Project GYM: Physical Fitness Improves Wellbeing in Young Men With Hemophilia
Researchers investigated the safety and feasibility of Project GYM, a personal trainer-led physical fitness program for young men with hemophilia.
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Combination Regimen Highly Effective for Acquired Hemophilia A
Researchers sought to determine whether CyDRi would be a safe and effective treatment for patients with acquired hemophilia A.
Assay Developed for the Measurement of Anti-Factor VIII Antibodies in Patients With Hemophilia A
Researchers sought to develop an assay for measuring neutralizing inhibitors and non-neutralizing antibodies in patients with hemophilia A.
Physical Activity and Fitness Appears Lower Among Patients With Moderate to Severe Hemophilia
Researchers sought to determine the rates of physical activity and fitness in patients with moderate to severe hemophilia.
Stable Clinical Outcomes Seen With Octocog Alfa Prophylaxis in Hemophilia A
Researchers sought to determine whether prophylaxis with octocog alfa would lead to stable bleeding rates in patients with hemophilia A.
VWF Binding Aptamer Demonstrates Benefit in Type 2B Von Willebrand Disease
Researchers sought to determine whether rondoraptivon pegol would improve VWF and factor VIII levels in patients with type 2B von Willebrand disease.
FDA Accepts Resubmitted BLA for Valoctocogene Roxaparvovec for Hemophilia A
Valoctocogene roxaparvovec is administered as a single infusion to produce clotting factor VIII.
VWF Variants Predict Desmopressin Response Among Patients With Von Willebrand Disease
Researchers sought to determine whether the VWF gene variant would be predictive of desmopressin response in patients with VWD.
Non-Alcoholic Fatty Liver Disease Prevalent Among Patients With Hemophilia
Researchers sought to determine the prevalence of NAFLD and associated factors in patients with hemophilia.
Intranasal Desmopressin Compared With Exercise in Mild Hemophilia A
Researchers sought to compare exercise with intranasal desmopressin to examine changes in factor VIII activity in adolescent male patients with mild hemophilia A.
Valoctocogene Roxaparvovec BLA Resubmitted for Hemophilia A
Valoctocogene roxaparvovec is an investigational adeno-associated virus gene therapy.
Real-World Analysis Shows Emicizumab Is Safe, Effective in Pediatric Patients With Hemophilia A
Researchers sought to determine whether emicizumab would be effective in treating pediatric patients with hemophilia A.
Including a Clinical Pharmacists Beneficial to Hemophilia Care Team
Researchers sought to determine whether incorporating a clinical pharmacist into the comprehensive care model for patients with hemophilia would improve outcomes.
FLT180a May Improve Factor IX Levels in Patients With Hemophilia B
Researchers sought to determine whether gene therapy FLT180a would improve factor IX levels in patients with hemophilia B.
Emicizumab Prophylaxis Improves Joint Health in Patients With Hemophilia A
Researchers sought to determine whether there were improvements in joint health in patients with hemophilia A taking emicizumab prophylaxis therapy.
Study Compares Physical Activity Monitoring in Young Patients With Hemophilia
Researchers compared a consumer-grade activity monitor and a research-grade accelerometer for measuring activity in patients with hemophilia.
My Life, Our Future: Large Scale Genotyping Analysis of Patients With Hemophilia
Researchers sought to improve the understanding of hemophilia by conducting a wide-scale genotype analysis for patients in the United States.
New Study Characterizes Clinical and Financial Burden in Severe Hemophilia A
Researchers sought to determine the clinical and financial burden of patients with severe hemophilia A.
Rebinyn Approval for Hemophilia B Expanded to Include Routine Prophylaxis
Rebinyn is a recombinant DNA-derived coagulation Factor IX concentrate with an extended half-life.
Recombinant von Willebrand Factor Prophylaxis May Reduce ABR in Patients With von Willebrand Disease
Researchers sought to determine whether prophylaxis with recombinant VWF would lead to a decrease in annualized bleed rate in patients with VWD.
Outcomes With Extended Half-Life rFVIIIFc in Previously Untreated Hemophilia A
Researchers sought to determine whether extended half-life recombinant factor VIII would lead to a lower ABR in pediatric patients with severe hemophilia A.
Fibrinogen in Acute Posttraumatic Hypofibrinogenemia in Severe Brain Injury
In a randomized clinical trial, researchers evaluated clinical outcomes following fibrinogen administration in patients with severe TBI and concurrent hypofibrinogenemia.
ProvayBlue Now Available in New Single-Dose Vials for Methemoglobinemia
The new ProvayBlue presentation is supplied in a box of five 10mL single-dose vials; each vial contains 50mg of methylene blue.
Long-Term Analysis of SCT800 in Patients With Severe Hemophilia A
Researchers sought to assess SCT800 in patients with severe hemophilia A.
Older Patients With Hemophilia Experience Lower Quality of Life and More Comorbidities
Researchers sought to determine whether older patients with hemophilia would have similar quality of life scores and comorbidities compared with younger patients.
Circulating Extracellular Vesicles and Factor VIIa Treatment in Patients With Hemophilia
Researchers characterized the abundance and impacts of extracellular vesicles associated with recombinant factor VIIa treatment in patients with hemophilia.
Use of a Smart Medical Internet of Things System on Quality of Life in Patients With Hemophilia
Researchers sought to determine whether home nursing based on smart medical Internet of Things systems could improve the quality of life of patients with hemophilia.
Long-Term Prophylaxis Cost-Effective for Von Willebrand Disease
Researchers sought to determine whether long-term prophylaxis against bleeds in patients with von Willebrand disease would be cost effective.
Dental Caries and Other Aspects of Oral Health That Impact Patients With Hemophilia
Researchers sought to determine patterns of oral health in patients with hemophilia.
Plasma-Derived VWF Effective for Young Children With Severe Von Willebrand Disease
Researchers sought to determine whether plasma-derived VWF would be effective in treating hemorrhage in young children with severe VWD.
Trials Included in Many Coagulation Factor Package Inserts Have Significant Biases
Researchers sought to determine whether biases were present in clinical trials included in the package inserts of coagulation factors used for the treatment of hemophilia.
New Study Evaluating BPA Use With Emicizumab Among Patients With Hemophilia A
Researchers are seeking to evaluate outcomes after initiation of emicizumab and concomitant BPAs in patients with hemophilia A and FVIII inhibitors.
Extension Study Shows Safety, Efficacy of rVIII-SingleChain for Previously Treated Hemophilia A
Researchers sought to determine whether rVIII-SingleChain therapy would be safe and effective as a long-term prophylaxis option for previously treated patients with hemophilia A.
PRO Tool, GOAL-Hēm, Now More Patient-Centric for Patients With Hemophilia
Researchers sought to revise the GOAL-Hēm patient-reported outcomes tool to be more patient-centric for patients with hemophilia.
Efanesoctocog Alfa Appears Effective in Severe Hemophilia A
Researchers sought to determine whether efanesoctocog alfa may yield high sustained FVIII activity and improve bleed protection in patients with severe hemophilia A.
Valoctocogene Roxaparvovec Evaluated in Severe Hemophilia A
In open-label study, substantial increase seen in factor VIII activity at weeks 49 to 52 of follow-up.
Gene Therapy Gets Fast Track Status for Hemophilia A
In preclinical studies, ASC618 has been shown to have at least a 10-fold increase in the biosynthesis and secretion of FVIII compared with native human FVIII bioengineered constructs.
Reduced Cardiovascular Morbidity Seen in Patients With Hemophilia
Researchers sought to determine the incidence of fatal and nonfatal CVD during 5 years of follow up in adults with hemophilia.
Millennials With Hemophilia: Attitudes and Expectations From Treatment
Researchers sought to determine the attitudes and expectations of patients with hemophilia belonging to Generation Y regarding their treatment.
Perspectives on Participation in Sports in Patients With Hemophilia
Researchers sought to provide perspectives and recommendations for engaging patients with hemophilia in physical activity.
Durable Response Observed With Gene Therapy for Hemophilia A
The phase 3 GENEr8-1 study evaluated valoctocogene roxaparvovec in adults with severe hemophilia and residual FVIII levels less than or equal to 1 IU/dL.
Social Participation, Self-Perception, and Social Support in Boys With Hemophilia
Researchers sought to evaluate relationships between self-perception and social participation in boys with hemophilia.
Researchers Developed a Parenting Booklet on Physical Activity Restrictions and Recommendations in Pediatric Hemophilia
Researchers sought to develop a booklet for parents of children with hemophilia to help them manage physical activity restrictions based on the self-determination theory.
Care Delivery Model for Patients With Severe Hemophilia A in Tehran, Iran
Researchers sought to develop a care delivery model for patients with severe hemophilia A to improve on care and the use of resources.
Immune Tolerance Induction Therapy With Recombinant Antihemophilic Factor Seems Effective in Hemophilia A
Researchers sought to determine whether ITI therapy with ADVATE® would be safe and effective in patients with hemophilia A and neutralizing antibodies to coagulation factor VIII.
Experts Fail To Reach Consensus on Treatment of Hemophilia A
An expert panel sought to determine how to treat some patients with severe hemophilia A.
Magnetic Resonance Imaging of Joints Over Time in Boys With Severe Hemophilia A
Researchers sought to determine whether MRI would be useful in evaluating joint changes in boys with severe hemophilia.
Dronedarone Plus DOACs Increase Risk for Bleeding Events in AF
A study finds that dronedarone increased the risk for major bleeding events in patients with atrial fibrillation also taking a DOAC.
Strenuous Physical Activity Increases Bleed Risk in Patients With Hemophilia A
Researchers sought to determine whether patients with hemophilia A would be at higher risk of bleeding events when participating in high-collision physical activity.
Health Outcomes With Extended Vs Standard Half-Life Factor Products for Patients With Hemophilia
Researchers sought to determine whether switching to EHL factor concentrates would have an impact on health-related quality of life for patients with hemophilia.
Patient Care Provided by Hemophilia Treatment Centers in the United States
Researchers sought to summarize hemophilia treatment centers in the United States, how they function, and the benefits of this care approach.
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