BioMarin recently shared findings from a phase 3 clinical trial (GENEr8-1) evaluating the presence of vector DNA in patients two years after they received a single dose of valoctocogene roxaparvovec, the company’s investigational hemophilia A gene…
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Dr. Andrew Yee is an assistant professor of pediatrics within the Department of Pediatrics, Hematology-Oncology Section at Baylor College of Medicine. Dr. Yee earned his doctorate from Rice University where he studied the biological responses of…
You are cordially invited to participate in a Meet-Up Event for people affected by ultra-rare inherited bleeding disorders (URBDs) being held on September 9 – 11 at the Marriott SW, in Burr Ridge, Illinois. We know that you face significant…
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Speaker(s): Peter Aguero
NHF's Wednesday Webinars are a free education series open to providers and community members. Register to attend and learn about the latest in research, breakthrough developments, and more.…
The annual Awards of Excellence Ceremony is held yearly at the Bleeding Disorders Conference.PHYSICIAN OF THE YEAR(Named in honor of Dr. Kenneth Brinkhous) 2025 Jordan Wright, MD 2024 Mark Reding, MD 2023 Doris Quon,…
The Plasma Protein Therapeutics Association (PPTA) recently posted an online update that addresses safety concerns relevant to the recent monkeypox virus (MPXV) outbreak and plasma protein therapies.
According to the new statement, while…
The 2022 Bleeding Disorders Conference has kicked off with first issue of the NHF Show Daily!
Read it now
The European Commission granted conditional approval to BioMarin's Hemophilia A gene therapy Valoctocogene Roxaparvovec which will be known as Roctavian.With this approval in Europe the regulators will monitor the use of Valrox for 15 years to…