Home / Health / Scotland Approves Gene Therapy "Functional Cure" for Sickle Cell
Scotland Approves Gene Therapy "Functional Cure" for Sickle Cell
19 Jan
Summary
- Groundbreaking gene therapy for sickle cell disorder approved for NHS use in Scotland.
- The treatment, Casgevy, modifies a patient's own stem cells to produce healthy red blood cells.
- This approval marks a significant milestone for patients with sickle cell in Scotland.
A significant advancement for sickle cell disorder patients in Scotland has been made with the approval of a new gene therapy for use on the NHS. This groundbreaking treatment, known as exagamglogene autotemcel or Casgevy, is being hailed as a "functional cure" for the condition.
Casgevy works by modifying a patient's own blood stem cells, which are then returned to their body. These altered cells are designed to produce healthy red blood cells, effectively addressing the debilitating symptoms of sickle cell disorder. Previously, a stem cell transplant was the only potential cure, but finding a compatible donor was a major barrier.
Patient advocacy groups and health organizations have welcomed the Scottish Medicines Consortium's decision. They emphasize that this approval ensures equitable access to advanced treatments for individuals in Scotland, aligning with advancements made in England last year. The move is seen as a crucial step in recognizing and addressing the needs of the sickle cell community.
