An eight-year-old Cheshire girl with a rare degenerative disease has become one of the first to receive a pioneering new treatment that will attempt to save her eyesight.
Amelia Carroll, from Poynton near Stockport, was diagnosed with CLN2 Batten disease – an incurable and fatal condition that affects roughly around 30-50 children in the UK – when she was just two.
The disease is caused by a change in a gene that is responsible for making a specific enzyme in the nervous system, and it results in seizures, a gradual decline in a child’s ability to walk, speak and see, as well as progressive dementia.
Amelia’s 11-year-old brother Ollie also has the same condition, yet has sadly already lost his eyesight.
“We have watched our son Ollie go blind, and now the same is starting to happen to Amelia,” mother Lucy Carroll explained, which is why her and her husband Mike decided to contribute to a fundraising campaign together with families of other children affected by the disease alongside the Battens Disease Family Association (BDFA) to help fund a world-first trail of a groundbreaking new treatment at Great Ormond Street Hospital (GOSH).
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Over £200,000 was raised through the campaign, and clinicians and researchers at GOSH and ICH say the treatment programme has the potential to transform the quality of life for children living with CLN2 Batten disease.
Amelia became one of the first children in the world to receive the treatment, and is said to already be responding to it “perfectly”, CheshireLive reports.
For the past two years, enzyme replacement therapy has been used to help prevent neurological deterioration in children with CLN2 disease. The drug, called Brineura, was first approved for use on the NHS in 2019 and is administered directly into the brain by a regular infusion.
It has shown to restore enzyme activity and slow the onset of disability.
However, this infusion into the brain does not prevent children going blind, as the enzyme cannot cross the blood-retina barrier, and this means that the nerves in the eye cannot function and vision is lost, which is why clinicians and researchers at GOSH and ICH are trying a the treatment to prevent the onset of blindness instead.
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To do this, GOSH said they are using the tiny amount of the drug leftover from the brain infusion and injecting it directly into the back of the eye.
The 18-month treatment trail – which is said to be being given on a compassionate use basis – hopes to prevent eyesight deterioration in children by providing the enzyme that the nerves in the back of the eye need to function.
Ms Carroll said “to save a child’s sight would be incredible”, and if it does indeed work, it would “make such a big difference to Amelia’s quality of life”.
“We just pray it works,” she added.
Speaking on the groundbreaking treatment trail, Professor Paul Gissen, from GOSH and the UCL GOS Institute of Child Health, said: “If successful, we hope our work can pave the way to saving the sight of more children with this disease to preserve their quality of life for as long as possible.”
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Amelia’s father, Mike Carroll, said the “amazing” nurses and doctors treating his daughter were “incredible”.
Advice issued as ‘quademic’ of winter bugs continues to take hold in Greater Manchester
Daisy Jackson
People in Greater Manchester are being urged to get any vaccinations they are due done, as a ‘quademic’ of winter bugs runs rife.
Cases of flu, Covid, norovirus and RSV (respiratory syncytial virus) are continuing to take hold across the country, and here in Greater Manchester.
The city is seeing more flu cases and hospital admissions than usual for this time of year.
So now health chiefs are urgently reminding eligible people to get their flu jabs and other vaccinations, to protect themselves and others.
Mancs are also being reminded to follow key public health advice, such as frequent hand washing and staying at home when you’re ill.
The flu vaccination drive runs until the end of March and people who are eligible for it can get it from their GP, pharmacy or one of the city’s walk-in clinics, where no appointment is needed.
Dr Cordelle Ofori, Manchester’s Director of Public Health, said: “Our advice is very similar to recommendations made during the Covid pandemic.
“It’s really important to keep washing hands well and regularly; cough or sneeze into tissues or your arm; wipe down key communal surfaces like handles on doors; and where possible, stay at home if you are ill to avoid passing on any infections.
“And, in addition to this we would urge anyone who is also feeling financial or food pressures to ring our free cost of living advice line on 0800 023 2692 or text on 07860 022876.”
Councillor Thomas Robinson, Executive Member for Healthy Manchester and Social Care, at Manchester City Council, said: “Manchester people are well-known for how they look after one another – and passing the flu message on could save lives, as well as unnecessary illness at a time when people face many pressures.
“We want to make it as easy as possible for people to have their vaccination, so please do go to our walk-in clinics if you are 18 and over and eligible for the vaccine. No appointment is needed: it’s quick, easy and done by teams who want to help you.”
Nurses plea for Government support as 90% say patient safety is being ‘compromised’
Emily Sergeant
Patients dying in corridors, a lack of equipment, and generally unsafe practices are the findings of a harrowing new report into nursing.
Towards the end of last month, the Royal College of Nursing (RCN) – which is biggest nursing union and professional body in the world, with more than 500,000 members – asked its members to answer a short survey into the state of nursing throughout 2024, and thousands of nurses across the UK responded.
The report documents the experiences of more than 5,000 NHS nursing staff, with several raw, unedited, and often heartbreaking responses included, all of which confirm that ‘corridor care’, as it’s known, is “widespread” nationwide.
Almost seven in 10 (66.8%) respondents said they’re delivering care in “over-crowded or unsuitable places”, such as corridors, converted cupboards, and even car parks, on a daily basis.
More than nine in 10 (90.8%) said patient safety is being “compromised”.
According to the findings from the survey, demoralised nursing staff reported caring for as many as 40 patients in a single corridor, and find they are unable to access oxygen, cardiac monitors, suction, and other lifesaving equipment during this time.
Some of the more shocking accounts including in the report are of female patients miscarrying in corridors, and nurses being unable to provide adequate or timely CPR to patients having heart attacks.
Nursing staff also report cancer patients being put in corridors and other “inappropriate” spaces.
Nurses are calling for Government support as 90% say patient safety is being ‘compromised’ / Credit: Stephen Andrews (via Unsplash)
More than a quarter of nursing staff surveyed said they weren’t told the corridor they were providing care in was classed as a “temporary escalation space”, which is what the report says NHS England referred to it as, and this means that certain risk protocols and measures may not be in place to ease pressures and protect patients.
This is why the RCN is calling on officials to publish how many patients are being cared for in corridors and other inappropriate places.
The union is urging the Government and NHS England from an RCN-led coalition.
RCN General Secretary and Chief Executive, Professor Nicola Ranger, said this devastating testimony from frontline nursing staff shows patients are coming to harm “every day”, and are “forced” to endure unsafe treatment.
“The revelations from our wards must now become a moment in time,” Professor Ranger said.
“A moment for bold Government action on an NHS which has been neglected for so long. Ministers cannot shirk responsibility and need to recognise that recovering patient care will take new investment, including building a strong nursing workforce.”
Emily Sergeant -
14th January 2022 
