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…
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…
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…
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…
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…
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…
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…
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…
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…
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…
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…
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…
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…
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….
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…
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…
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…
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…
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…