£1.65m gene-editing therapy offers hope of cure for some blood disorder patients
A £1.65 million treatment has been approved for use for some NHS patients, offering some with an inherited blood disorder hope of a cure. Campaigners reacted with joy as health officials approved a gene-editing therapy for certain children and adults with severe sickle cell disorder.
Revolutionary gene-editing therapy for sickle cell ‘offers hope of a cure’ for NHS patients
NHS patients with sickle cell disease will be able to benefit from a groundbreaking gene-editing treatment that offers the prospect of a cure for the condition. The one-off gene therapy, known as exagamglogene autotemcel (or ‘exa-cel’),
'Will it really work?': Young sickle cell patient among the first to start new gene therapy
Since two groundbreaking treatments were approved in late 2023, only a handful of patients have been able to access the multimillion dollar, medically-intensive therapies.
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.
NICE approves gene editing therapy for patients with severe sickle cell disease
The National Institute for Health and Care Excellence (NICE) has approved a one-off gene editing therapy to treat severe sickle cell disease (SCD) in people 12 years and over. In final draft guidance1 NICE approved exagamglogene autotemcel (exa-cel,
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.
NHS to offer 'groundbreaking' sickle cell therapy as patients given 'very real prospect of cure'
A groundbreaking £1.65million gene therapy treatment offering hope for sickle cell disease patients has been approved for use on the NHS in England. The one-time treatment, called Casgevy (exa-cel), has shown remarkable success in clinical trials,
Revolutionary gene-editing therapy for sickle cell approved
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Gene Therapy’s Pipeline Has a Steady Flow of More Than 2,000 Candidates
The majority are in the very early preclinical stage of development, but applications for regulatory approval have been ...
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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 ...
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 ...
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President Trump signed an executive order on the day of his inauguration that created doubts about the future of a program to ...
Gene therapy and bone marrow transplant alleviate ultra-rare genetic disease symptoms
In two new papers, researchers from The Jackson Laboratory (JAX) report the successful use of two approaches—gene therapy and ...
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Gene Therapy Stabilizes Rare Chronic HPV Disease
For recurrent respiratory papillomatosis (RRP), novel gene therapy to jump-start immunity to the human papillomavirus (HPV) ...