A groundbreaking sickle cell disease treatment has been approved for use in the UK's National Health Service (NHS). The treatment offers patients with severe forms of the condition hope for a ...

Right now, approximately 20 billion red blood cells are busy traveling through your blood vessels. They are delivering oxygen ...

One sickle cell patient told ITV News the new gene therapy being rolled out for use on the NHS means they "finally have something to look towards". | ITV National News ...

Drug-based therapy, starting with hydroxyurea ... The World Coalition on Sickle Cell Disease, launched in 2023, is an excellent example of the type of collaboration that will be needed to expand ...

As treatment and care continues to evolve, we must ensure that people living with this disease are able to advocate for ...

Read about exa-cel being rolled out on the NHS, providing a 'functional cure' for some patients with severe sickle cell ...

Sickle cell anaemia is a genetic blood disorder characterised by the production of an abnormal haemoglobin, known as ...

New gene therapies could transform sickle cell treatment forever. A future without pain crises or hospitalizations may finally be within reach.

Despite promising Phase 3 results, safety concerns, namely liver injury, have impacted Agios stock and could affect Pyrukund's approval and market adoption. Read the full report here.

Filed for Regulatory Approval of Mitapivat (PYRUKYND®) for the Treatment of Adult Patients with Non-Transfusion-Dependent and ...