News
NHF Supports Senate Bill to Stop Spread of Short-Term Plans
Industry News & Research
NHF has applauded Senators Baldwin (D-WI) and Jones (D-AL) for introducing the No Junk Plans Act, which seeks to stop the spread of short-term health…
St. Jude/WFH to Bring Hemophilia B Gene Therapy Trials to Developing World
Industry News & Research
The St. Jude Children’s Research Hospital and the World Federation of Hemophilia (WFH) recently announced that they have entered into a unique…
Researchers Look at Clinical Outcomes in FXIII Babies
Industry News & Research
Individuals with factor XIII deficiency (FXIII), a very rare inherited bleeding disorder, may experience a number of symptoms including umbilical cord bleeding,…
Sigilon Releases Study Data on Hemophilia Cell Therapy
Industry News & Research
At the recent 2019 American Society of Gene and Cell Therapy Annual Meeting in Washington, DC, Sigilon Therapeutics presented data on an…
CHOP Researchers Study Factor VIIa Prophylaxis Through Gene Transfer
Industry News & Research
In a newly published study, researchers at the Children’s Hospital of Philadelphia (CHOP) described how they utilized gene transfer technology to illicit…
CMS Finalizes Policy to Limit Co-Pay Accumulator Programs
Industry News & Research
On April 18, 2019, the Centers for Medicare and Medicaid Services (CMS) released the final Notice of Benefit and Payment Parameters Rule…
Sangamo and Pfizer Announce Interim Trial Data for Hemophilia A Gene Therapy
Industry News & Research
Sangamo Therapeutics, Inc and Pfizer, Inc. recently announced interim data from the phase 1/2 Alta study designed to evaluate SB-525, the company’s…
New Financial and Patient Support Services for People Living With Hemophilia
Industry News & Research
The Patient Access Network (PAN) Foundation and the National Hemophilia Foundation (NHF) announced they are launching a new alliance to offer a…
New CME-Accredited Activity on Evaluating Patients with Suspected VWD
Industry News & Research
Register today for a new CME-accredited educational activity on “Recognizing and Remediating von Willebrand Disease – A Case Study in Collaborative Care,” provided by Haymarket…
Cell-based Gene Therapy May Offer Long-term Treatment for Hemophilia A, Mouse Study Suggests
Industry News & Research
Cell therapy with endothelial progenitor cells and stem cells genetically engineered to produce a functional clotting factor VIII (FVIII) may provide a…
uniQure Announces Updated Data from Phase 2B Dose Confirmation of AMT-061
Industry News & Research
uniQure is excited to announce that updated data from their Phase 2B dose-confirmation study of AMT-061, an investigational gene therapy for the…
NHF and Partners Announce Innovative Infusion Logging Tool
Industry News & Research
The National Hemophilia Foundation (NHF) has announced a new two-year pilot program that aims to improve the efficacy of patient infusion logs…
Online Health Information: Is It Reliable?
Industry News & Research
by: Paul Clement It’s an exciting time for people with inhibitors. In addition to bypassing agents, new products may prevent or control…
PT Study Focuses on Comprehensive Survey of Physical Activities Risks for 101 Activities
Industry News & Research
Physical therapists (PTs) at hemophilia treatment centers (HTCs) often consult with hemophilia patients about engaging in new physical activities like exercise, team/individual…
Catalyst Reports Positive Trial Updates for Subcutaneous Therapy for Hemophilia
Industry News & Research
Catalyst Biosciences, Inc recently reported updated interim data from their phase 2/3, multinational clinical study of marzeptacog alfa (MarzAA), a subcutaneous (administered…
Takeda Completes Acquisition of Shire
Industry News & Research
Takeda Pharmaceutical Company Limited recently announced the completion of its acquisition of Shire plc on January 8, 2019. The acquisition was approved…
Sangamo Announces First Patient Treated in Hemophilia B Gene Therapy Trial
Industry News & Research
Sangamo Therapeutics, Inc. recently announced it has treated the first patient in a Phase 1/2 clinical trial evaluating SB-FIX, the company’s investigational…
Two New MASAC Documents Issued
Industry News & Research
The National Hemophilia Foundation’s (NHF) Medical and Scientific Advisory Council has issued two new documents on November 21, 2018 that were approved…
Inhibitor Chapter Updated in Nurses’ Guide to Bleeding Disorders
Industry News & Research
The National Hemophilia Foundation’s (NHF’s) Nursing Working Group continues to revise and expand its Nurses’ Guide to Bleeding Disorders (NGBD). The latest chapter that has…
Results of Bayer’s PROTECT VIII Extension Study Announced
Industry News & Research
Bayer announced findings from an analysis of the PROTECT VIII extension study. This study found that long-term safety and efficacy was consistent…
Bioverativ and Sobi Release Long-Term Study Results for Extended Half-Life Therapies
Industry News & Research
Swedish Orphan Biovitrum AB (Sobi) and Bioverativ Inc. announced the results of ASPIRE and B-YOND, the most comprehensive long-term studies of extended…
Bayer Receives FDA Approval for Jivi, New Hemophilia A Treatment With Step-Wise Prophylaxis Dosing Regimen
Industry News & Research
Jivi’s extended half-life allows for twice-weekly initial dosing and may be adjusted to every five days and further individually adjusted to less…
They Thought Hemophilia Was a ‘Lifelong Thing.’ They May Be Wrong.
Industry News & Research
In a recent New York Times article by Gina Kolata, scientists are edging closer to defeating a longtime enemy of human health:…
UniQure Reports Successful Delivery of FIX Gene Despite Antibodies
Industry News & Research
UniQure presented new hemophilia B clinical trial data on May 19, 2018 at the American Society of Gene and Cell Therapy (ASGCT)…