News

Octapharma’s Wilate® Receives Expanded Approval from FDA

Industry News & Research

October 9, 2019 Octapharma recently announced that their plasma-derived von Willebrand Factor (VWF)/Factor VIII (FVIII) product Wilate®, was approved by the U.S….

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Genentech Issues Statement on Hemlibra Particles; MASAC Responds

Industry News & Research

October 7, 2019 Today, Genentech provided the following statement regarding reports of particulate matter in vials of Hemlibra. You can read Genentech’s…

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HFA and NHF Share an Update on Product Safety Conversation with the FDA

Industry News & Research

October 7, 2019 HFA and NHF are pleased to share this brief update with the community concerning a recent meeting we had with the…

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WFH Lead Research Points to Higher Global Hemophilia Prevalence

Industry News & Research

The World Federation of Hemophilia (WFH) recently announced the publication of a new study on the global prevalence of hemophilia, the results…

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NHF and HFA Letter to the Community

Industry News & Research

October 1, 2019 Recently there has been a lot of news related to the safety of the bleeding disorders community. HFA &…

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Introducing NHF’s Community-Powered Registry, MyBDC

Industry News & Research

The National Hemophilia Foundation (NHF) has created a community-powered registry for people with bleeding disorders, MyBDC. MyBDC will help researchers understand what…

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WFH Lead Research Points to Higher Global Hemophilia Prevalence

Industry News & Research

The World Federation of Hemophilia (WFH) recently announced the publication of a new study on the global prevalence of hemophilia, the results…

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Dedicated Hemotologist and Patient Advocate Dies at 95

Industry News & Research

August 12, 2019 saw the passing of longtime hematologist Shelby Lee Dietrich, MD at the age 95. Born in 1924, Dietrich spent…

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Bayer Responds to NHF-HFA Joint Statement

Industry News & Research

Bayer announced the recall of two lots of Kogenate® FS antihemophilic factor (recombinant) 2000 IU vials on July 19, 2019. Bayer announced that…

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Freeline Provides Updates on Hemophilia Gene Therapy Programs

Industry News & Research

The UK-based biotechnology company Freeline announced updates for its investigative gene therapies for hemophilia A and B at the recent XXVII Congress…

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Joint Statement on Recall of Bayer Kogenate® FS Lots

Industry News & Research

Last week, Bayer announced the recall of two lots of Kogenate® FS antihemophilic factor (recombinant) 2000 IU vials. That highly concerning announcement…

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Investigators Report Progress on New Subcutaneous Therapy

Industry News & Research

Results from a pair of phase 2 clinical trials of the investigational subcutaneous therapy, Concizumab were presented at the recent XXVII Congress…

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Casebia Presents Dual Approach to Gene Therapy

Gene Therapy, Industry News & Research, Living with a Bleeding Disorder

Cambridge, Massachusetts-based Casebia Therapeutics recently presented new research on a novel therapy being developed for patients with hemophilia A. Casebia Therapeutics is…

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Sanofi Presents Final Trial Data for rFVIII Candidate

Industry News & Research

Sanofi Genzyme reported positive data from their recently completed phase 1/2 EXTEN-A study of their investigational factor replacement product for hemophilia A….

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BioMarin to Submit Marketing Applications to FDA and EMA

Industry News & Research

BioMarin Pharmaceutical recently announced that they plan to submit marketing applications to both the European Medicines Agency (EMA) and the U.S. Food…

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uniQure Announces New Gene Therapy Trial Data

Industry News & Research

uniQure recently announced updated data related to a phase IIb clinical study of AMT-061, the company’s investigational gene therapy candidate for patients…

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Genentech Announces New Data from Hemlibra® Clinical Trial Program

Industry News & Research

Through a series of abstracts and oral presentations at the recent 2019 Congress of the International Society on Thrombosis and Haemostasis (ISTH),…

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Octapharma Presents Final Data on Personalized Therapy with Nuwiq®

Industry News & Research

Octapharma recently announced new data on the benefits of their recombinant factor VIII therapy Nuwiq® in patients with hemophilia A. Nuwiq® is…

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NHF Joins Organizations Urging Court to Protect ACA

Industry News & Research

Patient groups are urging the Fifth Circuit Court of Appeals to prioritize patient protections, including those for people with pre-existing conditions, when…

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Sangamo and Pfizer Announce Interim Gene Therapy Findings

Industry News & Research

In an oral presentation at the XXVII Congress of the International Society on Thrombosis and Haemostasis (ISTH), Sangamo Therapeutics, Inc and Pfizer,…

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Comparing Adherence and Physical Activity in Hemophilia A Patients

Industry News & Research

In a recently published study, a team of Italian investigators studied potential correlations between levels of physical activity and adherence to a…

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NHF-McMasters Guidelines on ECRI

Industry News & Research

The ECRI Institute, an independent, nonprofit patient safety organization, has published the NHF-McMaster University Guideline on Care Models for Haemophilia Management in the…

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Report Focuses on Two Cases of Sporadic CJD in the UK

Industry News & Research

Medscape Education recently launched a new activity, the focus of which is a report from the journal Emerging Infectious Diseases (EID) on surveillance as…

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BioMarin Provides Trial Updates for Hemophilia A Gene Therapy

Industry News & Research

BioMarin recently announced clinical trial updates relevant to valoctocogene roxaparvovec (formerly known as BMN 270) its investigational gene therapy for the treatment…

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