Individuals with thalassaemia minor, often referred to as thalassaemia trait, inherit one mutated gene from either of their ...
Alpha thalassaemia trait (mild anemia) Three genes mutated: Haemoglobin H disease (moderate to severe anemia) Four genes mutated: Alpha thalassaemia major (hydrops fetalis), often fatal before or ...
The FDA approved betibeglogene autotemcel (beti-cel; Zynteglo) for adult and pediatric patients with transfusion-dependent ...
it is referred to as "HbAS" or "sickle cell trait". Other, rarer forms of sickle-cell disease include sickle-haemoglobin C disease (HbSC), sickle beta-plus-thalassaemia (HbS/β+) and sickle beta ...
Vertex and CRISPR Therapeutics have won the backing of NICE for their gene-editing therapy Casgevy as a treatment for the blood disorder beta thalassaemia, a few months after turning it down for ...
Therefore people with sickle cell trait – just one sickle cell gene ... new research into sickle cell disease. Thalassaemia is one of a group of genetic blood disorders referred to as ...