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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

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

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

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

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

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

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

Molecular Complete Remission of Clonal Hematopoiesis in Paroxysmal Nocturnal Hemoglobinuria (PNH) after Syngeneic Transplantation.

Debra Frei-Lahr, MD, Norimitsu Inoue, Carl Wittwer, Mark Herrmann, William Babcock, MD, Taroh Kinoshita, Charles J. Parker, MD

Blood (2006) 108 (11): 5362.

https://doi.org/10.1182/blood.V108.11.5362.5362

Published: 2006

Journal Articles

Comparison of Gene Expression in Dominant and Non-Dominant PIG-A Mutant Clones in Paroxysmal Nocturnal Hemoglobinuria (PNH).

Gabrielle Meyers, MD, Charles J. Parker, MD

Blood (2004) 104 (11): 2830.

https://doi.org/10.1182/blood.V104.11.2830.2830

Published: 2004

Journal Articles

Identification of a Novel Hematopoietic Cell Translocation in a Patient with Paroxysmal Nocturnal Hemoglobinuria (PNH).

Gabrielle Meyers, MD, William H. Babcock, MD, Robert G. Best, PhD, Charles J. Parker, MD

Blood (2004) 104 (11): 3717.

https://doi.org/10.1182/blood.V104.11.3717.3717

Published: 2004

Journal Articles

Results of a Survey of the Prevalence of Paroxysmal Nocturnal Hemoglobinuria (PNH).

Charles J. Parker, MD, Wendell F. Rosse, MD

Blood (2004) 104 (11): 3720.

https://doi.org/10.1182/blood.V104.11.3720.3720

Published: 2004

NARROW

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

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

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

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

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

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.

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

Molecular Complete Remission of Clonal Hematopoiesis in Paroxysmal Nocturnal Hemoglobinuria (PNH) after Syngeneic Transplantation.

Comparison of Gene Expression in Dominant and Non-Dominant PIG-A Mutant Clones in Paroxysmal Nocturnal Hemoglobinuria (PNH).

Identification of a Novel Hematopoietic Cell Translocation in a Patient with Paroxysmal Nocturnal Hemoglobinuria (PNH).

Results of a Survey of the Prevalence of Paroxysmal Nocturnal Hemoglobinuria (PNH).

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American Society of Hematology

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ASH Publications

American Society of Hematology

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