News

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…

This study designed to assess the link between physical activity levels, FVIII infusion, and occurrence of bleeding episodes. Results of a recently…

Dr. Glenn Pierce reflects on the passing of former NHF CEO, Val Bias. Val and I first got to know one another…

Former CEO spent decades inspiring the inheritable blood disorders community. NHF is deeply saddened to announce that former NHF CEO and community…

The latest breaking news from the All Copays Count Coalition. In response to additional rulemaking for the Centers for Medicare & Medicaid…

In this video, Tammuella Chrisentery-Singleton, M.D., Chief of Hematology for the Louisiana Center for Advanced Medicine, gives an overview of gene therapy…

Here’s what we know about these ultra-rare platelet disorders. Most bleeding disorders are relatively rare. Even the most common bleeding disorder, von…

The National Hemophilia Foundation (NHF) today, in conjunction with the American Kidney Fund (AKF), Arthritis Foundation, and American Autoimmune Related Diseases Association (AARDA), released findings from…

The onset of childbirth and the postpartum period are times when women with von Willebrand disease (VWD) are at an increased risk…

Paper Highlights: Largest gene therapy study in hemophilia B achieved primary endpoint of non-inferiority in annualized bleeding rate after stable Factor IX…

This data was drawn from a phase 1/2 clinical trial of SPK-8011, Spark’s investigational gene therapy for hemophilia A. Spark Therapeutics announced…

On Tuesday, November 4, the U.S. Food and Drug Administration (FDA) placed the Pfizer/Sangamo hemophilia A gene therapy program, including the pivotal…

Historically, data on infants and toddlers (ITs) with von Willebrand disease (VWD), particularly relevant to bleeding patterns, has been lacking. To address…