News
Recruitment Reopened for Phase 3 AFFINE Gene Therapy Trial
Industry News & Research
This trial is evaluating the efficacy and safety of a single infusion of an investigational gene therapy in individuals with moderately severe…
BioMarin Shares Community Update for Valoctocogene Roxaparvovec
Industry News & Research
Biomarin recently provided an “Update for the Hemophilia Community,” on the ongoing clinical trial program for its investigational hemophilia A gene therapy,…
Statement from Patient Groups in Response to Court Ruling on Cost-Free Coverage of Preventive Health Services
Industry News & Research, Living with a Bleeding Disorder
HFA and 25 other patient groups issued the following statement in response to Wednesday’s court ruling in Braidwood v. Becerra (a lawsuit formerly known…
Rare Factor X Deficiency Survey Shows Disparity in Care
Industry News & Research
Initial data from the survey were presented at the recent Thrombosis and Hemostasis Summit of North America and NHF’s Bleeding Disorders Conference…
FDA Grants Priority Review for Sanofi’s BIVV001
Industry News & Research
BIVV001 is a recombinant factor VIII therapy (rFVIII) developed for the prevention of bleeding episodes in hemophilia A patients via once weekly…
von Willebrand Disease (VWD) and Iron Deficiecy – Speaker: Bethany Samuelson, M.D.
Health and Well Being, Industry News & Research, Living with a Bleeding Disorder
In this video, Dr. Bethany Samuelson Bannow, of Oregon Health & Science University, discusses the role of iron in the body, the…
Gene Therapy and Bleeding Disorders: Making Sense of It All
Health and Well Being, Industry News & Research
Gene Therapy is definitely a dominant topic in the bleeding disorders community, with new articles being published regularly. Read some of the…
Supreme Court Decision and Genetic Testing
Health and Well Being, Industry News & Research
by: Laurie Kelley Thoughts on how the Supreme Court decision on abortion might affect genetic testing outcomes. Read more here. The recent…
Does Gene Therapy Face Uphill Battle? Gene Therapies for Hemophilia Treatment Will Face Strong Market Headwinds, Research Suggests
Industry News & Research
Solid market position of Hemlibra and strong prescriber interest in RNAi and TFPI treatment options, combined with patient concerns regarding gene therapies,…
Gene Therapy and Bleeding Disorders: Where Are We Now, and What’s on the Horizon?
Industry News & Research
The National Hemophilia Foundation’s 16th Workshop on Novel Technologies and Gene Transfer for Hemophilia, held in November, addressed these questions and more….
Hemophilia B— An Excellent Candidate for Gene Therapy
Industry News & Research
Current hemophilia B treatments offer strong protection from bleeds, but impact patient lifestyle and require consistent infusions to keep protection levels high.1…
Making Sense of It All – Gene Therapy
Industry News & Research
What exactly is a gene? What happens when genes mutate? And can gene therapy research help solve problems related to mutations? Learn…
Paper Features Q&A Resource on Shared Decision Making and Gene Therapy
Industry News & Research
The Q&A resource highlighted here is meant to encompass all stages of a patient’s decision making journey in gene therapy. As investigational…
BioMarin Presents Abstract on Vector DNA Transmission Risk at ISTH
Industry News & Research
The focus of the abstract is the investigational hemophilia A gene therapy, valoctocogene roxaparvovec. BioMarin recently shared findings from a phase 3…
The High Cost of Hemophilia
Advocacy & Legislation, Health and Well Being, Industry News & Research
Just one bleed in people with severe hemophilia A receiving prophylaxis has the potential to negatively affect health-related quality of life, and raise…
Prophylactic Therapy for Glanzmann Thrombasthenia Moves Towards Clinical Trials
Industry News & Research
Glanzmann Thrombasthenia is an ultra-rare inherited bleeding disorder that is characterized by poorly functioning platelets. The recent Congress of the International Society…
Sanofi Provides Fitusiran Trial Updates at ISTH
Industry News & Research
These updates were presented during the International Society on Thrombosis and Haemostasis (ISTH) Congress in London. Sanofi recently shared new clinical trial…
Novel Pilot Program to Focus on Genetic Diseases and Precision Medicine
Industry News & Research
A newly launched program is designed to advance a diagnostic and precision medicine approach in newborns. The Rady Children’s Institute for Genomic…
NHF Requests Risk Evaluation and Mitigation Strategy for Pending Gene Therapy Treatments
Industry News & Research
Read a citizen’s petition that was formally submitted to the Food and Drug Administration by NHF. On July 1, the National Hemophilia…
New Genotyping Recommendations Issued by MASAC
Industry News & Research
The new recommendation describes the proven benefits of genotyping people with hemophilia NHF’s Medical and Scientific Advisory Council (MASAC) has approved a…
Diabetes Risk in Hemophilia vs Unaffected Groups Compared in Retrospective Study
Industry News & Research, Living with a Bleeding Disorder
The hemophilia data for this study was drawn from de-identified medical records from four North American hemophilia treatment centers. The U.S. diabetes…
Bayer Announces Discontinuation of Kogenate® FS
Industry News & Research
Kogenate® was first approved by the U.S. Food and Drug Administration in 1993. Bayer has announced that they are discontinuing the recombinant…
FDA Grants Breakthrough Status to BIVV001
Industry News & Research
BIVV001 is an investigational recombinant factor VIII therapy (rFVIII) developed for the prevention of bleeding episodes in hemophilia A patients via once…
BioMarin Announces Delay in Application to FDA
Industry News & Research
Roctavian is Biomarin’s investigational hemophilia A gene therapy, which is currently in multiple ongoing clinical trials. BioMarin recently announced that their planned…