Geneticists have used a cutting-edge DNA base editing treatment on several patients to cure thalassemia, a prevalent and severe genetic blood disease.

With gene therapy offering new hope for thalassemia, experts urge Indian govt to invest in early diagnosis, genetic testing, ...

The development and approval of gene therapies has been a potential game changer for patients with beta-thalassaemia ... eligibility for gene therapy for thalassemia comes down to two main ...

A stem cell transplant and gene therapy may also be options ... symptoms related to moderate or severe anemia. With beta thalassemia major, you may have more symptoms. They may be severe.

KJ Muldoon, a 10-month-old baby, was diagnosed with the genetic disease carbamoyl-phosphate synthetase 1 deficiency after he ...

The first therapy that uses gene-editing ... living with beta thalassaemia with a potential cure for those facing this debilitating disorder now available on the NHS.” Beta thalassemia mainly ...

Exagamglogene autotemcel (exa-cel) (Casgevy®) is a gene therapy for treating sickle cell disease in patients 12 years and older with recurrent vaso-occlusive crises and transfustion-dependent beta ...

A new gene therapy developed at the Children's Hospital ... Nabeel was born with an inherited blood disorder called beta thalassemia. "It was just, like, so painful," she said.

"We have about eight patients that are in the insurance approval process," she said, for gene therapy treatments for beta thalassemia and sickle cell disease. "It's something that we do expect to ...

Two gene mutations: Beta-thalassemia major (Cooley’s anaemia ... coupled with iron chelation therapy to manage complications arising from iron overload. The annual cost of treatment can ...

New gene therapy improves the life of 18-year-old with Sickle Cell Disease which affects 5,000 New Jersey residents living ...