Two Pee Dee siblings are officially cured of sickle cell disease after more than a decade of living with the genetic blood ...

Gene therapy is a medical treatment that works by changing or fixing a person's genes to treat or prevent disease ...

Researchers have found a link between the release from malformed red blood cells of the vital protein that carries oxygen and episodes of severe pain in people with sickle cell disease. The discovery ...

If you or someone you know has sickle cell disease (SCD), you may have heard of Lyfgenia, one of two gene therapies approved for this condition. Gene therapies are the only treatments for SCD that can ...

AND017 is an investigational oral hypoxia-inducible factor prolyl hydroxylase inhibitor. The Food and Drug Administration has granted Orphan Drug designation to KIND Pharmaceutical’s AND017 for the ...

Beam Therapeutics’ experimental gene-editing therapy for sickle cell disease now has its first cut of patient data showing signs of treating the rare blood disorder in a way that could differentiate ...

Key HighlightsMotixafortide effectively mobilized sufficient hematopoietic stem cells (HSCs) in patients with sickle cell disease and beta-thalassemia enabling accelerated access to gene therapies73% ...

Sickle cell anemia is an inherited condition that impacts red blood cells. It can cause a variety of symptoms, which may include anemia, infections, and blockages in blood vessels. Sickle cell disease ...

Sickle Cell Anemia is a genetic blood disorder that affects the shape and function of red blood cells. It can lead to severe health complications, including pain, infections, and anemia. Understanding ...

Sickle cell anemia is the most serious form of sickle cell disease (SCD). It occurs when a person inherits an irregular hemoglobin gene from each parent. Certain groups of people are more at risk of ...