News

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 into…

Case Series: Plasma Exchange Can Help Reduce Bleeding in AHA

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…

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…

COVID-19 May Trigger Acquired Hemophilia A, Case Study Says

SARS-CoV-2, the virus that causes COVID-19, might have triggered acquired hemophilia A (AHA) in a 65-year-old man with a clinical history of autoimmune thyroid disease, a U.S. case study reports. This case suggests that COVID-19 infection should be considered when looking for potential causes of AHA. The study, “…