News

Novel Pilot Program to Focus on Genetic Diseases and Precision Medicine

Industry News & Research

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

Read More

NHF Requests Risk Evaluation and Mitigation Strategy for Pending Gene Therapy Treatments

Industry News & Research

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

Read More

New Genotyping Recommendations Issued by MASAC

Industry News & Research

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

Read More

Diabetes Risk in Hemophilia vs Unaffected Groups Compared in Retrospective Study

Industry News & Research, Living with a Bleeding Disorder

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

Read More

Bayer Announces Discontinuation of Kogenate® FS

Industry News & Research

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

Read More

FDA Grants Breakthrough Status to BIVV001

Industry News & Research

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

Read More

BioMarin Announces Delay in Application to FDA

Industry News & Research

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

Read More

Honoring the Legacy of Dr. Charles Drew

GLHF News, Industry News & Research

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

Read More

GLHF Awarded Continued Cooperative Agreement from HRSA for the Northern States Region

GLHF News, Industry News & Research

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

Read More

Bone Health in VWD and Hemophilia Carriers the Focus of Retrospective Study

Industry News & Research

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

Read More

FDA Accepts Priority Review BLA for Hemophilia B Gene Therapy

Industry News & Research

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

Read More

Anxiety, Depression Need Better Management in Hemophilia: Survey

Health and Well Being, Industry News & Research, Living with a Bleeding Disorder

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…

Read More

New Data Shows Eptacog Beta Safe and Effective in Pediatric Patients

Industry News & Research

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

Read More

Meet Dr. Bosula Oluwole

Industry News & Research

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

Read More

Pfizer Reports First Quarter 2002 Update of Gene Therapy Trial

Industry News & Research

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…

Read More

New Data Shows Eptacog Beta Safe and Effective in Pediatric Patients

Industry News & Research

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

Read More

Bleeding Disorders A to Z: How Bleeding Disorders Are Diagnosed

Health and Well Being, Industry News & Research

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

Read More

Hemophilia History: The Romanov Family – Insights From Historian Dr. Helen Rappaport

Industry News & Research, Living with a Bleeding Disorder

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

Read More

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