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

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 ...

A renowned Harvard hematologist, Maureen Okam Achebe, has called for the institutionalisation of continuous newborn screening ...

Oregon Health Plan (OHP) members will see increased access to lower-cost, lifesaving cell and gene therapies. The state is ...

Updated Data from 17 Patients Consistent with Previously Presented Data; All Patients Treated with BEAM-101 Achieved Hemoglobin F (HbF) Induction of ...

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) ...