Non-transfusion-dependent thalassemia encompasses three clinically distinct forms -- beta-thalassaemia intermedia, hemoglobin E/beta-thalassaemia (mild and moderate forms), and alpha-thalassaemia ...

Non-transfusion-dependent thalassemia encompasses three clinically distinct forms -- beta-thalassaemia intermedia, hemoglobin E/beta-thalassaemia (mild and moderate forms), and al ...

Meanwhile, Editas Medicine is advancing EDIT-301, a therapy for sickle cell disease and beta-thalassemia that takes a novel approach using CRISPR-Cas12a (Cpf1). This method edits the HBG1 and HBG2 ...

The FDA approved betibeglogene autotemcel (beti-cel; Zynteglo) for adult and pediatric patients with transfusion-dependent ...

In particular, patients suffering from severe disease (beta thalassemia ... with α-thalassemia and 11 of 15 (73%) with β-thalassemia, achieved the primary endpoint of hemoglobin response ...

Thalassemia is a genetic blood disorder that affects hemoglobin production, leading to severe anemia and requiring lifelong medical management, including regular blood transfusions. The impact of ...

There were no serious adverse events or treatment-related mortalities. Evidence Rating Level: 2 (Good) Study Rundown: Transfusion-dependent β-thalassemia (TDT) is a condition that results in iron ...

Genetic mutations in the β-globin gene lead to a decrease or removal of the β-globin chain, causing the build-up of unstable alpha-hemoglobin. This condition is referred to as beta-thalassemia (BT).

A & B. Bone marrow smear (MGGx1000). Pseudogaucher cells: big cells with large cytoplasm with onion layers appearance and eccentric nuclei. PCG are histiocytes with round contour and cytoplasm ...