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Etranacogene dezaparvovec (AMT-061 phase 2b): normal/near normal FIX activity and bleed cessation in hemophilia B

Blood advances, 2019-11, Vol.3 (21), p.3241-3247

2019

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Stable and durable factor IX levels in patients with hemophilia B over 3 years after etranacogene dezaparvovec gene therapy

Blood advances, 2023-10, Vol.7 (19), p.5671-5679

2023

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Pharmacokinetic‐tailored approach to hemophilia prophylaxis: Medical decision making and outcomes

Research and practice in thrombosis and haemostasis, 2020-02, Vol.4 (2), p.326-333

2020

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Patient and Family Engagement during Treatment Change to Emicizumab at a Hemophilia Treatment Center

Blood, 2019-11, Vol.134 (Supplement_1), p.5796-5796

2019

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Decreased Skeletal Health Negatively Impacts Quality of Life of People with Hemophilia

Blood, 2018-11, Vol.132 (Supplement 1), p.1207-1207

2018

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Telephone assessment of stroke outcome is reliable

Stroke (1970), 2005-02, Vol.36 (2), p.232-233

2005

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Assessment of neonatal platelet adhesion, activation, and aggregation

Journal of thrombosis and haemostasis, 2016-04, Vol.14 (4), p.815-827

2016

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Durability of Bleeding Protection and Factor IX Activity Levels Are Demonstrated in Individuals with and without Adeno-Associated Virus Serotype 5 Neutralizing Antibodies (Titers <1:700) with Comparable Safety in the Phase 3 HOPE-B Clinical Trial of Etranacogene Dezaparvovec Gene Therapy for Hemophilia B

Blood, 2022-11, Vol.140 (Supplement 1), p.4904-4906

2022

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Adults with Severe or Moderately Severe Hemophilia B Receiving Etranacogene Dezaparvovec in the HOPE-B Phase 3 Clinical Trial Continue to Experience a Stable Increase in Mean Factor IX Activity Levels and Durable Hemostatic Protection after 24 Months’ Follow-up

Blood, 2022-11, Vol.140 (Supplement 1), p.4910-4912

2022

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Etranacogene Dezaparvovec (AAV5-Padua hFIX variant), an Enhanced Vector for Gene Transfer in Adults with Severe or Moderate-Severe Hemophilia B: Two Year Data from a Phase 2b Trial

Blood, 2020-11, Vol.136 (Supplement 1), p.13-13

2020

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Lack of Inhibitor Development in the American Thrombosis and Hemostasis Network (ATHN)-2 Factor Switching Study: Preliminary Report of Primary Outcome

Blood, 2019-11, Vol.134 (Supplement_1), p.1114-1114

2019

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One Year Data from a Phase 2b Trial of AMT-061 (AAV5-Padua hFIX variant), an Enhanced Vector for Gene Transfer in Adults with Severe or Moderate-Severe Hemophilia B

Blood, 2019-11, Vol.134 (Supplement_1), p.3348-3348

2019

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Dosing, Patient Satisfaction and Other Patient-Reported Outcomes after Switching to Rurioctocog Alfa Pegol in Athn 2: A Longitudinal, Observational Study of Previously Treated Hemophilia Patients Switching Coagulation Replacement Factor Products

Blood, 2020-11, Vol.136 (Supplement 1), p.17-18

2020

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First Data from the Phase 3 HOPE-B Gene Therapy Trial: Efficacy and Safety of Etranacogene Dezaparvovec (AAV5-Padua hFIX variant; AMT-061) in Adults with Severe or Moderate-Severe Hemophilia B Treated Irrespective of Pre-Existing Anti-Capsid Neutralizing Antibodies

Blood, 2020-11, Vol.136 (Supplement_2), p.LBA-6-LBA-6

2020

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Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: research priorities in health services; diversity, equity, and inclusion; and implementation science

Expert review of hematology, 2023-03, Vol.16 (sup1), p.87-106

2023

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Integrated Hemophilia Patient Care via a National Network of Care Centers in the United States: A Model for Rare Coagulation Disorders

Journal of blood medicine, 2021-01, Vol.12, p.897-911

2021

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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

The Lancet. Haematology, 2024-04, Vol.11 (4), p.e265-e275

2024

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Identification of Qualitative Platelet Disorders in Adolescent Women with Heavy Menstrual Bleeding

Blood, 2016-12, Vol.128 (22), p.4922-4922

2016

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Silent ischemic lesion recurrence on magnetic resonance imaging predicts subsequent clinical vascular events

Archives of neurology (Chicago), 2006-12, Vol.63 (12), p.1730

2006

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Stable and durable factor IX levels in hemophilia B patients over 3 years post etranacogene dezaparvovec gene therapy

Blood advances, 2022-12

2022

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Pilot study of novel lab methodology and testing of platelet function in adolescent women with heavy menstrual bleeding

Pediatric research, 2018-03, Vol.83 (3), p.693-701

2018

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Impact of establishing a primary stroke center at a community hospital on the use of thrombolytic therapy: the NINDS Suburban Hospital Stroke Center experience

Stroke (1970), 2003-06, Vol.34 (6), p.e55

2003

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Lift-off for early literacy: Directed reading opportunities for struggling students

Brookes Publishing Company, 2011

2011

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Blood donors in England and North Wales: demography and patterns of donation

Transfusion (Philadelphia, Pa.), 2015-01, Vol.55 (1), p.91-99

2015

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Etranacogene dezaparvovec (AMT-061 phase 2b): normal/near normal FIX activity and bleed cessation in hemophilia B

Stable and durable factor IX levels in patients with hemophilia B over 3 years after etranacogene dezaparvovec gene therapy

Pharmacokinetic‐tailored approach to hemophilia prophylaxis: Medical decision making and outcomes

Patient and Family Engagement during Treatment Change to Emicizumab at a Hemophilia Treatment Center

Decreased Skeletal Health Negatively Impacts Quality of Life of People with Hemophilia

Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B

Telephone assessment of stroke outcome is reliable

Assessment of neonatal platelet adhesion, activation, and aggregation

Adults with Severe or Moderately Severe Hemophilia B Receiving Etranacogene Dezaparvovec in the HOPE-B Phase 3 Clinical Trial Continue to Experience a Stable Increase in Mean Factor IX Activity Levels and Durable Hemostatic Protection after 24 Months’ Follow-up

Etranacogene Dezaparvovec (AAV5-Padua hFIX variant), an Enhanced Vector for Gene Transfer in Adults with Severe or Moderate-Severe Hemophilia B: Two Year Data from a Phase 2b Trial

Lack of Inhibitor Development in the American Thrombosis and Hemostasis Network (ATHN)-2 Factor Switching Study: Preliminary Report of Primary Outcome

One Year Data from a Phase 2b Trial of AMT-061 (AAV5-Padua hFIX variant), an Enhanced Vector for Gene Transfer in Adults with Severe or Moderate-Severe Hemophilia B

Dosing, Patient Satisfaction and Other Patient-Reported Outcomes after Switching to Rurioctocog Alfa Pegol in Athn 2: A Longitudinal, Observational Study of Previously Treated Hemophilia Patients Switching Coagulation Replacement Factor Products

First Data from the Phase 3 HOPE-B Gene Therapy Trial: Efficacy and Safety of Etranacogene Dezaparvovec (AAV5-Padua hFIX variant; AMT-061) in Adults with Severe or Moderate-Severe Hemophilia B Treated Irrespective of Pre-Existing Anti-Capsid Neutralizing Antibodies

Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: research priorities in health services; diversity, equity, and inclusion; and implementation science

Integrated Hemophilia Patient Care via a National Network of Care Centers in the United States: A Model for Rare Coagulation Disorders

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

Identification of Qualitative Platelet Disorders in Adolescent Women with Heavy Menstrual Bleeding

Silent ischemic lesion recurrence on magnetic resonance imaging predicts subsequent clinical vascular events

Stable and durable factor IX levels in hemophilia B patients over 3 years post etranacogene dezaparvovec gene therapy

Pilot study of novel lab methodology and testing of platelet function in adolescent women with heavy menstrual bleeding

Impact of establishing a primary stroke center at a community hospital on the use of thrombolytic therapy: the NINDS Suburban Hospital Stroke Center experience

Lift-off for early literacy: Directed reading opportunities for struggling students

Blood donors in England and North Wales: demography and patterns of donation

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