Hemoglobin disorders such as HbS and HbC affect millions of people worldwide. Countries around the world spend substantial resources each year identifying carriers, often relying on detailed ...

(MEMPHIS, Tenn. – July 03, 2023) Gene therapy that alters hemoglobin genes may be an answer to curing sickle cell disease (SCD) and beta thalassemia. These two common life-threatening anemias afflict ...

AstraZeneca Plc (NASDAQ:AZN) released data from the Phase 3 ALPHA trial of danicopan as an add-on to the standard of care for paroxysmal nocturnal hemoglobinuria (PNH). The data showed that danicopan ...

Michigan's health department announced a new plan to improve care and support for the 4,000 residents with sickle cell disease.

Agios' mitapivat shows statistically significant increase in hemoglobin response rate in thalassemia patients. Energize is first phase 3 study to demonstrate efficacy of an oral treatment for ...

In neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS), Parkinson’s, and Alzheimer’s, healthy brain cells are damaged by aberrant reactive oxygen species (ROS). A potential ...

Sickle cell disease (SCD) is a group of hereditary red blood cell disorders. Sickle cell anemia is a type of SCD that occurs when a person inherits genes containing an atypical form of hemoglobin.

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare disorder caused by a mutation in the PIGA gene. This mutation causes defective hematopoietic stem cells, which develop into defective red blood ...

Adenosine base editing restarted fetal hemoglobin expression in cells from patients with sickle cell disease. Gene therapy that alters hemoglobin genes may be an answer to curing sickle cell disease ...