Area of research
Hematology · Genetics
Research interest
Research interests include Hemophilia Treatment and Research, Platelet Disorders and Treatments, Blood Coagulation and Thrombosis Mechanisms, and Coagulation, Bradykinin, Polyphosphates, and Angioedema.
Completion of phase 2b trial of etranacogene dezaparvovec gene therapy in patients with hemophilia B over 5 years
Natural history of preexisting AAV5 antibodies in adults with hemophilia B during the lead-in of the etranacogene dezaparvovec phase 3 study
Etranacogene dezaparvovec gene therapy for haemophilia B (HOPE-B): 24-month post-hoc efficacy and safety data from a single-arm, multicentre, phase 3 trial
Hereditary antithrombin deficiency pilot project registry from the American Thrombosis and Hemostasis Network
Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B
Direct oral anticoagulants in pediatric venous thromboembolism: Experience in specialized pediatric hemostasis centers in the United States
Effect of Anticoagulant Therapy for 6 Weeks vs 3 Months on Recurrence and Bleeding Events in Patients Younger Than 21 Years of Age With Provoked Venous Thromboembolism
Stable and durable factor IX levels in patients with hemophilia B over 3 years after etranacogene dezaparvovec gene therapy
Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A
Integrated Hemophilia Patient Care via a National Network of Care Centers in the United States: A Model for Rare Coagulation Disorders
Young adult outcomes of childhood prophylaxis for severe hemophilia A: results of the Joint Outcome Continuation Study
The prospective Hemophilia Inhibitor PUP Study reveals distinct antibody signatures prior to FVIII inhibitor development
Inhibitors and mortality in persons with nonsevere hemophilia A in the United States
Etranacogene dezaparvovec (AMT-061 phase 2b): normal/near normal FIX activity and bleed cessation in hemophilia B
Factor VIII: Long-established role in haemophilia A and emerging evidence beyond haemostasis
Assessments of pain, functional impairment, anxiety, and depression in US adults with hemophilia across patient‐reported outcome instruments in the Pain, Functional Impairment, and Quality of Life (P‐FiQ) study
Self‐reported prevalence, description and management of pain in adults with haemophilia: methods, demographics and results from the Pain, Functional Impairment, and Quality of life (P‐FiQ) study
Impact of pain and functional impairment in <scp>US</scp> adults with haemophilia: Patient‐reported outcomes and musculoskeletal evaluation in the pain, functional impairment and quality of life (P‐FiQ) study
Construct validity of patient-reported outcome instruments in US adults with hemophilia: results from the Pain, Functional Impairment, and Quality of life (P-FiQ) study
Genes Influencing the Development and Severity of Chronic ITP Identified through Whole Exome Sequencing
Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B
Impact of Acute Bleeding on Daily Activities of Patients with Congenital Hemophilia with Inhibitors and Their Caregivers and Families: Observations from the Dosing Observational Study in Hemophilia (DOSE)
Results from a large multinational clinical trial (guardian™1) using prophylactic treatment with turoctocog alfa in adolescent and adult patients with severe haemophilia <scp>A</scp>: safety and efficacy
A genome-wide association study of resistance to HIV infection in highly exposed uninfected individuals with hemophilia A
Variable response to propranolol treatment of kaposiform hemangioendothelioma, tufted angioma, and Kasabach–Merritt phenomenon
Effect of Acute Bleeding on Daily Quality of Life Assessments in Patients with Congenital Hemophilia with Inhibitors and Their Families: Observations from the Dosing Observational Study in Hemophilia