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…
NHF Responds to Roe v. Wade Reversal
Advocacy & Legislation
NHF Statement in Response to the June 24, 2022 Supreme Court Ruling “The National Hemophilia Foundation (NHF) is aware of today’s Supreme…
Black Women with Bleeding Disorders Are Striving to Be Seen and Heard
Health and Well Being, Living with a Bleeding Disorder, Women with Bleeding Disorders
by: Andrea Collier Black women who have inheritable blood and bleeding disorders often face obstacles all along their journey—from diagnosis to treatment…
Parents in the Bleeding Disorders Community Share Their Best Advice
Health and Well Being, Living with a Bleeding Disorder
HemAware asked parents for their best advice on what has helped them navigate their children’s bleeding disorders in the first few years…
Ask a Social Worker: How Dangerous Is Cutting for Someone with Hemophilia?
Health and Well Being, Living with a Bleeding Disorder
Understanding the reasons why people self-harm, and what you can do to help. Ask a Social Worker is a monthly column featuring…
The Role of Pharmacy Benefits Managers (PBMs)
Advocacy & Legislation
Pharmacy benefits managers (PBMs) are middlemen that create and administer drug formularies on behalf of health insurers, self-insured employers, Medicaid, and other…
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…
My Biggest Fear Comes True for My Adult Son With Hemophilia
Health and Well Being, Living with a Bleeding Disorder
by: Cazandra Campos-MacDonald My biggest fear for my sons, who live with severe hemophilia, is an auto accident. When my oldest son, Julian, began to…
Managing Bleeding Disorders Requires Attention to Mental Health
Health and Well Being, Living with a Bleeding Disorder
by: Jennifer Lynne It’s easy to spot physical damage, such as swollen joints, nosebleeds, limps, and bruises, when one suffers from a…
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…
What Older People with Bleeding Disorders Need to Know About Osteoporosis
Health and Well Being, Living with a Bleeding Disorder
Compared with other groups, people with hemophilia have a higher risk of the disease. by: Kathryn Anne Stewart Osteoporosis, a disease of…