It is critical that an individual with a bleeding disorder who presents in an emergency department (ED) be triaged and administered care as expeditiously as possible, as even briefs delays can have serious health implications for the patient. To…
How FDA is Putting the Patient Voice at the Forefront of Gene Therapy Clinical Trials for Hemophilia
The article following article is a summary of the impact of an FDA "Listening Session" on gene therapy for hemophilia.
By Najat Bouchkouj, MD, Bindu George, MD, and Karen Jackler, MPH
A person with hemophilia can experience aspects…
Novo Nordisk has announced the launch of ESPEROCT®, the company’s long-acting recombinant factor VIII product for the prevention and treatment of bleeding in individuals with hemophilia A. The new therapy is specifically indicated for routine…
Objective:
Hemophilia is marked by frequent joint bleeding, resulting in acute and chronic pain and functional impairment. Surveys in US adults with hemophilia demonstrate suboptimal pain management and quality of life (QoL). The…
For the 10th consecutive year, the National Hemophilia Foundation (NHF) has attained the prestigious 4-star rating from Charity Navigator, the largest independent charity evaluator in the US.
“Only two percent of the charities we evaluate have…
The National Hemophilia Foundation (NHF) has been monitoring the latest information on the current coronavirus (2019-nCoV) as it pertains to the bleeding disorders community.
The Plasma Protein Therapeutics Association (PPTA) has issued a…
The U.S. Food and Drug Administration (FDA) has released several new gene therapy guidance documents to provide “regulatory clarity” for developers of the many investigational therapies designed to treat any number of chronic conditions, including…
The National Hemophilia Foundation is pleased to endorse The Hemophilia SNF Access Act, critical legislation that will rectify a long-standing problem of Medicare beneficiaries with hemophilia and other bleeding disorders being able to access…
The National Hemophilia Foundation (NHF) and Hemophilia Federation of America (HFA) convened a Safety Summit in Washington, DC, on January 29-30th, 2020, to discuss monitoring, educating and communicating issues around bleeding disorders product…
Baltimore, MD (January 28, 2020) – The Green Park Collaborative (GPC) is partnering with the National Hemophilia Foundation (NHF) to develop a publicly available, content-validated, patient-reported outcome measure (PROM) that…