News
First Hemophilia B Patient Dosed with Gene Therapy Product
Industry News & Research
HEMGENIX is indicated for the treatment of adults with hemophilia B who currently use factor IX prophylaxis therapy, or have current or…
Pfizer Announces Positive Results from Hemophilia A and B Trial
Industry News & Research
Pfizer Inc. today announced their Phase 3 BASIS clinical trial evaluating marstacimab has met its primary endpoints, having demonstrated statically significant and…
HFA Launches Clinical Trials Search Site
Industry News & Research
Hemophilia Federation of America has launched a new resource and search feature for clinical trials for patients with bleeding disorders. “Patients and families have asked for…
CSL Behring Announces the First Patient has Received FDA ApprovedHEMGENIX® (etranacogene dezaparvovec-drlb) for Hemophilia B
Industry News & Research
HEMGENIX®, the first and only FDA approved gene therapy for hemophilia B,has been proven to elevate and sustain factor IX levels for…
Announcement from NHF CEO and President
Industry News & Research
Dr. Leanord Valentino announces his impending retirement. His statement reflects on the progress and initiatives made during his three and a half…
FDA Finalizes Blood Donor Eligibility Guidance
Industry News & Research
On May 11, 2023, the U.S. Food and Drug Administration released final guidance providing recommendations for evaluating blood and plasma donor eligibility using individual risk-based…
Pfizer Highlights Phase 3 Trial Results of Marstacimab
Industry News & Research
Marstacimab is a laboratory-engineered monoclonal antibody developed to treat hemophilia A and B patients, with or without inhibitors. Pfizer has announced positive…
Gene Therapy as a Novel Approach to Achieving ITI in FVIII Inhibitors Reviewed
Industry News & Research
The review provides a comprehensive overview of the persistent challenges and current mitigation strategies related to treating and managing hemophilia patients with…
FDA Finalizes Blood Donor Eligibility Guidance
Industry News & Research
On May 11, 2023, the U.S. Food and Drug Administration released final guidance providing recommendations for evaluating blood and plasma donor eligibility using individual risk-based…
Pfizer Highlights Phase 3 Trial Results of Marstacimab
Industry News & Research
Marstacimab is a laboratory-engineered monoclonal antibody developed to treat hemophilia A and B patients, with or without inhibitors. Pfizer has announced positive…
The Low Cost of Being Female with a Bleeding Disorder?
Health and Well Being, Industry News & Research, Living with a Bleeding Disorder
Female hemophilia A patients and carriers have more frequent bleeds than males, yet a smaller proportion receive factor to prevent or stop them, a U.S….
Now Enrolling: Own Your Path
Industry News & Research
Are you a male, 18-29 years old, and on a prophy regimen for hemophilia treatment? You may qualify for a new program!…
Four New Documents from MASAC
Industry News & Research
The new guidelines cover recommendations for physical therapy, gene therapy readiness, vaccine guidance, and treatment. Download here. Source: National Hemophilia Foundation
New in Research: Updates on the “My Life Our Future” Project
Industry News & Research
Learn how this project from NHF, the American Thrombosis and Hemostasis Network, Bloodworks Northwest, and Bioverativ/Sanofi helped inform people with hemophilia on…
Inhibitor Risk Paper Informed by Genotyping Project
Industry News & Research
Between 2013-2017, the “My Life Our Future” (MLOF) project offered eligible individuals with hemophilia free genotyping, which is historically hard to access,…
Important: Action Needed Regarding Medicaid and Badgercare Eligibility
Advocacy & Legislation, Industry News & Research
As you may have heard, the Medicaid and CHIP (called Badgercare in Wisconsin) programs are re-starting eligibility reviews. Community members may receive…
FDA Requests Additional Information on Concizumab from Novo Nordisk
Industry News & Research
The company received a Complete Response Letter from the FDA seeking more information relevant to their BLA for concizumab. Novo Nordisk has…
Updates on Generic Desmopressin Product
Industry News & Research
Desmopressin has the capacity to boost both factor VIII and von Willebrand factor levels. In July 2020 Ferring Pharmaceuticals suspended manufacturing of…
New Survey Spotlights Ongoing Concerns for People Living with Hemophilia B
Health and Well Being, Industry News & Research, Living with a Bleeding Disorder
Carefully controlled schedules. Undercurrents of uncertainty. Persistent pain. People who are living with hemophilia B, and those who love them, face a…
HTCs Participate in New CDC Community Counts Data Collection for COVID-19
COVID-19, Industry News & Research
Patients with bleeding orders may be asked some new questions during their next comprehensive clinic visit. COVID-19 information will now be collected…
Sanofi Announces Publication of Two Phase 3 Fitusiran Trials
Industry News & Research
Fitusiran is being investigated for the prophylactic treatment of adults and adolescents with hemophilia A or B. Sanofi recently announced that results…
Hemochromatosis Classification: Update and Recommendations by the BIOIRON Society
Industry News & Research
This is a related article to: Hemochromatosis redefined Abstract Hemochromatosis (HC) is a genetically heterogeneous disorder in which uncontrolled intestinal iron absorption may…
FDA approves once-weekly ALTUVIIIO™, a new class of factor VIII therapy for hemophilia A that offers significant bleed protection
Industry News & Research, Living with a Bleeding Disorder
FDA approves once-weekly ALTUVIIIO™, a new class of factor VIII therapy for hemophilia A that offers significant bleed protection Paris and Stockholm – February 23, 2023 – The U.S. Food and Drug Administration (FDA) has…
Introducing the WFH Gene Therapy Registry
Industry News & Research
The new registry will enhance knowledge of gene therapy and help keep patients safe. The World Federation of Hemophilia (WFH) is a nonprofit organization…