News

The world’s first book on the world’s most commonly inherited bleeding disorder, by Laureen A. Kelley and Paul Clement, is back in…

Advocacy groups representing various disease communities voice concerns over Payer Matrix’s classification as a ‘Patient Advocacy Company.’ Read about their viewpoints and…

Takeda recently raised an issue with some BAXJECT® II reconstitution devices produced between October 2021 and January 2022 for use in conjunction…

In an effort to ensure that all copays count with insurance companies, and address the challenges of Copay Accumulator Adjustors, the Wisconsin…

“Insurance companies assert that restricting accumulator and maximizer programs will cause higher health insurance premiums for everyone, but that claim is false….

The US Food and Drug Administration (FDA) approved Roctavian, a one-time gene therapy for adults with severe hemophilia A who do not…

Reports from 26 States Show More than 1.5 M Beneficiaries Have Already Been Disenrolled. Wisconsin residents: Congress told states it was time…

Concern grows over the mounting tally of Medicaid coverage losses impacting people who remain eligible for the program. Read how federal regulators…

The new approval will allow consumers to purchase this oral contraceptive medication without a prescription at stores and online. The U.S. Food…

This paper takes a broad, holistic view of patient safety in the era of hemophilia gene therapy. After several decades of preclinical…

Yesterday, the U.S. Food and Drug Administration approved Roctavian (valoctocogene roxaparvovec), the first gene therapy for hemophilia A. This new treatment option, for the treatment of…

Pivotal data from the Phase 3 XTEND-Kids study evaluating ALTUVIIIO™ [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein] once-weekly prophylaxis, a factor VIII replacement…

BioMarin Pharmaceutical Inc. today announced the U.S. Food and Drug Administration approved ROCTAVIAN™ (valoctocogene roxaparvovec-rvox) gene therapy for the treatment of adults…

After several decades of preclinical and clinical research, pitfalls and progress, gene therapies for hemophilia A and B have become a reality…

that the call for applications for the Susan Skinner Memorial Fund (SSMF) Scholarship program is now open! Please spread the word among…

Early data on Medicaid “unwinding” shows an alarming rate of coverage losses for enrollees who may still be eligible. Colorado and Texas…

Authors of the review posit that reference intervals such as those utilized in thrombosis and hemostasis, are based on structural inequities. A…

HEMGENIX is indicated for the treatment of adults with hemophilia B who currently use factor IX prophylaxis therapy, or have current or…

Pfizer Inc. today announced their Phase 3 BASIS clinical trial evaluating marstacimab has met its primary endpoints, having demonstrated statically significant and…

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…

HEMGENIX®, the first and only FDA approved gene therapy for hemophilia B,has been proven to elevate and sustain factor IX levels for…

Dr. Leanord Valentino announces his impending retirement. His statement reflects on the progress and initiatives made during his three and a half…

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…

Marstacimab is a laboratory-engineered monoclonal antibody developed to treat hemophilia A and B patients, with or without inhibitors. Pfizer has announced positive…