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…
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…
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…
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…
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…
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…
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…
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…
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…
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…
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…
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…
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…
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…
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…
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…
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…
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…
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…
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…
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…
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…
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…
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…