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.
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
Printworks to unveil its £21m transformation with huge free party
Manchester’s landmark venue Printworks will be celebrating the completion of its £21m refurbishment with a massive free party.
The huge new-look venue will have TikTok sensation DJ SUAT headlining the launch party on 21 March.
SUAT will be live-streaming from Manchester city centre during the day, running around town on his signature portable DJ decks and handing out VIP tickets for the Printworks party.
He’ll be performing around Exchange Square and streaming live from the roof of Printworks, with a third location yet to be revealed.
SUAT now has more than one million followers on social media thanks to his viral public live streams, which have included him DJing while paragliding over a festival and kayaking down Amsterdam Canal.
When the launch party itself kicks off in the evening, SUAT, who has played at some of the biggest club nights and festivals in the world, will be delivering a crowd-pleasing dance party for hundreds of guests at the city centre venue.
He’ll be joined for the evening by Bohemian House Choir and a host of other performers and dancers.
It’s all to celebrate the new-look Printworks, which will be an immersive, digital-first leisure destination and entertainment venue.
As part of the refurb, Printworks will be home to the largest digital ceiling in Europe, bathing the internal streets in colourful light and sound.
Printworks will throw a huge free launch party after its refurb.
Lydia Ellis from Printworks comments: “SUAT has been on a mission to change stereotypes in electronic dance music and has captured the hearts of fans across the globe.
“He’s the perfect star to celebrate the new, digitally immersive Printworks. The Centre will always be free to enter so everyone can come and experience the spectacular content, which has to be seen to be believed.
“We’re breaking new ground with our digital screens and SUAT is breaking new ground musically and with his style of performance. It’ll be a launch party like no other.”
The free Printworks launch party will take place from 7pm on Thursday 21 March, with SUAT headlining at 8.30pm.
Tina – The Tina Turner Musical is coming to Manchester on first-ever UK tour
Tina – The Tina Turner Musical has announced its first-ever UK and Ireland tour, and it’s coming to Manchester.
The smash hit West End production, which documents the life of the iconic singer and is packed with her biggest hits, will visit the Palace Theatre.
Tina – The Tina Turner Musical has been running at the Aldwych Theatre for more than five years, smashing box office records, and eight productions have opened worldwide.
The show is currently on tour in North America and Australia but details of a UK and Ireland leg have finally been announced, with a huge run in Manchester.
The glittering production tells the story of the 12-time Grammy Award-winning Queen of Rock n Roll.
It’s a true and inspiring story which captures how she dared to dream fiercely, shatter barriers and defy the bounds of age, gender, and race to conquer the world against all odds.
Its soundtrack includes her hits like The Best, What’s Love Got To Do With It?, Private Dancer and River Deep, Mountain High.
Producer Tali Pelman said: “Tina Turner played to packed out arenas across the UK and Ireland throughout her extensive career.
TINA – THE TINA TURNER MUSICAL. Credit: Matt Crockett Tina – The Tina Turner Musical is coming to Manchester’s Palace Theatre. Credit: Manuel Harlan
“We’re thrilled to now be able to bring her extraordinary story to stages across the UK and Ireland for the first time with TINA – THE TINA TURNER MUSICAL, celebrating the life and times of a truly inspirational woman whose story continues to move and elate audiences around the world every night.”
Tina – The Tina Turner Musical is dedicated to the memory of Tina Turner, who sadly passed away in May 2023.
Tina – The Tina Turner Musical will be at the Palace Theatre in Manchester from Tuesday 25 November 2025 to Saturday 3 January 2026.
Tickets will go on sale at 10am on Tuesday 5 March here.
Tina – The Tina Turner Musical UK tour dates in full
Thursday 6 – Saturday 22 March 2025, Leicester Curve
Wednesday 26 March – Saturday 5 April 2025, Sunderland Empire
Thursday 8 – Saturday 26 April 2025, Bristol Hippodrome
Tuesday 6 – Saturday 24 May 2025, Grand Opera House, Belfast
Tuesday 27 May – Saturday 14 June 2025, Bord Gais Energy Theatre, Dublin
Tuesday 17 – Saturday 28 June 2025, Birmingham Hippodrome
Tuesday 1 – Saturday 12 July 2025, Theatre Royal, Plymouth
Tuesday 15 July – Saturday 2 August 2025, Theatre Royal, Nottingham
Tuesday 12 – Saturday 23 August 2025, Empire Theatre, Liverpool
Tuesday 26 August – Saturday 6 September 2025, Cliffs Pavilion, Southend
Tuesday 9 – Saturday 20 September 2025, Sheffield Lyceum
Tuesday 23 September – Saturday 4 October 2025, King’s Theatre Glasgow
Tuesday 7 – Saturday 18 October 2025, Mayflower Theatre, Southampton
Tuesday 28 October – Saturday 8 November 2025, Marlowe Theatre, Canterbury
Tuesday 11 – Saturday 22 November 2025, Playhouse Theatre, Edinburgh
Tuesday 25 November 2025 – Saturday 3 January 2026, Palace Theatre, Manchester
Tuesday 13 – Saturday 24 January 2026, Theatre Royal, Norwich
Tuesday 27 January – Saturday 7 February 2026, Milton Keynes Theatre
Tuesday 10 – Saturday 28 February 2026, Wales Millennium Centre, Cardiff
Tuesday 3 – Saturday 14 March 2026, Theatre Royal, Newcastle
Tuesday 17 March – Saturday 4 April 2026, Leeds Grand Theatre