Page 1 | Search Results | Blood | American Society of Hematology

1-20 of 485

Sort by

Sort Order Select

Journal Articles

Gene therapy for hemophilia: a review on clinical benefit, limitations, and remaining issues

Frank W. G. Leebeek, Wolfgang Miesbach

Blood (2021) 138 (11): 923–931.

https://doi.org/10.1182/blood.2019003777

Published: 2021

Journal Articles

A new “FIX” for hemophilia B gene therapy

Graça Almeida-Porada

Blood (2021) 137 (21): 2860–2861.

https://doi.org/10.1182/blood.2021011753

Published: 2021

Journal Articles

Gene therapy for hemophilia B using CB 2679d-GT: a novel factor IX variant with higher potency than factor IX Padua

Nisha Nair, Dries De Wolf, Phuong Anh Nguyen, Quang Hong Pham, Ermira Samara-Kuko, Jeff Landau, Grant E. Blouse, Marinee K. Chuah, Thierry VandenDriessche

Blood (2021) 137 (21): 2902–2906.

https://doi.org/10.1182/blood.2020006005

Published: 2021

Includes: Supplemental data

Journal Articles

No CpGs for AAVs?

Lindsey A. George

Blood (2021) 137 (6): 721–723.

https://doi.org/10.1182/blood.2020009285

Published: 2021

Journal Articles

BAX 335 hemophilia B gene therapy clinical trial results: potential impact of CpG sequences on gene expression

Barbara A. Konkle, Christopher E. Walsh, Miguel A. Escobar, Neil C. Josephson, Guy Young, Annette von Drygalski, Scott W. J. McPhee, R. Jude Samulski, Ivan Bilic, Maurus de la Rosa, Birgit M. Reipert, Hanspeter Rottensteiner, Friedrich Scheiflinger, John C. Chapin, Bruce Ewenstein, Paul E. Monahan

Blood (2021) 137 (6): 763–774.

https://doi.org/10.1182/blood.2019004625

Published: 2021

Includes: Supplemental data

Journal Articles

Antithrombin- and Heparin-Binding Exosites Regulate Extravascular Binding and Clearance of Factor IXa

Pamela Westmark, PhD, John P. Sheehan, MD

Blood (2019) 134 (Supplement_1): 2392.

https://doi.org/10.1182/blood-2019-131580

Published: 2019

Journal Articles

Pharmacoeconomic Analysis of Ideal Vs. Actual Body Weight-Based Treatment for Adult Persons with Hemophilia Α

Ming Y. Lim, MBBChir,MA, Jeffrey A. Gilreath, PharmD, Paul F. Bray, MD, George M. Rodgers, MD PhD

Blood (2019) 134 (Supplement_1): 4684.

https://doi.org/10.1182/blood-2019-121679

Published: 2019

Journal Articles

Long-Term AAV-Mediated Factor VIII Expression in Nine Hemophilia A Dogs: A 10 Year Follow-up Analysis on Durability, Safety and Vector Integration

Giang N. Nguyen, BS, John K. Everett, PhD, Hayley Raymond, BA, Samita Kafle, BS, Elizabeth P. Merricks, PhD, Haig H. Kazazian, MD, Timothy C. Nichols, MD, Frederic D. Bushman, PhD, Denise E. Sabatino, PhD

Blood (2019) 134 (Supplement_1): 611.

https://doi.org/10.1182/blood-2019-126007

Published: 2019

Journal Articles

Fitusiran, an RNAi Therapeutic Targeting Antithrombin to Restore Hemostatic Balance in Patients with Hemophilia a or B with or without Inhibitors: Management of Acute Bleeding Events

Steven Pipe, MD, Margaret V. Ragni, MD MPH, Claude Négrier, MD PhD, Qifeng Yu, PhD, Naghmana Bajwa, MD, John Caminis, MD, Baisong Mei, MD PhD, Shauna R. Andersson, MD PhD

Blood (2019) 134 (Supplement_1): 1138.

https://doi.org/10.1182/blood-2019-124108

Published: 2019

Journal Articles

Estimating the Risk of Myocardial Infarction in Persons with Hemophilia A Using a Machine-Learning Approach with US Claims Data

Imi Faghmous, MPH, Carlos Flores, MPH, Kati Sarouei, MD, Tiffany Y. Chang, MD MAS, Anisha M. Patel, PhD, Cami S. Sima, MD, Peter Kuebler, PharmD

Blood (2019) 134 (Supplement_1): 1133.

https://doi.org/10.1182/blood-2019-123761

Published: 2019

Journal Articles

Toll-like Receptor 9 Activation Accelerates Inhibitor Formation in Response to Factor VIII

Radoslaw Kaczmarek, Alexandra Sherman, DVM, Moanaro Biswas, PhD, Roland W Herzog, PhD

Blood (2019) 134 (Supplement_1): 1113.

https://doi.org/10.1182/blood-2019-125348

Published: 2019

Journal Articles

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

Robert F. Sidonio, Jr., MD MSc, Dunlei Cheng, PhD, Christine Guelcher, PPCNP-BC, Janna M. Journeycake, MD, Susan U Lattimore, Clifford Takemoto, MD, Michael D. Tarantino, MD, Crystal Watson, Ellis J. Neufeld, MD, Carol Fedor, RN

Blood (2019) 134 (Supplement_1): 1114.

https://doi.org/10.1182/blood-2019-128612

Published: 2019

Journal Articles

Low-Grade Inflammation in Hemophilia

Lynn M Knowles, PhD, Hermann Eichler, Jan Pilch, MD

Blood (2019) 134 (Supplement_1): 1115.

https://doi.org/10.1182/blood-2019-125127

Published: 2019

Journal Articles

Impact of Systematic Pharmacokinetic (PK) Profiles in a Cohort of 29 Patients Treated with Conventional and Extended-Half Life (EHL) Products

Teresa Ceglie, Berardino Pollio, Irene Ricca, Maria Messina, Claudia Linari, Mauro Pagliarino

Blood (2019) 134 (Supplement_1): 1123.

https://doi.org/10.1182/blood-2019-132199

Published: 2019

Journal Articles

Efficacy and Safety of Subcutaneous Prophylaxis with Concizumab in Patients with Severe Hemophilia a without Inhibitors: Results from the Phase 2 explorer5 Trial

Hermann Eichler, Pantep Angchaisuksiri, MD, Jan Astermark, MD PhD, Gary Benson, Katarina Cepo, Pratima Chowdary, MD, Ute Friedrich, PhD, Kaan Kavakli, Allison P Wheeler, MD, Johannes Oldenburg, MD PhD

Blood (2019) 134 (Supplement_1): 2417.

https://doi.org/10.1182/blood-2019-122300

Published: 2019

Journal Articles

Real-World Clinical Management of Patients with Hemophilia and Inhibitors: Effectiveness and Safety of aPCC in Patients with >18 Months' Follow-up in the FEIBA Global Outcome Study (FEIBA GO)

Jerzy Windyga, Pal A Holme, C. Hermans, Ana R Cid, Nirjhar Chatterjee, Hongyu Jiang, Viridiana Cano-Garcia, Carmen Escuriola

Blood (2019) 134 (Supplement_1): 2418.

https://doi.org/10.1182/blood-2019-124324

Published: 2019

Journal Articles

Immediate and Long-Term Outcomes Following Orthopedic Procedures in Patients with Hemophilia: A Three-Decade Retrospective Review

CHU Yang Lin, MD, Haowei Sun, MD

Blood (2019) 134 (Supplement_1): 5874.

https://doi.org/10.1182/blood-2019-123207

Published: 2019

Journal Articles

Clearance of Vector DNA Following Systemic Administration of AAV5-hFIX or AAV5-hFIX Padua in Patients with Severe or Moderate-Severe Hemophilia B

Eileen K Sawyer, PhD, Sander Gielen, PhD, Jaap Twisk, Alison Long, Robert Gut

Blood (2019) 134 (Supplement_1): 2062.

https://doi.org/10.1182/blood-2019-129786

Published: 2019

Journal Articles

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

Steven Pipe, MD, Adam Giermasz, MD PhD, Giancarlo Castaman, MD, Nigel S. Key, MBChB, FRCP, Susan U Lattimore, Frank Leebeek, MD PhD, Wolfgang Miesbach, MD PhD, Michael Recht, MD PhD, Esteban Gomez, MD, Alison Long, Robert Gut, Annette von Drygalski, MD PharmD

Blood (2019) 134 (Supplement_1): 3348.

https://doi.org/10.1182/blood-2019-128765

Published: 2019

Journal Articles

Seroprevalence of Pre-Existing Nabs Against AAV1, 2, 5, 6 and 8 in South African Hemophilia B Patient Population

Anna Majowicz, PhD, Nolukholo Ncete, MBBCH, Floris van Waes, MSc, Nikki Timmer, BSc, Sander J van Deventer, MD PhD, Johnny N Mahlangu, MBBCH, MMed, FCPath, Valerie Ferreira, PhD

Blood (2019) 134 (Supplement_1): 3353.

https://doi.org/10.1182/blood-2019-128217

Published: 2019

Update search

NARROW

Publications

Format

Subjects

Article Type

Topics

Date

Availability

Gene therapy for hemophilia: a review on clinical benefit, limitations, and remaining issues

A new “FIX” for hemophilia B gene therapy

Gene therapy for hemophilia B using CB 2679d-GT: a novel factor IX variant with higher potency than factor IX Padua

No CpGs for AAVs?

BAX 335 hemophilia B gene therapy clinical trial results: potential impact of CpG sequences on gene expression

Antithrombin- and Heparin-Binding Exosites Regulate Extravascular Binding and Clearance of Factor IXa

Pharmacoeconomic Analysis of Ideal Vs. Actual Body Weight-Based Treatment for Adult Persons with Hemophilia Α

Long-Term AAV-Mediated Factor VIII Expression in Nine Hemophilia A Dogs: A 10 Year Follow-up Analysis on Durability, Safety and Vector Integration

Fitusiran, an RNAi Therapeutic Targeting Antithrombin to Restore Hemostatic Balance in Patients with Hemophilia a or B with or without Inhibitors: Management of Acute Bleeding Events

Estimating the Risk of Myocardial Infarction in Persons with Hemophilia A Using a Machine-Learning Approach with US Claims Data

Toll-like Receptor 9 Activation Accelerates Inhibitor Formation in Response to Factor VIII

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

Low-Grade Inflammation in Hemophilia

Impact of Systematic Pharmacokinetic (PK) Profiles in a Cohort of 29 Patients Treated with Conventional and Extended-Half Life (EHL) Products

Efficacy and Safety of Subcutaneous Prophylaxis with Concizumab in Patients with Severe Hemophilia a without Inhibitors: Results from the Phase 2 explorer5 Trial

Real-World Clinical Management of Patients with Hemophilia and Inhibitors: Effectiveness and Safety of aPCC in Patients with >18 Months' Follow-up in the FEIBA Global Outcome Study (FEIBA GO)

Immediate and Long-Term Outcomes Following Orthopedic Procedures in Patients with Hemophilia: A Three-Decade Retrospective Review

Clearance of Vector DNA Following Systemic Administration of AAV5-hFIX or AAV5-hFIX Padua in Patients with Severe or Moderate-Severe Hemophilia B

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

Seroprevalence of Pre-Existing Nabs Against AAV1, 2, 5, 6 and 8 in South African Hemophilia B Patient Population

Email alerts

ASH Publications

American Society of Hematology

Update search

Email alerts

ASH Publications

American Society of Hematology

This Feature Is Available To Subscribers Only