The National Hemophilia Foundation (NHF), through its Nursing Working Group (NWG), has released new guidelines to help hemophilia A patients self-administer Hemlibra (emicizumab). In its announcement, the foundation also highlighted the issuance of new guidelines for nurses to ensure safe and effective administration of standard into-the-vein…
GreenGene F (beroctocog alfa), a lab-made version of coagulation factor VIII, has been approved by regulators in China for the control and prevention, or prophylaxis, of bleeding episodes associated with hemophilia A, the therapy’s developer, GC Pharma,  announced. GreenGene F will be available in China in the first…
Registration is now open for Global Genes‘ 2021 RARE Patient Advocacy Summit. This year’s hybrid event will be livestreamed from California Sept. 27-29, and some seats also are available for attending the event in person in San Diego. “Here you’ll have the opportunity to connect and engage with others…
Medexus Pharmaceuticals has completed patient enrollment in its clinical trial investigating Ixinity as a preventive treatment for children younger than 12 with hemophilia B. The open-label Phase 4 trial (NCT03855280) achieved its target enrollment goal of 22 participants and is being conducted in 15 centers in Brazil,…
Belief Biomed is launching a Phase 1/2 clinical trial in China to test BBM-H901, its investigational one-time gene therapy for hemophilia B. The announcement comes after China’s National Medical Products Administration (NMPA) approved the company’s investigational new drug application, which had been submitted in April, Belief Biomed said in…
Applications are now open for the Hemophilia Federation of America’s 2021 Young Adult Advocacy Summit, or YAAS, which will be held virtually Sept. 19-20. The deadline to apply is Aug. 20. The summit targets adults between the ages of 18 and 30 who have a bleeding disorder…
Registration is now open for the 2021 Rare Diseases and Orphan Products Breakthrough Summit, which will be held virtually Oct. 18–19. The event, also known as the National Organization for Rare Disorders (NORD) Summit, brings the rare disease community together to network and discuss developments in treatments and research…
Therapeutic plasma exchange (TPE) was found to reduce bleeding and ease disease symptoms in six people with acquired hemophilia A (AHA), a case series reported. These findings support TPE — in which the liquid part of blood is replaced with substitute plasma — as an alternative treatment option in…
InSourceRx is partnering with the National Hemophilia Foundation (NHF) to help those with inheritable blood disorders, such as hemophilia, better afford medications. InSourceRx is a pharmacy discount card company that teams up with nonprofit and patient advocacy organizations to provide discounts for essential medicines and supplies. Blood…
More than 600 people participated in the 10th annual Rare Disease Week on Capitol Hill 2021, held virtually July 14–22, to advocate for the rare disease community. Hosted by the EveryLife Foundation’s Rare Disease Legislative Advocates (RDLA) program, the event brings together community members from across the U.S. to…
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