Patients with severe inherited blood disorders will be offered stem cell transplants on the NHS that could cure their condition and help avoid life-long blood transfusions. Thalassaemia is a rare disorder that affects the production of haemoglobin in the blood, leading to severe anaemia and debilitating tiredness, with patients needing to have blood transfusions every […]
Babies and toddlers with a rare and fatal genetic condition can now receive a life-saving treatment on the NHS for the first time. Sebelipase alfa (Kanuma®), an enzyme replacement therapy, will become the first treatment available on the NHS for Wolman disease, a condition which presents in babies and children under two years old. Following […]
Nearly three in four babies born with a rare muscle-wasting disease are now surviving for two years or more thanks to advances in NHS treatment, new data shows. Around 70 children are born with spinal muscular atrophy (SMA) each year in the UK, a rare genetic condition that causes muscle weakness, progressive loss of movement […]
The NHS has launched plans to expand pioneering subscription-style drug contracts to develop lifesaving antibiotics of the future. It comes as data shows more people are contracting drug-resistant superbugs than ever before, with antimicrobial resistance (AMR) predicted to cause 10 million global deaths each year by 2050 if no action is taken. NHS England is […]
Two hundred children in England have now received life-saving treatment for hepatitis C as part of a world-first NHS programme, according to latest figures. Thanks to a new national service, the children – aged three to 18 years – were identified for treatment over the past two years and became the first in the world […]
A 19-month-old baby girl called Teddi has become the first child in the UK to receive a life-saving gene therapy treatment for the fatal disorder, metachromatic leukodystrophy (MLD). The revolutionary gene therapy, known by its brand name Libmeldy®, has a list price of £2.8 million and was the most expensive drug in the world when […]
The NHS will be able to prescribe cannabidiol to patients with a rare, seizure-causing genetic disorder in England from today (Tuesday) after the National Institute for Health and Care Excellence (NICE) issued final guidance recommending its use. Around one in every 6,000 people suffer from tuberous sclerosis complex (TSC) that causes seizures, which severely affect […]
The NHS will roll out an innovative, potentially life-saving treatment for one of the most aggressive forms of breast cancer thanks to a new drug deal. Up to 1,600 women a year affected by high risk triple-negative breast cancer are set to benefit from the confidential deal struck by the NHS and the manufacturer. Pembrolizumab […]
The NHS in England is on course to become the first country in the world to stop new cases of HIV before 2030, by offering a full armoury of HIV-busting drugs. Thanks to a series of national deals struck by the NHS, 87,000 people currently being treated for HIV and 61,000 people receiving preventative treatment, […]
The NHS has today launched a new online vaccine site finder to make it as easy as possible for eligible people to get their monkeypox jab. Almost 50,000 people have so far been vaccinated for monkeypox since the NHS rolled out the programme at speed once the first cases were identified. The UK Health Security […]