The gene therapy Casgevy, which will now be offered to some patients in England, could help people live disease-free, experts say.

A cutting-edge gene therapy has been approved for NHS use for some patients with severe sickle cell disease. Casgevy, also ...

January 31, 2025 expert reaction to NICE final draft guidance on exagamglogene autotemcel (exa-cel) for severe sickle cell disease . Scientists comment on final draft guidance fro ...

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 ...

A systematic literature review found no evidence to support that physical exertion without rhabdomyolysis (muscle breakdown) or heat injury can cause sudden death for individuals with sickle cell ...

Immvention Therapeutix is partnering with Novo Nordisk to develop oral treatments for sickle cell disease (SCD) and other ...

Base editing utilizes enzymes to modify single amino acids at the most foundational unit of DNA, called a base.

Abnormal cells can clog blood vessels, leading to severe pain episodes and restricting oxygen delivery to vital organs ...