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Journal Articles

Interim Safety and Efficacy Results from a Phase 1/2 Study of Zinc Finger Nuclease-Modified Autologous Hematopoietic Stem Cells for Sickle Cell Disease (PRECIZN-1)

Asif Alavi, Mehrdad Abedi, Suhag Parikh, Richard Boismenu, Michael Chen, Ben L. Hsu, Bettina M. Cockroft, Isobelle Galeon, Pablo Rendo, Mark C. Walters

Blood (2022) 140 (Supplement 1): 4907–4909.

https://doi.org/10.1182/blood-2022-163725

Published: 2022

Journal Articles

Quantitative Systems Pharmacology Model of Sickle Cell Disease and Response to Gene Editing Therapy to Support Clinical Development of SAR445136 (BIVV003)

Chanchala Kaddi, Danielle Holz, Mengdi Tao, Isobelle Galeon, David Reiner, Pablo Rendo, Susana Zaph

Blood (2021) 138 (Supplement 1): 1860.

https://doi.org/10.1182/blood-2021-147330

Published: 2021

Journal Articles

Prophylaxis with rFIXFc Reduces the Frequency and Delays Time to First Spontaneous Bleed Event in Previously Untreated Patients with Hemophilia B: A Post Hoc Analysis of the PUPs B-LONG Trial

Beatrice Nolan, Michael Recht, Pablo Rendo, Aletta Falk, Meredith Foster, Sandra Casiano, Antoine Rauch, Amy D. Shapiro

Blood (2021) 138 (Supplement 1): 498.

https://doi.org/10.1182/blood-2021-148765

Published: 2021

Journal Articles

Preliminary Safety and Efficacy Results from Precizn-1: An Ongoing Phase 1/2 Study on Zinc Finger Nuclease-Modified Autologous CD34+ HSPCs for Sickle Cell Disease (SCD)

Asif Alavi, Lakshmanan Krishnamurti, Mehrdad Abedi, Isobelle Galeon, David Reiner, Sharon E Smith, Lin Wang, Anne Ramezi, Pablo Rendo, Mark C. Walters

Blood (2021) 138 (Supplement 1): 2930.

https://doi.org/10.1182/blood-2021-151650

Published: 2021

Journal Articles

Zinc Finger Nuclease-Mediated Disruption of the BCL11A Erythroid Enhancer Results in Enriched Biallelic Editing, Increased Fetal Hemoglobin, and Reduced Sickling in Erythroid Cells Derived from Sickle Cell Disease Patients

Samuel Lessard, PhD, Pauline Rimmele, PhD, Hui Ling, PhD, Kevin Moran, MSc, Benjamin Vieira, BS, Yi-Dong Lin, PhD, Vu Hong, PhD, Andreas Reik, PhD, Denny Dang, Naoya Uchida, MD, John F. Tisdale, MD, Pablo Rendo, MD, Ahmed Daak, PhD, Alexandra Hicks, PhD

Blood (2019) 134 (Supplement_1): 974.

https://doi.org/10.1182/blood-2019-126048

Published: 2019

Journal Articles

Impact of Treatment Regimen with Moroctocog Alfa (AF-CC) on Bleeding Frequency in Pediatric Aged Subjects with Moderately Severe to Severe Hemophilia A

Mark P. Smith, MD FRACP, FRCPA, Jeremy Rupon, MD, Yasser Wali, MD, Hala Remawi, MD, Joan Korth-Bradley, PharmD, PhD, Lynne Smith, MBA, Pablo Rendo, MD

Blood (2018) 132 (Supplement 1): 1205.

https://doi.org/10.1182/blood-2018-99-111891

Published: 2018

Journal Articles

An Open-Label Study of the Safety and Efficacy of Morotocog Alfa (AF-CC) in Previously Untreated Patients Aged <6 Years in Usual Care Settings

Kaan Kavakli, MD, James Baumann, Francois Pierre Huard, MS, MBA, Lynne Smith, MBA, Christine Alvey, Joan Korth-Bradley, PharmD, PhD, Pablo Rendo, MD, Jeremy Rupon, MD

Blood (2017) 130 (Supplement 1): 1091.

https://doi.org/10.1182/blood.V130.Suppl_1.1091.1091

Published: 2017

Journal Articles

Once-Weekly Prophylactic Treatment Versus on-Demand Treatment of Nonacog Alfa in Patients with Moderately Severe to Severe Hemophilia B

Kaan Kavakli, Lynne Smith, Kazimierz Kuliczkowski, MD PhD Prof., Joan Korth-Bradley, Chur Woo You, Joanne Fuiman, Silva Zupancic-Salek, Faraizah Abdul Karim, Pablo Rendo

Blood (2014) 124 (21): 1523.

https://doi.org/10.1182/blood.V124.21.1523.1523

Published: 2014

Journal Articles

Nonacog Alfa: Analysis Of Safety Data From 6 Prospective Clinical Studies In Different Patient Populations With Hemophilia B Treated With Different Therapeutic Modalities

Pablo Rendo, MD, Lynne Smith, MBA, Lee Hsiao-Yu, Frank E. Shafer, MD

Blood (2013) 122 (21): 3604.

https://doi.org/10.1182/blood.V122.21.3604.3604

Published: 2013

Journal Articles

Lack of Seasonal Variation in Bleeding and Patient-Assessed Pain Patterns in Patients with Hemophilia B Receiving On-Demand Therapy

Frank E. Shafer, MD, Lynne Smith, MBA, Nicholas Vendetti, MS, Pablo Rendo, MD, Marcus E. Carr, MD

Blood (2012) 120 (21): 1136.

https://doi.org/10.1182/blood.V120.21.1136.1136

Published: 2012

Journal Articles

Evaluation of Two Secondary Prophylaxis Regimens of Recombinant Factor IX(r-IX) in Moderately Severe to Severe( FIX ≤2%) Hemophilia B Patients

Pablo Rendo, MD, Mary-Kate Barrette-Grischow, MA, MPH, Lynne Smith, MBA, JM Korth-Bradley, Robert Charnigo, Frank E. Shafer, MD

Blood (2012) 120 (21): 4628.

https://doi.org/10.1182/blood.V120.21.4628.4628

Published: 2012

Journal Articles

Pharmacokinetic/Pharmacodynamic Assessment of Reformulated Recombinant Coagulation Factor IX in Adults and Children with Severe Hemophilia B

JM Korth-Bradley, Leonard A. Valentino, MD, FAAP, Pablo Rendo, MD, Frank E. Shafer, MD, Lynne Smith, James A. Baumann, Robert Charnigo

Blood (2011) 118 (21): 1190.

https://doi.org/10.1182/blood.V118.21.1190.1190

Published: 2011

Journal Articles

Safety of Recombinant Human Factor IX, Nonacog Alfa, for Usual Use In Pediatric Patients: Results From a Prospective Registry of European Hemophilia B Patients.

Pablo Rendo, MD, Erik Berntorp, MD, PhD, David M. Keeling, MD, Michael Makris, MD, Cedric R. Hermans, MD., PhD., Ereline Pauline Mauser-Bunschosten, Roberto Musso, Annarita Tagliaferri, Carl Kollmer, James Baumann, Robert Charnigo

Blood (2010) 116 (21): 3664.

https://doi.org/10.1182/blood.V116.21.3664.3664

Published: 2010

NARROW

Interim Safety and Efficacy Results from a Phase 1/2 Study of Zinc Finger Nuclease-Modified Autologous Hematopoietic Stem Cells for Sickle Cell Disease (PRECIZN-1)

Quantitative Systems Pharmacology Model of Sickle Cell Disease and Response to Gene Editing Therapy to Support Clinical Development of SAR445136 (BIVV003)

Prophylaxis with rFIXFc Reduces the Frequency and Delays Time to First Spontaneous Bleed Event in Previously Untreated Patients with Hemophilia B: A Post Hoc Analysis of the PUPs B-LONG Trial

Preliminary Safety and Efficacy Results from Precizn-1: An Ongoing Phase 1/2 Study on Zinc Finger Nuclease-Modified Autologous CD34+ HSPCs for Sickle Cell Disease (SCD)

Zinc Finger Nuclease-Mediated Disruption of the BCL11A Erythroid Enhancer Results in Enriched Biallelic Editing, Increased Fetal Hemoglobin, and Reduced Sickling in Erythroid Cells Derived from Sickle Cell Disease Patients

Impact of Treatment Regimen with Moroctocog Alfa (AF-CC) on Bleeding Frequency in Pediatric Aged Subjects with Moderately Severe to Severe Hemophilia A

An Open-Label Study of the Safety and Efficacy of Morotocog Alfa (AF-CC) in Previously Untreated Patients Aged <6 Years in Usual Care Settings

Once-Weekly Prophylactic Treatment Versus on-Demand Treatment of Nonacog Alfa in Patients with Moderately Severe to Severe Hemophilia B

Nonacog Alfa: Analysis Of Safety Data From 6 Prospective Clinical Studies In Different Patient Populations With Hemophilia B Treated With Different Therapeutic Modalities

Lack of Seasonal Variation in Bleeding and Patient-Assessed Pain Patterns in Patients with Hemophilia B Receiving On-Demand Therapy

Evaluation of Two Secondary Prophylaxis Regimens of Recombinant Factor IX(r-IX) in Moderately Severe to Severe( FIX ≤2%) Hemophilia B Patients

Pharmacokinetic/Pharmacodynamic Assessment of Reformulated Recombinant Coagulation Factor IX in Adults and Children with Severe Hemophilia B

Safety of Recombinant Human Factor IX, Nonacog Alfa, for Usual Use In Pediatric Patients: Results From a Prospective Registry of European Hemophilia B Patients.

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American Society of Hematology

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ASH Publications

American Society of Hematology

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