Hemlibra (emicizumab-kxwh) safely supports joint health in children with hemophilia A without inhibitors through three years of treatment, according to interim data from a post-marketing study in Japan. The ongoing study, AOZORA (jRCT1080224629), is evaluating the long-term safety of Hemlibra and its impact on joint health in children ages…

Geneventiv Therapeutics said it had a “positive and constructive” meeting with the U.S. Food and Drug Administration (FDA) on its plan to develop GENV-HEM, a gene therapy for hemophilia A. The meeting “represents a key regulatory milestone for Geneventiv,” Damon Race, CEO of Geneventiv, said in a…

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 strategic priorities, today we are announcing the decision to pursue options to divest Roctavian and remove it from…

Last in a series. Read parts one and two. For 21-year-old Domenic Catrine, hemophilia B once dictated every part of life, from wearing a helmet during childhood playtime to college routines shaped by weekly factor infusions. Then he underwent gene therapy during his sophomore year in…

Hemophilia has a substantial impact on sexual function and well-being for many men with the disease, but they rarely discuss it with their doctors, a study from the Netherlands found. “To address this gap, tools designed to provide and assess information about sexual functioning and well-being to normalize the…

Second in a series. Read part one. For most of his life, 21-year-old film student Domenic Catrine assumed weekly infusions were his destiny while living with hemophilia B. By the time he entered college, the rhythm of mixing vials and finding veins had become routine — even if…

First in a series.  Treatment for people living with hemophilia has long meant a lifetime of infusions, careful planning, and constant vigilance. But new therapies are rewriting what’s possible, particularly gene therapy. Domenic Catrine is a charismatic 21-year-old who lives with severe hemophilia B. A junior in college…

Nearly all adults with acquired hemophilia A (AHA) safely achieved complete remission with either standard or low doses of rituximab when combined with corticosteroids and cyclophosphamide, a small study shows. Key features of disease remission, including an increase in clotting factor levels and a reduction in inhibitor levels,…