Thousands of patients with sickle cell disorder are to be offered a new “life-changing” treatment on the NHS from today (3 May), following the approval and roll-out of a new drug that could significantly improve their quality of life. Up to 4,000 people with sickle cell disease in England could benefit from the drug Voxelotor […]
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 […]
Premature babies are to be routinely offered a “life-changing” treatment to prevent blindness on the NHS for the first time, the head of the health service has announced today. The drug, ranibizumab, could save the eyesight of babies born with a condition known as retinopathy of prematurity (ROP), which affects blood vessels in the retina, […]
Hundreds of people with a range of rare conditions primarily affecting the central nervous system will be fast-tracked to diagnosis and specialist care, thanks to the launch of a pioneering new NHS service. Patients with inherited white matter disorders (IWMDs), also known as leukodystrophies, will now have rapid access to expert teams, increased virtual support […]
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 is on track to eliminate Hepatitis C by 2025 thanks to a pioneering drug deal that is helping dramatically cut deaths – five years ahead of global targets. Following a five-year contract worth almost £1 billion to buy antiviral drugs for thousands of patients, deaths from Hepatitis C – including liver disease and […]
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 […]
A five-month old baby has become the first patient to receive a potentially life-saving drug on the NHS that can prevent paralysis and prolong the lives of children with Spinal Muscular Atrophy (SMA). Arthur Morgan, who was diagnosed with SMA earlier this month, received the one-off gene therapy at Evelina London Children’s Hospital on May […]
A world-leading treatment that halves the risk of lung cancer patients suffering a return of the disease after undergoing treatment, is to be rolled out by NHS England. In a trial of the drug – the first of its kind – around nine out of ten patients treated, remained alive and disease-free after two years […]