News

GreenGene F, Next-gen Hemophilia A Treatment, Approved in China

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…

Register Now for Global Genes’ RARE Patient Advocacy Summit

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…

Phase 4 Trial of Ixinity for Pediatric Hemophilia B Is Fully Enrolled

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,…

China OKs Hemophilia B Trial Testing BBM-H901 Gene Therapy

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…

Apply Now for Online 2021 Young Adult Advocacy Summit

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…

NORD Rare Disease Summit, Online Oct. 18-19, Open for Registration

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…

NHF, InSourceRx Offering Prescription Savings

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…

Advocates Lobby US Congress During Virtual Rare Disease Week

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…

Injection Therapy Like Hemlibra Favored Over Infusions by Adults

Adults across the general public in Canada think an under-the-skin injection treatment for hemophilia A would be preferable to those needing to be infused intravenously, or directly into a vein, a study based on interviews reported. Results of the analysis, sponsored by Roche Canada and conducted by Broadstreet…