A single one-time gene therapy could free patients with α-thalassemia, a rare and debilitating blood disorder, from the burden of lifelong transfusions. A single one-time gene therapy could free ...

With colorful headphones and eyes glued to her favorite Phineas and Ferb Disney movie on the family iPad, Ada reclined in her hospital bed tethered to a bag of medications and syringes of blood. She ...

Feeling tired due to low hemoglobin is a common health concern, but if you experience recurrent anemia despite treatment, the ...

A bone marrow transplant can effectively cure thalassemia major, moving beyond traditional care methods. This life-changing ...

For the first time, U.S. regulators have cleared a treatment using CRISPR, the gene-editing technology, for patients. The product is Casgevy, a treatment for sickle cell disease and beta thalassemia, ...

SAN DIEGO — Kamila Saradpon, an 11-year-old girl from San Diego, is living with beta thalassemia, a rare inherited blood disorder that affects less than 2% of people worldwide. To raise awareness and ...

Agios Pharmaceuticals said Monday that its drug called mitapivat reduced the need for blood transfusions in patients with a severe form of beta-thalassemia, an inherited blood disorder. The results ...

The annual programme is aimed at educating students about thalassemia, an inherited blood disorder, and encouraging early ...

Thanks to groundbreaking in-utero blood transfusion technology, what was once a fatal diagnosis in the womb can now result in live births. However, this medical advancement created a new challenge: a ...

This week marks an incredible win for modern medicine. The first CRISPR-based gene therapy has just been approved for clinical use in the United Kingdom. Casegvy (Exa-cel), developed by Vertex and ...