Beginning on Feb. 29, Rare Disease Day, chapters from notable scientific books and clinical review articles covering rare disorders will be available free-of-charge from Elsevier. The offer runs through April 30, and aims to supports work by researchers and clinicians into a better understanding of and treatments for rare diseases, as well…
When given at low doses, extended half-life factor IX concentrates Alprolix (eftrenonacog alfa) and Idelvion (albutrepenonacog alfa) can prevent spontaneous bleeds in patients with severe hemophilia B, a study has found. The study, “A single centre retrospective study of low dose prophylaxis with…
Obesity increases the frequency of joint bleeds and degree of pain, and lowers overall life quality of people with hemophilia in the United States, most healthcare providers agreed in an online survey. But a sizable number, about 40%, did not discuss weight with patients during care…
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…
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…
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