News

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…

The so-called “family glitch” is one of the persistent deficiencies in the Affordable Care Act. In their end-of-April post, the Hemophilia Federation…

The company’s investigational gene therapy has been developed for individuals with severe or moderately severe hemophilia B. The UK-based biotechnology company Freeline…

The new grant will fund basic and translational studies to improve gene therapies for hemophilia A. A group of investigators are embarking…

A group of investigators are embarking on a new research program designed to unpack some of the outstanding fundamental questions associated with…

Government health agencies have begun preparing for the end of the COVID-19 public health emergency. When the emergency period ends, state Medicaid…

An update for the hemophilia community from BioMarin, regarding the ongoing Phase 3 BioMarin hemophilia A gene therapy study, and a serious…

Read the original press pelease here. Efanesoctocog alfa met primary and key secondary endpoints in pivotal study in hemophilia A, demonstrating superiority to prior…

Freeline TherapeuticsHoldings plc announced that the first patient was dosed in its Phase 1/2 B-LIEVE dose-confirmation clinical trial of FLT180a for the…

Answers to common questions about this potentially game-changing treatment. After several decades of research, gene therapy for hemophilia is moving closer to…

Much is the same when it comes to hemophilia A and B, but a few key differences change how common—and how treatable—they…

Over 10,000 individuals worldwide have been treated with various gene therapy products. Gene therapy is a medical treatment that uses DNA to…

Cell therapy is a fascinating approach to treat hemophilia. But recently the clinical trials hit a stumbling block. What happened? Read more…

Dunn looks to diversity and advocacy as the incoming head of MASAC. One patient changed Dr. Amy Dunn’s entire career in her…

Takeda recently announced that the U.S. Food and Drug Administration (FDA) has approved VONVENDI® for routine prophylaxis to reduce the frequency of…

BioMarin’s investigational gene therapy for hemophilia A has not been approved for use; clinical trials are ongoing. Please click this link to read…

A Statement from the National Hemophilia Foundation: Like many reading this note, NHF is profoundly heartbroken over the recent passing of Val…

The United States Food and Drug Administration’s Office of Patient Affairs is sharing recent highlights with the public from 2021 and is…

The phase 1/2 trial of SIG-001 for hemophilia A has been on an FDA clinical hold since July 2021. Sigilon Therapeutics recently…