Toddler with fatal genetic disease given world’s most expensive drug – but it’s too late to save sister
A 19-month-old girl has been successfully treated for a fatal genetic disease with the world’s most expensive drug.
Teddi Shaw is the first person in the UK to receive Libmeldy, a treatment that corrects the underlying genetic fault that causes metachromatic leukodystrophy (MLD).
The gene therapy, which replaces a defective gene inside the body’s cells, has a list price of £2.8m.
Even with a “significant confidential discount” by UK-based biotech company Orchard Therapeutics, NHS England says it is still the most expensive drug licensed in Europe.
Teddi is thriving after having the treatment
But Teddi now shows no sign of MLD, a previously incurable disease that causes serious damage to the nervous system and organs.
Newly diagnosed patients have a life expectancy of just five to eight years.
Teddi’s mum, Ally, said: “Teddi is doing absolutely brilliant. She is walking, running, a chatterbox, absolutely no signs so far of MLD.
“She is an absolute character and has everyone around her laughing all the time.”
Teddi and her three-year-old sister Nala were both diagnosed with MLD in April last year.
Nala was unable to have treatment because of clinical guidance that it should start before the irreversible damage caused by the disease progresses too far.
Nala (R) also has MLD but it’s too late for her to get the same treatment as her sister
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 heartbreaking and hardest thing to come to terms with.
“However, amongst the pain, was hope for our younger daughter, Teddi.
“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.”
Teddi has been treated with Libmeldy
‘Miracle treatment’
Teddi was treated by Royal Manchester Children’s Hospital – in collaboration with Manchester’s Centre for Genomic Medicine at Saint Mary’s Hospital.
It’s the only hospital in the UK to offer treatment, and one of just five European sites to do so.
Ally feels privileged Teddi is getting the drug but hopes all babies can be screened for MLD in future
During treatment, stem cells are removed from the bone marrow or blood circulation. The faulty gene is the corrected with a healthy copy and the cells reinfused.
Amanda Pritchard, NHS England chief executive, called it a “miracle treatment”.
She 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.
“It means that children like Teddi can do the things that all children should be able to, like going to school and playing with friends.”
Around four babies a year are born in England with MLD. Previously, treatment was limited to managing symptoms and supportive care.
In future, Libmeldy will be available to children who have yet to develop symptoms or are in the early stages of the disease.
Ally urged the NHS to start genetic testing of babies so children like Nala can be diagnosed in time.
She said: “We can only hope that one day, a treatment becomes available for all stages of MLD, and we feel strongly that it should be added to the newborn screening test to save more families from having to go through this heartache.”
Weather warnings are in place for snow, wind and rain across much of the UK on New Year’s Day.
The start of 2025 will bring a “multi-hazard storm, combining severe gales, heavy rain and possibly significant snow as the rain runs into cold air,” said Sky News meteorologist Christopher England.
Pic: Met Office
The most significant snowfall on New Year’s Day will be from Donegal, across Northern Ireland and into the north of England and south of Scotland.
The wind looks strongest in the south, Mr England added, where 60mph winds are possible inland and 80mph gusts around the coasts of the Irish Sea.
The stormy weather continues on 2 January, with the Met Office issuing yellow weather warnings across England, Wales and parts of Scotland.
The UK has faced widespread disruption over the last few days, as thick fog descended.
Tens of thousands of passengers had flights delayed or cancelled, while drivers were warned about dangerous conditions with poor visibility.
Revellers are also set for a “wet and rather windy” New Year’s Eve, with the potential for a snowy Hogmanay in Scotland.
There could be some “possibly disruptive weather” on 31 December, Met Office meteorologist Simon Partridge said, with Scotland likely to see the worst of it.
The Met Office issued a yellow weather warning for winds of up to 70mph that may cause travel disruption on New Year’s Eve.
The alert, in place from 7am until 11pm on Tuesday, covers most of Northern Ireland as well as north of York in England up to Glasgow, Edinburgh and Greenock.
The forecaster also warned that delays to transport are likely, with gusts of up to 60mph expected in most areas.
Those affected should check road conditions for driving and the latest travel information as well as preparing for in advance for potential power cuts.
A pensioner who is refusing to leave what’s being branded Britain’s loneliest housing estate has told Sky News he is facing bullying and intimidation tactics to force him out.
Nick Wisniewski’s ex-council flat in Wishaw, North Lanarkshire is set to be demolished because the local authority wants to redevelop the area, which has become a derelict eyesore.
The vast area on the outskirts of Glasgow used to have almost 1,000 properties, but slowly the bulldozers have moved in, creating huge mounds of rubble where homes once stood.
The land is overgrown, with all remaining properties boarded up – apart from the retired bank worker’s home, which demolition teams have been unable to touch as he refuses to budge.
Nick Wisniewski claims he is being bullied to leave his home
Rubble where homes on the estate once stood
‘They’ll basically need to drag me out’
The 68-year-old, whose neighbours were all rehomed in 2023, bought the flat in 2017 under the discounted right-to-buy scheme and is now mortgage-free.
He told Sky News he rejected an initial offer from North Lanarkshire Council of £35,000 plus two year’s rent as it would not be enough to buy a new place.
Contemplating a time in the future when he is forced out, he said: “By law they can smash my door down, they can come in here, manhandle me to get me out. That’s the law, and they’ll basically need to drag me out.”
Asked if he is prepared to go to that length to stop himself being removed, he said: “I am hoping it doesn’t come to that, but if it does then so be it.”
He reveals a compulsory purchase order process is now under way.
A compulsory purchase order is the legal power given to a local council to buy a property without the owner’s consent.
The legal battle will now be resolved by the Scottish government in due course.
There used to be almost a thousand properties on the estate
He said: “It’s been all intimidation, bullying, lies. They offered me £40,000 over a year ago. I thought they’d have offered me a wee bit more. I would accept, probably, £60,000.
“They had a meeting a couple of weeks ago saying that they are starting legal, which means compulsory purchase order.
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“There’s so many stages to that and it’ll take a while for that to come in to force.”
North Lanarkshire Council told Sky News it “completely refutes” any allegations of bullying or intimidation.
A spokesman said: “We are continuing to try and engage with the remaining resident and our focus is on progressing our ambitious plans to transform this area of Gowkthrapple for the benefit of the local community.”
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