Hemophilia BNews & Research
15 curated articles for Hemophilia B — FDA updates, peer-reviewed research, clinical-trial milestones, and sponsor press releases. Sorted newest-first.
- Therapeutic advances in hematology May 8, 2026
Marstacimab for People with Severe Hemophilia A or Moderate to Severe Hemophilia B Without Inhibitors: A Plain Language Summary of Publication of the BASIS Study.
What is this summary about? This is a summary of the results from a clinical study of treatment for people with severe hemophilia A or moderately severe to severe hemophilia B without inhibitors. Because severe hemophilia A and B predominantly affect men and boys, this study only included men and bo...
Why it matters: Recent peer-reviewed research on Hemophilia B that may be relevant for patients and caregivers.
- Plastic and reconstructive surgery. Global open May 8, 2026
Occult Hemophilia B and Plastic Surgery: Preventing Bleeding Events.
Hemophilia, particularly Hemophilia B, is a rare bleeding disorder resulting from factor IX deficiency. Evolution in long-acting factor IX concentrates have enhanced surgical suitability. Due to inherent bleeding concerns and limited literature on hemophilia and other coagulopathies like von Willebr...
Why it matters: Recent peer-reviewed research on Hemophilia B that may be relevant for patients and caregivers.
- Thrombosis research Apr 17, 2026
Pseudoexon inclusion induced by three deep intronic variants in hemophilia B and correction achieved through an antisense oligonucleotide-based strategy.
Although studies have identified deep intronic variants associated with hemophilia B in patients undiagnosed by conventional genetic testing, knowledge in this field remains limited. Long range-PCR of entire F9 gene was used to screen variants of three unrelated genetically unresolved severe hemophi...
Why it matters: Recent peer-reviewed research on Hemophilia B that may be relevant for patients and caregivers.
- Blood advances Apr 15, 2026
Safety, efficacy and patient-reported outcomes 6 years after fidanacogene elaparvovec in adults with hemophilia B.
Fidanacogene elaparvovec is a single-dose gene therapy designed to express the high-activity factor IX (FIX) variant FIX-R338L. Participants (N=15) with FIX activity ≤2% were dosed with 5×1011 vector genomes/kg infusion of fidanacogene elaparvovec and completed the 1-year dosing trial. A...
Why it matters: Recent peer-reviewed research on Hemophilia B that may be relevant for patients and caregivers.
- Journal of thrombosis and haemostasis : JTH Apr 12, 2026
Diagnosis and management of hemophilia A and B.
Hemophilia A and hemophilia B are inherited bleeding disorders caused by a deficiency or absence of coagulation factor VIII (FVIII) and factor IX (FIX), respectively. The severity of bleeding manifestations generally correlates with the degree of factor deficiency, which is determined by the type of...
Why it matters: Recent peer-reviewed research on Hemophilia B that may be relevant for patients and caregivers.
- Thrombosis research Apr 11, 2026
Optimized AAVrh10 and Factor 9 vectors demonstrate improved phenotypic rescue in hemophilia B mice.
Hemophilia B is an X-linked bleeding disorder caused by a functional deficiency of coagulation factor IX (FIX). Adeno-associated virus (AAV) mediated gene therapy based on vector serotypes 5 and 8, has shown promising clinical outcomes. Nonetheless, a high prevalence of neutralizing antibodies in th...
Why it matters: Recent peer-reviewed research on Hemophilia B that may be relevant for patients and caregivers.
- Molecular biotechnology Apr 6, 2026
Transforming Hemophilia Management: Lessons from Gene Therapy Clinical Trials.
Gene therapy signifies a transformative revolution in hemophilia care, providing the possibility for sustained endogenous synthesis of coagulation factors and limiting the need for external factor supplementation. Preliminary experiments in hemophilia B via adeno-associated viral (AAV) vectors encou...
Why it matters: Recent peer-reviewed research on Hemophilia B that may be relevant for patients and caregivers.
- Expert review of clinical pharmacology Mar 16, 2026
Is marstacimab the next step in personalized and effective haemophilia management?
Is marstacimab the next step in personalized and effective haemophilia management?
Why it matters: Recent peer-reviewed research on Hemophilia B that may be relevant for patients and caregivers.
- Molecular therapy : the journal of the American Society of Gene Therapy Mar 7, 2026
Comparative evaluation of liver-directed knockin strategies with viral and nonviral vectors in mouse inherited disease models.
CRISPR-Cas9-mediated gene knockin has emerged as a promising strategy for early-onset genetic disease intervention. However, the therapeutic efficacy and editing outcomes of different knockin strategies remain incompletely understood. Here, we systematically evaluated three major liver-directed knoc...
Why it matters: Recent peer-reviewed research on Hemophilia B that may be relevant for patients and caregivers.
- Journal of blood medicine Mar 3, 2026
Current Status of Clinical Gene Therapy for Hemophilia and Globin Disorders.
In the last few years, gene therapy, holding the promise for long-term disease correction through a one-time treatment, has transitioned from experimental research to approved medicine. Several gene therapies are now available for congenital blood disorders, notably for hemophilia and hemoglobinopat...
Why it matters: Recent peer-reviewed research on Hemophilia B that may be relevant for patients and caregivers.
- Nature biotechnology Feb 17, 2026
Engineering a photoactivatable A-to-I RNA base editor for gene therapy in vivo.
Tunable and reversible regulation of exogenous and endogenous gene expression would be useful for improving the safety and efficacy of gene therapy. Current chemically inducible systems are limited by the rapid diffusion and extended metabolism of small molecules, and associated side effects. Here w...
Why it matters: Recent peer-reviewed research on Hemophilia B that may be relevant for patients and caregivers.
- Thrombosis and haemostasis Feb 5, 2026
Molecular Mechanisms of Factor IX Signal Peptide and Propeptide Mutations Underlying Hemophilia B.
Hemophilia B is a rare inherited bleeding disorder resulting from mutations in the coagulation factor IX (factor IX) gene. While mutations in factor IX catalytic domains directly compromise clotting activity, mutations in the signal peptide and propeptide domains contribute to disease pathogenesis t...
Why it matters: Recent peer-reviewed research on Hemophilia B that may be relevant for patients and caregivers.
- Therapeutic advances in hematology Jan 30, 2026
Nine areas with outstanding challenges for hemophilia B research.
Hemophilia A and B are rare, X-linked bleeding disorders characterized by deficiencies in coagulation factor VIII (FVIII) and factor IX (FIX), respectively. Numerous advances have helped to reduce disease burden. However, hemophilia B is not as well studied as hemophilia A, likely reflecting its low...
Why it matters: Recent peer-reviewed research on Hemophilia B that may be relevant for patients and caregivers.
- The New England journal of medicine Jan 28, 2026
Final Analysis of a Study of Etranacogene Dezaparvovec for Hemophilia B.
Prophylactic treatment for hemophilia B necessitates lifelong, regular intravenous factor IX infusions. Gene therapy offers the possibility of a single-dose treatment that produces durable endogenous factor IX expression and disease control. Etranacogene dezaparvovec comprises an adeno-associated vi...
Why it matters: Recent peer-reviewed research on Hemophilia B that may be relevant for patients and caregivers.
- Blood cells, molecules & diseases Nov 26, 2025
Effects of G-CSF and 5-FU as conditioning regimens in platelet-targeted gene therapy for hemophilia B.
Platelet-targeted gene therapy for hemophilia entails modifying a patient's hematopoietic stem cells (HSCs) ex vivo to produce platelets containing coagulation factors, offering a potential cure by localized factor release at injury sites. However, the associated bone marrow transplantation carries ...
Why it matters: Recent peer-reviewed research on Hemophilia B that may be relevant for patients and caregivers.
More on Hemophilia B
Articles aggregated from peer-reviewed journals (PubMed), sponsor press releases, SEC 8-K filings, and FDA announcements. Original-source links are preserved on each article page. Editorial tags (Breaking / Notable / Update) reflect UniteRare's curation-time priority assessment.