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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

In Vivo hemostatic Significance of Activated Protein C in Factor VIIIa Regulation

Amelia R. Wilhelm, B.S., Nicole A. Parsons, B.S., Charles T. Esmon, PhD, Rodney M. Camire, PhD, Lindsey A. George, MD

Blood (2019) 134 (Supplement_1): 93.

https://doi.org/10.1182/blood-2019-124493

Published: 2019

Journal Articles

Is Hemophilia B Clinically Less Severe Than Hemophilia a? Evidence from the Hugs Va and Vb Studies

Megan M. Ullman, MA, MPH, Randall Curtis, MBA, Mimi Lou, MS, Brenda Riske, MBA, MS, Judith Baker, DrPH, MHSA, Duc Quang Tran, Jr., MD, Joanne Wu, Michael B. Nichol, PhD

Blood (2019) 134 (Supplement_1): 58.

https://doi.org/10.1182/blood-2019-129439

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

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

Journal Articles

Efficacy and Safety in 15 Hemophilia B Patients Treated with the AAV Gene Therapy Vector Fidanacogene Elaparvovec and Followed for at Least 1 Year

Lindsey A. George, MD, Spencer K. Sullivan, MD, John E.J. Rasko, MBBS, PhD, Adam Giermasz, MD PhD, Benjamin J. Samelson-Jones, MD PhD, Jonathan M. Ducore, MD MPH, Jerome M. Teitel, MD FRCPC, Catherine E. McGuinn, MD, Alexa R. Runowski, MS, Fraser Wright, PhD, Xavier M. Anguela, PhD, Katherine A. High, MD, Denis Rybin, PhD, John E. Murphy, PhD, Jeremy Rupon, MD

Blood (2019) 134 (Supplement_1): 3347.

https://doi.org/10.1182/blood-2019-124091

Published: 2019

Journal Articles

Co-Prevalence of Pre-Existing Immunity to Different Serotypes of Adeno-Associated Virus (AAV) in Adults with Hemophilia

Kavitha Rajavel, Mila Ayash-Rashkovsky, Ying Tang, Bagirath Gangadharan, Maurus de la Rosa, Bruce Ewenstein

Blood (2019) 134 (Supplement_1): 3349.

https://doi.org/10.1182/blood-2019-123666

Published: 2019

Journal Articles

Adeno-Associated Virus Mediated Delivery of a TFPI Antibody Restores Hemostasis in Mouse Model of Hemophilia

Swapnil Rakhe, MS, Reema Jasuja, PhD, Rajani Shelke, Anagha Sawant, Suryanarayan Somanathan, PhD, John E. Murphy, PhD, Debra D Pittman, MS

Blood (2019) 134 (Supplement_1): 3357.

https://doi.org/10.1182/blood-2019-130054

Published: 2019

Journal Articles

Management of Major Surgical Procedures in Patients with Hemophilia: A Single-Center Experience of 49 Procedures

Veysel Suzan, MD, Nurgul Ozgur Yurttas, MD, Ahmet Emre Eskazan, MD, Deniz Ozmen, MD, Dilek Keskin, MD, Sevil Sadri, MD, Selin Berk, MD, Isil Erdogan, MD, Tugrul Elverdi, MD, Ayse Salihoglu, MD, Muhlis Cem Ar, MD PhD, Zafer Baslar, MD

Blood (2019) 134 (Supplement_1): 4933.

https://doi.org/10.1182/blood-2019-130305

Published: 2019

Journal Articles

Platelet Function Disorder Is More Common Than Von Willebrand Disease in Women with Inherited Bleeding Disorders: Report from a Public Sector University in Pakistan

Nazli Hossain, Sr., Tahir S. Shamsi, MBBS,FRCPath, Arshi Naz, Sidra Abbas, Jr., Nazeer Khan

Blood (2019) 134 (Supplement_1): 4937.

https://doi.org/10.1182/blood-2019-126454

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

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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

In Vivo hemostatic Significance of Activated Protein C in Factor VIIIa Regulation

Is Hemophilia B Clinically Less Severe Than Hemophilia a? Evidence from the Hugs Va and Vb Studies

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)

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

Efficacy and Safety in 15 Hemophilia B Patients Treated with the AAV Gene Therapy Vector Fidanacogene Elaparvovec and Followed for at Least 1 Year

Co-Prevalence of Pre-Existing Immunity to Different Serotypes of Adeno-Associated Virus (AAV) in Adults with Hemophilia

Adeno-Associated Virus Mediated Delivery of a TFPI Antibody Restores Hemostasis in Mouse Model of Hemophilia

Management of Major Surgical Procedures in Patients with Hemophilia: A Single-Center Experience of 49 Procedures

Platelet Function Disorder Is More Common Than Von Willebrand Disease in Women with Inherited Bleeding Disorders: Report from a Public Sector University in Pakistan

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

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