Columnist Jennifer Lynne reflects on a powerful event that kicked off this year's Hope Conference in Orlando, Florida.

Geneventiv said the FDA had "positive" feedback in a meeting on developing GENV-HEM, the company's gene therapy for ...

Inhibitors are one of the most severe complications of hemophilia. Caeleb’s complications didn’t stop with that diagnosis. He had at least eight ports, as they would become infected or break. Daily ...

Columnist G Shellye Horowitz writes that postponing factor infusions may be tempting, but prophylaxis is better than a bleed.

Hemophilia B is a rare genetic bleeding disorder that impairs the blood’s ability to clot properly. It is caused by a mutation in the F9 gene, located on the X chromosome, resulting in a deficiency in ...

Columnist Allyx Formalejo reminds readers this National Epilepsy Awareness Month that some stories about epilepsy begin with ...

Biomarin Pharmaceutical is looking to sell off rights to Roctavian (valoctocogene roxaparvovec-rvox), its approved gene therapy for hemophilia A. “As we focus on the business units aligned with our ...