News

Starting a 501(c)(3) tax-exempt nonprofit isn’t easy, but the National Organization for Rare Disorders gave a few tips for those  looking to begin the complex process in its Feb. 20 webinar.  William Whitman,…

People with hemophilia scored better on assessments of psychological well-being than healthy adults, a study found. The finding adds to a growing body of evidence that people with chronic diseases like hemophilia are not only able to handle daily challenges, but also to thrive and grow through hardship. The…

The Institute for Gene Therapies (IGT) has launched, with the aim of maximizing the potential of gene therapies in genetic disorders such as hemophilia. Comprised of industry leaders, scientists, and patient advocates, the IGT’s overarching goal is to set the foundation for a modernized regulatory and reimbursement…

The U.S. Food and Drug Administration (FDA) has accepted — under priority review — a marketing application for Valrox (valoctocogene roxaparvovec), BioMarin Pharmaceutical’s investigational gene therapy for hemophilia A. This is the first time the FDA has accepted an application for a gene therapy specifically…

AscellaHealth and Audaire Health together are offering on a hemophilia management program that aims to containing prescription costs and help patients better manage their treatment needs. The program leverages AscellaHealth’s network of specialty pharmacy partners and Audaire’s healthcare technology to offer pharmacy network design, therapy utilization management, and clinical…

An abundance of events are afoot around the world to mark Rare Disease Day 2020 on Feb. 29. The activities are focused on heightening awareness about rare diseases and the hundreds of millions of individuals they are thought to affect. Patients, caregivers, and advocates worldwide will sport denim ribbons…

Takeda presented new real-world data highlighting the underdiagnosis of hemophilia A and B, supporting the effectiveness and safety of its hemophilia therapies — Advate, Adynovate, and Feiba — and discussing potential benefits of personalized therapies. The company presented these latest data at the 13th Annual Congress…

Light Chain Bioscience, a unit of Swiss biotech Novimmune, has completed the discovery phase of its collaborative program with Takeda for developing bispecific antibodies — protective proteins produced by the immune system — that mimic the activity of blood clotting factor VIII as a treatment for…

When administered at a high dose, a gene therapy for hemophilia B known as FLT180a seems able to restore the activity of clotting factor IX (FIX) within a normal range in men with moderate-to-severe disease, early data from Phase 1/2 trial show. The findings were announced in an oral presentation,…

Esperoct (turoctocog alfa pegol) is now available in the U.S. to treat and control bleeding in adults and children with hemophilia A, the therapy’s manufacturer, Novo Nordisk, announced. The treatment’s use was approved by the U.S. Food and Drug Administration (FDA) in February 2019, but its availability…