News

What exactly is a gene? What happens when genes mutate? And can gene therapy research help solve problems related to mutations? Learn…

The Q&A resource highlighted here is meant to encompass all stages of a patient’s decision making journey in gene therapy. As investigational…

The focus of the abstract is the investigational hemophilia A gene therapy, valoctocogene roxaparvovec. BioMarin recently shared findings from a phase 3…

Just one bleed in people with severe hemophilia A receiving prophylaxis has the potential to negatively affect health-related quality of life, and raise…

Glanzmann Thrombasthenia is an ultra-rare inherited bleeding disorder that is characterized by poorly functioning platelets. The recent Congress of the International Society…

These updates were presented during the International Society on Thrombosis and Haemostasis (ISTH) Congress in London. Sanofi recently shared new clinical trial…

A newly launched program is designed to advance a diagnostic and precision medicine approach in newborns. The Rady Children’s Institute for Genomic…

Read a citizen’s petition that was formally submitted to the Food and Drug Administration by NHF. On July 1, the National Hemophilia…

The new recommendation describes the proven benefits of genotyping people with hemophilia NHF’s Medical and Scientific Advisory Council (MASAC) has approved a…

The hemophilia data for this study was drawn from de-identified medical records from four North American hemophilia treatment centers. The U.S. diabetes…

Kogenate® was first approved by the U.S. Food and Drug Administration in 1993. Bayer has announced that they are discontinuing the recombinant…

BIVV001 is an investigational recombinant factor VIII therapy (rFVIII) developed for the prevention of bleeding episodes in hemophilia A patients via once…

Roctavian is Biomarin’s investigational hemophilia A gene therapy, which is currently in multiple ongoing clinical trials. BioMarin recently announced that their planned…

NHF staff and experts share insights on the remarkable impact of Dr. Charles Drew. Watch Video. June 3 is the birth date…

GLHF is honored to announce that we have secured another five-year cooperative agreement with the Health Resources Services Administration (HRSA) to continue…

The study looked at the prevalence and risk factors of osteoporosis, osteoarthritis, and bone fractures. The issue of poor bone health represents…

The U.S. Food and Drug Administration (FDA) has accepted CSL Behring’s Biologics License Application (BLA) for priority review of etranacogene dezaparvovec, an…

by: Lindsey Shapiro Ph.D.  Symptoms of pain, anxiety, and depression generally increased with disease severity in people with hemophilia and were also associated with…

Newly published data from a phase 3 clinical study suggests that the recombinant factor VIIa bypassing agent (BPA) eptacog beta was used…

Dr. Oluwole is a recent JML Fellowship recipient. Watch Dr. Oluwole’s video, Dedicated to Finding a Cure for Sickle Cell Disease, here….

Read the original press release here: https://s28.q4cdn.com/781576035/files/doc_financials/2022/q1/Q1-2022-PFE-Earnings-Release.pdf During a recent quarterly stakeholder report, Pfizer announced that in March 2022 the US Food and…

Newly published data from a phase 3 clinical study suggests that the recombinant factor VIIa bypassing agent (BPA) eptacog beta was used…

Here’s a rundown of the various blood tests providers use to diagnose hemophilia, vonWillebrand Disease and other bleeding disorders. by: Michael Hickey…

Insights from historian Dr. Helen Rappaport – NHF sat down with historian Dr. Helen Rappaport to learn more about how hemophilia impacted…