Some doctors told him he'd live with the disease forever, but Khaled Alsheebani never stopped believing treatments would ...

Without the Sickle Cell Data Collection program, we will see greater burdens and higher costs for both families and health ...

Beam reports breakthrough results from BEAM-101's early-stage trial for severe sickle cell disease. Read more here.

Vertex Pharma's £1.65 million ($2.05 million) gene-editing therapy for sickle cell disease (SCD), Casgevy, has been given the green light for use by the NHS in England under a managed access ...

Two Italian women in their twenties on Tuesday received pioneering gene therapy for rare hereditary blood diseases in Pavia ...

Orphan Drug Designation is awarded to therapies intended to treat rare diseases that affect fewer than 200,000 people in the ...

BEAM-101, a genetically modified cell therapy for sickle cell, is designed to turn on HbF to counteract mutated adult ...

Oregon will increase access to affordable, lifesaving cell and gene therapies for individuals on the Oregon Health Plan (OHP) ...

A gene transfer approach to treating the bleeding disorder hemophilia B remains safe and effective long-term, as scientists ...

Glioblastoma has been deadly and hard to treat, but researchers say they’ve made progress with bivalent CAR T-cell therapy ...

The treatment worked; his sickle cell anemia has been effectively cured. The Wexner staff and his doctors threw a party for him June 9, 2025. Valverde works for the "Destination Medicine" program ...