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

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

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 ...

Discover the latest addition to Children's Hospital Los Angeles' 10 cell and gene therapy treatments, including Casgevy for sickle cell disease ...

The UK’s Medicines and Healthcare Regulatory Agency (MHRA) approved Casgevy for sickle cell disease in 2023, followed by ...

Vertex Pharmaceuticals (VRTX) announced a reimbursement agreement with NHS England for eligible sickle cell disease patients to access the ...

CRISPR Therapeutics is a mid-stage biotech specializing in gene editing. The company has just one product on the market, Casgevy, which treats transfusion-dependent beta-thalassemia and sickle cell ...

Vertex Pharmaceuticals Inc. (NASDAQ: VRTX) shares are trading higher Friday following two major developments that strengthen ...

Opens in a new tab or window An essential factor in distinguishing thalassemia phenotypes is transfusion dependence. While beta-thalassemia major requires life-long blood transfusions for survival ...