted not to be able to celebrate 20 Years of Hopes and Fears with you at the Co-Op Live on Sunday. This is due to ongoing technical issues at the venue and is entirely beyond our control.
Take That’s full statement reads: “Given the ongoing technical issues around the opening of Co-Op Live we have taken the difficult decision to move our May shows to the AO Arena where we have enjoyed many great nights over the years.
“This is not a decision we have taken lightly, but we wanted to give our fans as much notice as possible.
“We’re mindful many of you will already have travel and accommodation plans in place, so we have chosen this option to minimise inconvenience to as many people as possible. Our dates in June in Manchester remain unaffected.
“All bookings will be transferred to the new venue as below and, except for Wednesday 8th May, we are pleased to say that all the dates remain the same.”
Notably, the huge new venue by the Etihad Stadium had to turn away fans who were already queueing for A Boogie Wit Da Hoodie on Monday 1 May, after part of an air conditioning unit fell from the ceiling onto a (thankfully) empty arena floor.
Question marks had already been hanging over whether some upcoming gigs would be able to go ahead and now it seems that they will be taking place – just not at the Co-op Live…
Manchester’s decades-old AO Arena, the largest indoor venue in the UK (until the Co-op Live opens), will be welcoming A Boogie Wit Da Hoodie and Take That instead.
It’s an interesting twist when so many people having been pitting the two venues against one another over the last weeks and months.
One of Manchester’s newest arts venues to host brand-new comedy festival this summer
Emily Sergeant
A brand-new comedy festival is set to take over one of the city centre’s newest arts venues this summer.
That’s right… some of the UK’s best comedy names are coming to Manchester.
That’s because renowned production company, A Lovely Time, has put together an absolutely jam-packed lineup of television names, internet sensations, and hotly-tipped newcomers, for a weekend full of comedy shows in the heart of the city centre at the exciting new arts venue Fairfield Social Club – which is tucked away down at the Green Quarter.
The exciting new festival, which is called A Lovely Weekend, will kick off on Friday 7 June with a gala show hosted by the company’s very-own Amy Gledhill, who comedy fans may recognise from appearances on BBC‘s Would I Lie To You, and more.
Then, across the rest of the three-day long weekend lineup, you can expect to catch comedy sets and work-in-progress performances from the likes of Edinburgh Comedy Award nominees Phil Ellis (Icklewick FM on BBC Radio 4), and Olga Koch (Live at The Apollo on BBC One), Paddy Young, who recently won the BBC New Comedy Award, Chris Cantrill (Avoidance on BBC One), and Blackpool’s-finest Tom Lawrinson.
Internet sensations such as Stevie Martin and Abi Clarke are also on the lineup, as well as hotly-tipped Edinburgh debutante, Hannah Platt, and Geordie sketch supergroup, Metroland.
You can even catch something a little bit different from Poppy Hillstead too, who’s set to transfer her popular podcast to the stage.
One of Manchester’s newest arts venues is set to host a brand-new comedy festival this summer / Credit: @see_hef (via A Lovely Time on Instagram)
Split across two venues over three days, A Lovely Weekend is all looking set to be the largest undertaking from comedy production super-duo, A Lovely Time, which was created by promoter John Stansfield and comedian Chris Cantrill, and, as mentioned, it will take over the newly-opened Fairfield Social Club – a venue which has already hosted brilliant shows from the likes of Josie Long, Three Bean Salad, Ashley Gavin, and Nick Helm since opening back in March.
“We’re so excited to be bringing A Lovely Weekend to Manchester,” commented founder, John Stansfield.
“The city is long overdue a proper comedy festival, and across the three days at Fairfield Social Club, we’ve programmed some of our absolute favourites to ensure a lovely time for all.”
Tickets to A Lovely Weekend have just gone on sale yesterday (Wednesday 1 November), and are already selling fast – with both day and full weekend passes available from £20, and individual show tickets up for grabs from £6 each too.