News

The ACA’s “Family Glitch” – What it is and How it Could Impact You

Industry News & Research

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…

Read More

First Patient Dosed in Freeline’s Dose-Confirmation Trial for Hemophilia B Gene Therapy

Industry News & Research

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

Read More

NHLBI Grant Supports Scientific Research to Enhance Hemophilia A Gene Therapies

Industry News & Research

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

Read More

New Government Funded Research Program Created to Help Improve Gene Therapies for People with Hemophilia A

Industry News & Research

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

Read More

Medicaid Renewals Post Pandemic

Industry News & Research

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

Read More

BioMarin Provides Update to Ongoing Phase 3 Hemophilia A Gene Therapy Study

Industry News & Research

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

Read More

Sanofi and SOBI Announce Results of XTEND-1 Phase 3 Study

Industry News & Research

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

Read More

Freeline Announces First Patient Doses in Hemophilia B Trial

Industry News & Research

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…

Read More

An Update on Gene Therapy for Hemophilia

Industry News & Research

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

Read More

Hemophilia A vs. B: What’s the Difference?

Industry News & Research, Living with a Bleeding Disorder

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

Read More

A Brief History of Gene Therapy: What It Means and Its Promise for the Hemophilia B Community

Industry News & Research, Living with a Bleeding Disorder

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

Read More

Cell Therapy Hits a Stumbling Block

Industry News & Research

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

Read More

Meet MASAC’s New Chair: Dr. Amy Dunn

Industry News & Research

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

Read More

Takeda Announces Approval of Prophylactic Indication for VWD Therapy

Industry News & Research

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

Read More

Update from BioMarin Phase 3 Trial

Industry News & Research

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

Read More

Remembering Val Bias

GLHF News, Industry News & Research

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

Read More

The FDA’s Office of Patient Affairs Shares Highlights from 2021 Programs and Encourages you to Participate in their Upcoming 2022 Activities.

Advocacy & Legislation, COVID-19, Industry News & Research

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

Read More

Sigilon Announces a Priority Shift in Light of Clinical Trial Setbacks

Industry News & Research

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

Read More

New Study Looks at Physical Activity Risk in Patients with Hemophilia A

Industry News & Research, Living with a Bleeding Disorder

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

Read More

Remembering Val Bias: A Note from Dr. Glenn Pierce

GLHF News, Industry News & Research

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

Read More

National Hemophilia Foundation Mourns the Loss of Longtime CEO, Val Bias

GLHF News, Industry News & Research

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

Read More

All Copays Count Coalition Statement on Draft 2023 CMS Notice of Benefit and Payment Parameters

Advocacy & Legislation, Industry News & Research

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

Read More

Gene Therapy: Where Do We Go From Here?

Industry News & Research

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

Read More

Glanzmann’s Thrombasthenia and Bernard-Soulier Syndrome

GLHF News, Industry News & Research

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

Read More