One of the largest pharmaceutical companies in North Carolina is discontinuing a treatment produced in the state.

Eleven children who were legally blind at birth all gained visual acuity after receiving MeiraGTx's investigational gene ...

Pfizer is pulling further away from the gene therapy field with its decision to discontinue hemophilia product Beqvez. | ...

Pfizer’s decision to halt further marketing of Beqvez is further sign of the sparse patient interest in gene therapies for ...

Betty is a caregiver to three sons, Max, Rowen, and Charlie. Her oldest was diagnosed with Duchenne muscular dystrophy in 2010 at 4 years old. The other boys were diagnosed with the same in the ...