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

Transfusions of red blood cells (RBCs) can be life-saving treatments for patients suffering from anemia, where the body lacks enough healthy RBCs to effectively deliver oxygen around the body. But the ...

Vertex Pharma's £1.65 million ($2.05 million) gene-editing therapy for sickle cell disease (SCD), Casgevy, has been given the ...

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 ...

It is believed around 1,700 people could be eligible for the treatment, while the NHS estimates around 50 people a year will ...

Sickle cell disease can be life ... and support they deserve." The therapy has already been approved for another inherited blood disorder, transfusion-dependent beta thalassemia.

NHS patients with sickle cell disease will be able to benefit from a groundbreaking gene-editing treatment that offers the prospect of a cure for the condition. The one-off gene therapy, known as ...

Despite progress in healthcare services for individuals living with sickle cell disease (SCD) in Africa, substantial gaps ...

Vertex Pharmaceuticals Inc. (VRTX), a commercial-stage global biotechnology company, announced Friday a reimbursement agreement with ...

In 2018, she suffered a stroke, a pulmonary embolism and a blood transfusion ... for patients when a stem cell transplant is suitable but no donor can be found. The treatment was first rejected ...