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 ...

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

Vertex Pharmaceuticals (Nasdaq: VRTX) announced today a reimbursement agreement with NHS England for eligible sickle cell disease (SCD) patients to access the CRISPR/Cas9 gene-edited therapy, CASGEVY® ...

YolTech Therapeutics, a clinical-stage gene editing company dedicated to delivering lifelong cures, announced the initiation of a clinical trial for YOLT-204 , an investigational ...

In particular, patients suffering from severe disease (beta thalassemia major ... and 11 of 15 (73%) with β-thalassemia, achieved the primary endpoint of hemoglobin response, a ≥1.0 g/dL ...

Last January, the FDA approved Casgevy again to treat patients with beta-thalassemia, another hemoglobin-related disorder. Citing political uncertainty regarding healthcare, Bank of America ...

The disease is hallmarked by the abnormal production of hemoglobin, which causes chronic ... the company is exploring expansions into beta thalassemia and sickle cell disease.

According to research studies and demographic surveys by the Union Ministry of Tribal Affairs and genetic researchers, sickle cell disease is widespread in many tribal groups of India, especially amon ...