The first patient to receive a new gene therapy at the Children's Hospital of Philadelphia is now living pain-free for the ...

At Children’s Hospital of Philadelphia, an 11-year-old girl with severe beta thalassamia, became the first pediatric patient to receive Zyntelgo, the first FDA-approved gene therapy for ...

While gene therapy has emerged as a promising alternative for treating beta-thalassemia ... pointing out that better HLA typing is a major reason. However, a "big quantum leap" was the marked ...

Aruvant is developing ARU-1801, a one-time, potentially curative gene therapy for sickle cell disease and β-thalassemia. In an ongoing clinical phase 1/2 study, ARU-1801, administered with only ...

Thalassemia and sickle cell anaemia are common genetic disorders in India. Early testing, prenatal screening, and improved ...

the first CRISPR/Cas9 gene-editing therapy in the UAE. This achievement opens new horizons and offers innovative treatment for patients suffering from sickle cell disease (SCD) and transfusion ...

Thalassemia and Sickle Cell Anaemia are the commonest single-gene disorders globally ... The expected annual births of beta thalassemia major babies were also calculated for each district in ...