Breakthrough Bleeds Likely for Hem A Patients on Hemlibra, Study Finds
Most people with hemophilia AÂ on preventive treatment with Hemlibra (emicizumab) will experience spontaneous or traumatic bleeds at some…
Articles by Marisa Wexler, MS
Hemlibra Works to Reduce Bleeding Rates in Hemophilia A Children
Preventive treatment with Hemlibra (emicizumab) safely and effectively reduced bleeding rates in children with hemophilia A who were…
LeaderMed, OPKO to Develop Treatments in Asia
Two pharmaceutical companies, LeaderMed Health Group and OPKO Health, are teaming up to develop, manufacture, and commercialize two experimental…
BeneFIX Again Preventive Therapy for Children With Hemophilia B
The U.S. Food and Drug Administration (FDA) has restored its approval of BeneFIX as a routine prophylactic, or preventive, treatment…
MarzAA Reduced Bleed Frequency in Certain Male Patients
Preventive treatment with marzeptacog alfa activated (MarzAA) can reduce bleed frequency substantially in men with hemophilia A or…
Roctavian’s Benefits Sustained for Up to 5 Years in Trial Patients
Treatment with the investigational gene therapy Roctavian (valoctocogene roxaparvovec) can reduce bleeding frequency and the use of…
Patients’ Priorities for Gene Therapy: Durability and Bleeding Control
Effectiveness in controlling bleeds and the potential for less frequent use of a treatment are two priorities people with…
Fewer Bleeds Follow Move to Extended Half-life Therapy, Study Finds
People with severe hemophilia A who switch from standard to extended half-life replacement therapies experience fewer bleeds and require…
Medicaid Spending on Hemophilia Therapies in US Tripled in 2005-19
Spending on treatments for hemophilia by Medicaid, a U.S. government health insurance program for select groups, more than tripled…
Rare 2030 Action Campaign Pushing for European Plan for Rare Diseases
Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for…