‘Functional cure’ for sickle cell disease now offered on NHS
Read about exa-cel being rolled out on the NHS, providing a 'functional cure' for some patients with severe sickle cell disease.
NHS to offer 'groundbreaking' sickle cell gene therapy
The gene therapy Casgevy, which will now be offered to some patients in England, could help people live disease-free, experts say.
‘Much-needed hope’ as CRISPR gene editing therapy recommended for sickle cell disease
Experts hailed exciting news for some people with sickle cell disease in England as the health watchdog decided to grant access to gene-editing therapy on the NHS. View on euronews
NHS England to offer Casgevy for sickle cell disease patients
The UK’s Medicines and Healthcare Regulatory Agency (MHRA) approved Casgevy for sickle cell disease in 2023, followed by approval for transfusion-dependent beta-thalassemia (TDT) in January 2024. NHS England reached an earlier reimbursement agreement for Casgevy in TDT in August 2024.
Sickle cell patients share delight over approval of new treatment
NHS officials estimate that around 50 people a year will receive treatment now that it has been approved for use for certain patients with severe sickle cell disease. Here patients have reacted to the news:
UK health system to offer cutting-edge gene therapy for sickle cell disease
Britain's National Health Service (NHS) will provide a cutting-edge gene therapy that aims to cure sickle cell disease, the National Institute for Health and Care Excellence (NICE) said on Friday. The therapy from Vertex Pharmaceuticals and CRISPR Therapeutics will cost the state-funded healthcare system around 1.
Groundbreaking one-off gene therapy approved for severe sickle cell disease
People in England to benefit from groundbreaking one-off gene therapy for severe sickle cell disease approved by NICE
‘Ground-breaking’ gene treatment for sickle cell disease to be rolled out on NHS
A GROUNDBREAKING genetic treatment with the potential to cure sickle cell disease will be rolled out on the NHS. The one-off treatment costs more than £1million per patient but may essentially
Sickle cell disease: new treatment raises hopes of cure for sufferers
The Exa-cel gene therapy treatment is at the forefront of medical technology. It takes the person’s DNA and “edits” out the faulty sickle gene. It’s expensive, too. According to the manufacturer, Vertex, it costs £1.65 million per patient. But the NHS has negotiated a discount which is kept confidential.
Vertex, U.K. reach reimbursement deal for CRISPR-based medicine for sickle cell disease
People in England with sickle cell will be able to get the world’s first CRISPR-based medicine under an agreement between the U.K. and Vertex.
Sickle cell disease ‘cure’ to be offered to NHS patients
Around 1,700 people could be eligible for the one-time treatment, which is seen as a less risky alternative to a donor stem cell transplant
South Jersey Chapter of Jack and Jill teams up with American Red Cross for sickle cell disease blood drive
There will be a blood drive for sickle cell disease – the most common inherited blood disorder – this weekend in South Jersey ...
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‘Sickle cell pain is the worst I have ever felt in my life - it’s hard to put into words'
Everything you need to know about agonising sickle cell disease and the ‘revolutionary’ new DNA therapy that's being rolled ...