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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

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

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

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

Stable FIX Expression and Durable Reductions in Bleeding and Factor IX Consumption for up to 4 Years Following AMT-060 Gene Therapy in Adults with Severe or Moderate-Severe Hemophilia B

Wolfgang Miesbach, MD PhD, Karina Meijer, Michiel Coppens, MD, Peter Kampmann, MD, Dr. Klamroth, Roger Schutgens, MD PhD MSc, Giancarlo Castaman, MD, Erhard Seifried, MD PhD, Joachim Schwaeble, Halvard Bönig, Eileen K Sawyer, PhD, Frank W.G. Leebeek, MD PhD

Blood (2019) 134 (Supplement_1): 2059.

https://doi.org/10.1182/blood-2019-122535

Published: 2019

Journal Articles

Prophylactic Treatment in People with Severe Hemophilia B in the US: An Analysis of Real-World Healthcare System Costs and Clinical Outcomes

Declan Noone, MSc, Gabriel Pedra, PhD, Sohaib Asghar, MSc, Jamie O'Hara, MSc, Eileen K Sawyer, PhD, Nanxin (Nick) Li, PhD

Blood (2019) 134 (Supplement_1): 2118.

https://doi.org/10.1182/blood-2019-126576

Published: 2019

Journal Articles

Impaired Bone Health and Wound Dehiscence in Carriers of Hemophilia a and B

Divyaswathi Citla Sridhar, MD, Yazan Abou-Ismail, MD, Sindhoosha Malay, MPH, PharmD, Sanjay P Ahuja, MD

Blood (2019) 134 (Supplement_1): 3448.

https://doi.org/10.1182/blood-2019-130846

Published: 2019

Journal Articles

Hemophilia A Dogs Tolerant to Human Factor VIII Provide a Unique Model to Determine Efficacy and Safety of AAV Delivery of Novel Factor VIII Variants

Giang N. Nguyen, BS, Lauren E. Wimsey, DVM, Elizabeth P. Merricks, PhD, Katherine P. Ponder, MD, Timothy C. Nichols, MD, Denise E. Sabatino, PhD

Blood (2019) 134 (Supplement_1): 3628.

https://doi.org/10.1182/blood-2019-126085

Published: 2019

Journal Articles

Evaluation of the Human Factor IX Gene Therapy Vector TAK-748 in Hemophilia: Results from Non-Clinical Studies in Factor IX Knockout Mice and Rhesus Monkeys

Markus Weiller, Helen Wang, Sogué Coulibaly, Maria Schuster, Hanspeter Rottensteiner, Kefeng Sun, Marinee K Chuah, Thierry Vandendriessche, Friedrich Scheiflinger, Werner Höllriegl

Blood (2019) 134 (Supplement_1): 4633.

https://doi.org/10.1182/blood-2019-124734

Published: 2019

Journal Articles

Design and Characterization of FLT210, a Potent Next Generation AAV-hFVIII Vector Candidate

Blood (2019) 134 (Supplement_1): 4638.

https://doi.org/10.1182/blood-2019-128490

Published: 2019

Journal Articles

Spontaneous Bleeding and Poor Bleeding Response with Extended Half-Life Factor IX Products: A Survey of Select US and Canadian Hemophilia Treatment Centers

Lynn M Malec, MD, Stacy E. Croteau, Michael Callaghan, MD, Davide Matino, MD MSc, Kenneth Dale Friedman, MD, Robert F. Sidonio, Jr., MD MSc

Blood (2019) 134 (Supplement_1): 2407.

https://doi.org/10.1182/blood-2019-126196

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

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

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

NARROW

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

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

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

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

Stable FIX Expression and Durable Reductions in Bleeding and Factor IX Consumption for up to 4 Years Following AMT-060 Gene Therapy in Adults with Severe or Moderate-Severe Hemophilia B

Prophylactic Treatment in People with Severe Hemophilia B in the US: An Analysis of Real-World Healthcare System Costs and Clinical Outcomes

Impaired Bone Health and Wound Dehiscence in Carriers of Hemophilia a and B

Hemophilia A Dogs Tolerant to Human Factor VIII Provide a Unique Model to Determine Efficacy and Safety of AAV Delivery of Novel Factor VIII Variants

Evaluation of the Human Factor IX Gene Therapy Vector TAK-748 in Hemophilia: Results from Non-Clinical Studies in Factor IX Knockout Mice and Rhesus Monkeys

Design and Characterization of FLT210, a Potent Next Generation AAV-hFVIII Vector Candidate

Spontaneous Bleeding and Poor Bleeding Response with Extended Half-Life Factor IX Products: A Survey of Select US and Canadian Hemophilia Treatment Centers

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)

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

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

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American Society of Hematology

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ASH Publications

American Society of Hematology

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