News
The Complexity of Unwinding Medicaid
Industry News & Research
The long-heralded Medicaid “unwinding” has begun. As previously reported, state Medicaid programs have, since the start of the COVID-19 pandemic, operated under a law that…
BioMarin Update: FDA Extends PDUFA Target Action Date to June 30, 2023
Industry News & Research
BioMarin Pharmaceutical Inc., a global biotechnology company dedicated to transforming lives through genetic discovery, announced that it received notice this afternoon from the…
The Importance of Copay Assistance Protections: Awareness Grows
Industry News & Research, Living with a Bleeding Disorder
For over three decades, March has been designated “Bleeding Disorders Awareness Month.” HFA and member organizations across the country mark the occasion…
FAQ: Proposed Changes to the FDA Blood Donation Policy
Industry News & Research
Answering Your Questions on the recent FDA blood donation policy revisions. Answering Your Questions on the Proposed Change to the FDA’s Blood…
Aminocaproic Acid Manufacturer Shuttered Though Not Expected to Trigger Shortage
Health and Well Being, Industry News & Research, Living with a Bleeding Disorder
Aminocaproic acid, which helps prevent the premature breakdown of blood clots, is often used to treat more minor bleeds in the nose…
Summit Emphasizes Pharmacovigilance in the Inherited Bleeding Disorders Community
Industry News & Research
The NHF/HFA Safety Summit yielded a series of recommendations related to product safety in the inherited bleeding disorders community. Biomedical and pharmaceutical…
uniQure Announces Published Trial Results for Approved Gene Therapy
Industry News & Research
These results were based on the multi-year HOPE-B Trial program. uniQure recently announced publication of the clinical trial results associated with approval…
New Medscape Activity to Help Institutions Offer Gene Therapy
Gene Therapy, Industry News & Research, Living with a Bleeding Disorder
NHF is pleased to announce that its collaboration with Medscape continues with a new accredited educational activity designed to help healthcare providers…
BioMarin Announces FDA’s Extended Review of Investigational Hemophilia Gene Therapy
Industry News & Research
ROCTAVIAN™ is currently in multiple ongoing clinical trials to observe its safety and efficacy in adult patients with severe hemophilia A. On…
What You Need to Know About Hemochromatosis
Industry News & Research, Living with a Bleeding Disorder
This blood disorder affects more than 1 million Americans and has no cure, but there are effective treatments. Hemochromatosis, also known as…
Study Suggests Significant Rates of Depression and Anxiety for Individuals with VWD
Industry News & Research
Findings from this study indicate that a relatively high proportion of participants with von Willebrand disease met the criteria for both depression…
Pfizer Announces Trial Updates for Investigational Hemophilia Gene Therapy
Industry News & Research
A primary focus of the trial was to compare annualized bleeding rates associated with a single infusion of the investigational gene therapy…
Takeda Announces Trial Results on Investigational Therapy for Ultra Rare Blood Disorder
Industry News & Research
TAK-755 is being investigated for the treatment of congenital thrombotic thrombocytopenic purpura. Takeda recently announced favorable results from a phase 3 study…
Biomarin Provides Hemophilia A Clinical Development Program Updates
Industry News & Research
BioMarin recently shared a community update on their Hemophilia A Clinical Development Program for valoctocogene roxaparvovec, the company’s investigational gene therapy currently…
WFH Gene Therapy Registry Goes Live
Industry News & Research, Living with a Bleeding Disorder
The new registry will help monitor the long-term safety and efficacy of hemophilia gene therapies in people around the world. The World…
Medicaid and CHIP Continuous Enrollment Unwinding
Advocacy & Legislation, Industry News & Research
Get information on your state’s plan to restart yearly Medicaid and Children’s Health Insurance Program (CHIP) eligibility reviews. Do you or a…
NHF Assumes Leadership of the American Plasma Users Coalition
Advocacy & Legislation, Industry News & Research
NHF Assumes Leadership of the American Plasma Users CoalitionThe organization’s public policy representatives offer new perspectives in the mission for blood and…
New FIX Chromogenic Assay Launched by Precision Biologics
Industry News & Research
This assay is designed to help determine factor IX activity levels in the plasma of individuals with hemophilia B who are two…
FDA Accepts BioMarin’s Biologics License Application (BLA) for Valoctocogene Roxaparvovec AAV Gene Therapy for Adults with Severe Hemophilia A
Industry News & Research
If Approved, Would Be 1st Gene Therapy in U.S. for Treatment of Severe Hemophilia A PDUFA Target Action Date is March 31, 2023….
Positive Gene Therapy Results
Industry News & Research
Pfizer had positive results from the Phase 3 BENEGENE-2 study evaluating fidanacogene elaparvovec, an investigational gene therapy, for the treatment of adult…
Common Questions About von Willebrand Disease (VWD)
Health and Well Being, Industry News & Research, Living with a Bleeding Disorder
It’s the most prevalent, yet lesser known, bleeding disorder. Here’s what you need to know about VWD. by: Michael Hickey Of the…
NHF Fellowship: 50 Years of Insights, Innovation, and Breakthroughs
Industry News & Research
NHF’s Judith Graham Pool Postdoctoral Research Fellowship program turns 50. We spoke with five awardees about how the fellowship shaped their careers….
Can People with Bleeding Disorders Take Blood Thinners?
Industry News & Research, Living with a Bleeding Disorder
by: Donna Behen Blood Safety Millions of Americans who have or are at high risk for heart disease regularly take anti-platelet drugs…
Telehealth for the Bleeding Disorders Community
Health and Well Being, Industry News & Research, Living with a Bleeding Disorder
Telemedicine provided vital access to blood and bleeding disorders care during the pandemic. What does the future hold for this technology? Remote…