News

BioMarin is pleased to update the community regarding our ongoing gene therapy clinical trial program in hemophilia A. BioMarin’s investigational gene therapy…

The American Rescue Plan (ARP), signed into law on March 11, 2021, provides new opportunities to get health insurance coverage, and/or to…

Final analysis from the Phase IIIb STASEY study, including data from 193 people with hemophilia A, further support the benefit/risk profile of…

Sigilon’s cell-based therapy is designed to carry significant amounts of synthetic DNA material to elicit the production of factor VIII in individuals…

Read about some of the primary considerations relevant to investigational hemophilia gene therapies that employ adeno-associated viral (AAV) vectors. Source: National Hemophilia…

by: Sen. André Jacque and Rep. Paul Tittl | April 17, 2021 The health and economic impacts of the ongoing pandemic have…

NHF’s Annual Bleeding Disorders Conference (BDC) is their signature event and highlights their commitment to education. Leading community members and experts on…

The verdict is an important and significant step forward in the fight against racism, and the violence that it brings to our…

The COVID-19 Task Force of the World Federation of Hemophilia (WFH) has released a statement on risks of acquiring COVID-19 disease and…

These data were presented by BioMarin at the American Society of Gene & Cell Therapy Virtual Meeting, which was held May 11-14,…

The recent American Society of Gene & Cell Therapy (ASGCT) Virtual Meeting featured updates from the phase III HOPE-B clinical trial of…

June 3, 2021 – Statement from Nathan Schaefer, Vice President of Public Policy, National Hemophilia Foundation This June marks a painful anniversary…

Time for an important reminder – “Scholarship season” is now in full swing! NHF’s information service, HANDI, compiles an annual online list…

In this video, Robert Sidonio Jr., M.D., discusses the new von Willebrand Disease (VWD) Guidelines and the most significant changes that healthcare…

We are marking women’s health month with a series of webinars that focus on critical issues for women with bleeding disorders. Our…

This national survey is designed to assess patient demographics, their satisfaction with the HTC’s core treatment team, its services, and care processes….

Neuraxial anesthesia, a focus of this study, refers to the administration of local anesthetics and analgesia to targeted locations in the spinal…

The commercialization and licensing agreement between the two companies went into effect on May 6th. May 7, 2021 uniQure recently announced that…

Kevin Mills, PhD has joined the National Hemophilia Foundation as chief scientific officer. A graduate of University of Colorado Boulder, MIT, and…

Late last year the FDA put a clinical hold on the program in response to a single trial participant who was diagnosed…

Biochemist Gordon Vehar, PhD, vice president of external innovation at BioMarin Pharmaceutical Inc., has been at the forefront of a scientific revolution….

You have been reaching out to us with your questions and concerns about the COVID-19 vaccines and we are grateful that you…

The Platelet Disorder Support Association recently recorded a patient education webinar on the new COVID-19 vaccines and immune thrombocytopenic purpura (ITP) As…

The focus of the HOPE-B trial is etranacogene dezaparvovec, uniQure’s investigational gene therapy candidate for individuals with severe and moderately severe hemophilia…