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Journal Articles

Development of a Comprehensive Program for Evaluation of Hereditary Predisposition to Hematologic Malignancies

Jennie Vagher, Bryan D. Huber, Luke Maese, Archana M Agarwal, Afaf Osman, Ami B. Patel, Tsewang Tashi, Paul J Shami, Tibor Kovacsovics, Julie D. Asch, Charles J. Parker, Srinivas K. Tantravahi

Blood (2022) 140 (Supplement 1): 11468–11470.

https://doi.org/10.1182/blood-2022-170000

Published: 2022

Journal Articles

Disease Characteristics of AML Patients with Germline DDX41 Variants

Jennie Vagher, Shobi Venkatachalam, Peng Li, Julie D. Asch, Bryan D. Huber, Tsewang Tashi, Paul J Shami, Tibor Kovacsovics, Luke Maese, Charles J. Parker, Srinivas K. Tantravahi

Blood (2021) 138 (Supplement 1): 3384.

https://doi.org/10.1182/blood-2021-154117

Published: 2021

Journal Articles

Eighteen-Month Interim Analysis of Efficacy and Safety of Givosiran, an RNAi Therapeutic for Acute Hepatic Porphyria, in the Envision Open Label Extension

David J. Kuter, MD, Sioban Keel, MD, Charles J. Parker, MD, David C. Rees, MD, Ulrich Stölzel, MD, Paolo Ventura, MD, Manisha Balwani, Laurent Gouya, MD, PhD, Amy Simon, MD, Shangbin Liu, PhD, John J Ko, PharmD,MSc, Sean Rhyee, MD, M.P.H, Samuel M. Silver, MD PhD

Blood (2020) 136 (Supplement 1): 13.

https://doi.org/10.1182/blood-2020-134564

Published: 2020

Journal Articles

Erythropoietic Protoporphyria: Phase 2 Clinical Trial Results Evaluating the Safety and Effectiveness of Dersimelagon (MT-7117), an Oral MC1R Agonist

Manisha Balwani, Herbert L. Bonkovsky, MD, Kirstine J Belongie, PhD, Karl E Anderson, MD, Fumihiro Takahashi, PhD, Antonio Irizarry, MBA, Mark Amster, MD, D. Montgomery Bissell, MD, Bruce Wang, MD, Lydie Hazan, MD, Charles J. Parker, MD, Edward Cordasco, MD, Cynthia Levy, MD, Robert J. Desnick, MD PhD

Blood (2020) 136 (Supplement 1): 51.

https://doi.org/10.1182/blood-2020-142467

Published: 2020

Journal Articles

Association of GNPAT p.D519G with Higher Iron Phenotypes in Women with HFE p.C282Y Homozygosity

Gordon D. McLaren, MD, Pradyumna D. Phatak, MBBS, James C. Barton, MD, John D. Phillips, PhD, Wen-Pin Chen, MS, V. Nathan Subraminiam, PhD, Paul C. Adams, MD, Lyle C. Gurrin, PhD, Gregory J. Anderson, BSc, MSc, PhD, Grant A. Ramm, PhD, Lawrie W. Powell, MD PhD, Charles J. Parker, MD, Mary J. Emond, PhD, Christine E. McLaren, PhD

Blood (2017) 130 (Supplement 1): 2216.

https://doi.org/10.1182/blood.V130.Suppl_1.2216.2216

Published: 2017

Journal Articles

Explore: A Prospective, Multinational Natural History Study of Patients with Acute Hepatic Porphyrias (AHP) with Recurrent Attacks

Blood (2017) 130 (Supplement 1): 2211.

https://doi.org/10.1182/blood.V130.Suppl_1.2211.2211

Published: 2017

Journal Articles

Interim Data from a Randomized, Placebo Controlled, Phase 1 Study of Aln-AS1, an Investigational RNAi Therapeutic for the Treatment of Acute Hepatic Porphyria

Eliane Sardh, MD PhD, Pauline Harper, MD PhD, Nabil Al-Tawil, MD, Manisha Balwani, MD MS, Karl Anderson, MD, Joseph Bloomer, MD, D. Montgomery Bissel, MD, Robert J. Desnick, MD, Charles J. Parker, MD, John D. Phillips, PhD, Herbert L. Bonkovsky, MD, Craig Penz, MA, Amy Chan, PhD, Chang-Heok Soh, PhD, William Querbes, PhD, Amy Simon, MD, Penelope Stein, MD PhD, David C. Rees, FRCP, FRCPath, MA, MBBS

Blood (2016) 128 (22): 2318.

https://doi.org/10.1182/blood.V128.22.2318.2318

Published: 2016

Journal Articles

GNPAT p.D519G is Independently Associated with Markedly Increased Iron Stores in HFE p.C282Y Homozygotes

James C. Barton, MD, Wen-Pin Chen, MS, Mary J. Emond, PhD, Pradyumna D. Phatak, MD, V. Nathan Subramaniam, PhD, Paul C. Adams, MD, Lyle C. Gurrin, PhD, Gregory J. Anderson, BSc, MSc, PhD, Grant A. Ramm, PhD, Lawrie W. Powell, MD, PhD, Katrina J. Allen, FRACP, MD PhD, John D. Phillips, PhD, Charles J. Parker, MD, Gordon D. McLaren, MD, Christine E. McLaren, PhD

Blood (2016) 128 (22): 3617.

https://doi.org/10.1182/blood.V128.22.3617.3617

Published: 2016

Journal Articles

Identification of Polymorphic Gnpat As a Risk Factor for Porphyria Cutanea Tarda

John D Phillips, PhD, Colin P Farrell, MPH, Jessica Overbey, MS, Hetanshi Naik, BS, Gordon D. McLaren, MD, Christine E McLaren, PhD, Luming Zhou, PhD, Charles J. Parker, MD

Blood (2015) 126 (23): 3353.

https://doi.org/10.1182/blood.V126.23.3353.3353

Published: 2015

Journal Articles

Methylation of AR locus does not always reflect X chromosome inactivation state

Sabina I. Swierczek, Lucie Piterkova, Jaroslav Jelinek, Neeraj Agarwal, Sue Hammoud, Andrew Wilson, Kimberly Hickman, Charles J. Parker, Bradley R. Cairns, Josef T. Prchal

Blood (2012) 119 (13): e100–e109.

https://doi.org/10.1182/blood-2011-11-390351

Published: 2012

Includes: Supplemental data

Journal Articles

A novel approach to preventing the hemolysis of paroxysmal nocturnal hemoglobinuria: both complement-mediated cytolysis and C3 deposition are blocked by a monoclonal antibody specific for the alternative pathway of complement

Margaret A. Lindorfer, Andrew W. Pawluczkowycz, Elizabeth M. Peek, Kimberly Hickman, Ronald P. Taylor, Charles J. Parker

Blood (2010) 115 (11): 2283–2291.

https://doi.org/10.1182/blood-2009-09-244285

Published: 2010

Journal Articles

A Novel Approach to Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH): Both Hemolysis and C3 Deposition Are Blocked by a Monoclonal Antibody (mAb) Specific for the Alternative Pathway of Complement (APC) C3/C5 Convertase.

Ronald P Taylor, PhD, Margaret A Lindorfer, PhD, Andrew W Pawluczkowycz, MS, Charles J Parker, MD

Blood (2009) 114 (22): 157.

https://doi.org/10.1182/blood.V114.22.157.157

Published: 2009

Journal Articles

Monoallelic Methylation of the Human Androgen Receptor Gene as Detected by the HUMARA Clonality Assay Does Not Consistently Reflect X-Chromosome Inactivation and Is Therefore Unreliable for Assessment of Clonal Hematopoiesis.

Sabina Swierczek, PhD, Jaroslav Jelinek, PhD, Neeraj Agarwal, MD, Andrew Wilson, Kimberly Hickman, Charles J. Parker, MD, Josef T. Prchal, MD

Blood (2009) 114 (22): 4566.

https://doi.org/10.1182/blood.V114.22.4566.4566

Published: 2009

Journal Articles

Does HUMARA assay for assessment of clonal hematopoiesis have shortcomings?

Neeraj Agarwal, Roberto H. Nussenzveig, Sabina I. Swierczek, Charles J. Parker, Josef T. Prchal

Blood (2009) 114 (11): 2357–2358.

https://doi.org/10.1182/blood-2009-04-217653

Published: 2009

Journal Articles

Flow Cytometry Analysis of Peripheral Blood CD34+ Cells in Patients with Primary Myelofibrosis

Archana M Agarwal, MD, Scott James Samuelson, M.D., Sergey Preobrazhensky, PhD, Charles J. Parker, MD, Kimberly Hickman, Sherrie L. Perkins, MD, PhD, Josef T. Prchal, MD, David W. Bahler, MD, PhD.

Blood (2008) 112 (11): 5240.

https://doi.org/10.1182/blood.V112.11.5240.5240

Published: 2008

Journal Articles

Validation of a Novel Clonality Assay in Patients with Clonal Hematologic Disorders

Neeraj Agarwal, MD#, Sabina Swierczek, PhD#, Roberto Nussenzveig, PhD, Scott James Samuelson, M.D., Charles J. Parker, MD, Josef T. Prchal, MD

Blood (2008) 112 (11): 3718.

https://doi.org/10.1182/blood.V112.11.3718.3718

Published: 2008

Journal Articles

Preclinical Characterization of the JAK-2 Inhibitor, SGI-1252

Kausar Begam Riaz Ahmed, Roberto H Nussenzveig, PhD, Andrew T. Chen, DO, Josef T. Prchal, MD, Charles J. Parker, MD, Eric Gourley, Paul Severson, Jared Bearss, Carissa Jones, Steven L. Warner, David Vollmer, David J. Bearss

Blood (2008) 112 (11): 2629.

https://doi.org/10.1182/blood.V112.11.2629.2629

Published: 2008

Journal Articles

Revised criteria for the myeloproliferative disorders: too much too soon?

Scott J. Samuelson, Charles J. Parker, Josef T. Prchal

Blood (2008) 111 (3): 1741.

https://doi.org/10.1182/blood-2007-10-116897

Published: 2008

Journal Articles

Analysis of Mutant cMPL in Philadelphia Chromosome-Negative Myeloproliferative Disorders (Ph − MPDs) Using a Novel High-Sensitivity Assay.

Scott J. Samuelson, MD, Sabina Swierczek, Ph.D., Charles J. Parker, M.D., Kenneth Boucher, Ph.D., Jaroslav Jelinek, M.D., Josef Prchal, M.D.

Blood (2007) 110 (11): 2544.

https://doi.org/10.1182/blood.V110.11.2544.2544

Published: 2007

Journal Articles

Molecular basis of clonal expansion of hematopoiesis in 2 patients with paroxysmal nocturnal hemoglobinuria (PNH)

Norimitsu Inoue, Tomohisa Izui-Sarumaru, Yoshiko Murakami, Yuichi Endo, Jun-Ichi Nishimura, Ken Kurokawa, Maki Kuwayama, Hiroaki Shime, Takashi Machii, Yuzuru Kanakura, Gabrielle Meyers, Carl Wittwer, Zhong Chen, William Babcock, Debra Frei-Lahr, Charles J. Parker, Taroh Kinoshita

Blood (2006) 108 (13): 4232–4236.

https://doi.org/10.1182/blood-2006-05-025148

Published: 2006

NARROW

Development of a Comprehensive Program for Evaluation of Hereditary Predisposition to Hematologic Malignancies

Disease Characteristics of AML Patients with Germline DDX41 Variants

Eighteen-Month Interim Analysis of Efficacy and Safety of Givosiran, an RNAi Therapeutic for Acute Hepatic Porphyria, in the Envision Open Label Extension

Erythropoietic Protoporphyria: Phase 2 Clinical Trial Results Evaluating the Safety and Effectiveness of Dersimelagon (MT-7117), an Oral MC1R Agonist

Association of GNPAT p.D519G with Higher Iron Phenotypes in Women with HFE p.C282Y Homozygosity

Explore: A Prospective, Multinational Natural History Study of Patients with Acute Hepatic Porphyrias (AHP) with Recurrent Attacks

Interim Data from a Randomized, Placebo Controlled, Phase 1 Study of Aln-AS1, an Investigational RNAi Therapeutic for the Treatment of Acute Hepatic Porphyria

GNPAT p.D519G is Independently Associated with Markedly Increased Iron Stores in HFE p.C282Y Homozygotes

Identification of Polymorphic Gnpat As a Risk Factor for Porphyria Cutanea Tarda

Methylation of AR locus does not always reflect X chromosome inactivation state

A novel approach to preventing the hemolysis of paroxysmal nocturnal hemoglobinuria: both complement-mediated cytolysis and C3 deposition are blocked by a monoclonal antibody specific for the alternative pathway of complement

A Novel Approach to Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH): Both Hemolysis and C3 Deposition Are Blocked by a Monoclonal Antibody (mAb) Specific for the Alternative Pathway of Complement (APC) C3/C5 Convertase.

Monoallelic Methylation of the Human Androgen Receptor Gene as Detected by the HUMARA Clonality Assay Does Not Consistently Reflect X-Chromosome Inactivation and Is Therefore Unreliable for Assessment of Clonal Hematopoiesis.

Does HUMARA assay for assessment of clonal hematopoiesis have shortcomings?

Flow Cytometry Analysis of Peripheral Blood CD34+ Cells in Patients with Primary Myelofibrosis

Validation of a Novel Clonality Assay in Patients with Clonal Hematologic Disorders

Preclinical Characterization of the JAK-2 Inhibitor, SGI-1252

Revised criteria for the myeloproliferative disorders: too much too soon?

Analysis of Mutant cMPL in Philadelphia Chromosome-Negative Myeloproliferative Disorders (Ph − MPDs) Using a Novel High-Sensitivity Assay.

Molecular basis of clonal expansion of hematopoiesis in 2 patients with paroxysmal nocturnal hemoglobinuria (PNH)

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American Society of Hematology

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American Society of Hematology

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