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Journal Articles

Early Effects of a Repurposed Therapeutic PB-04 in Beta Thalassemia Intermedia and in Transgenic Sickle Mice

Betty S. Pace, Seyed Mehdi Nouraie, Kevin H.M. Kuo, Sylvia Singer, Mayuko Takezaki, Biaoru Li, Aidan D Faller, Abdullah Kutlar, Susan P Perrine

Blood (2022) 140 (Supplement 1): 5360–5361.

https://doi.org/10.1182/blood-2022-169310

Published: 2022

Journal Articles

The Benefits Trial of PB-04 to Evaluate Safety, PK, and Preliminary Efficacy in Inducing Fetal Globin Expression in Beta Thalassemia Intermedia and Sickle Cell Disease

Kevin H.M. Kuo, Sylvia Singer, Sujit Sheth, Hanny Al-Samkari, Gershwin Blyden, Lanetta Bronte-Hall, Betty S. Pace, Abdullah Kutlar, Franciscus A. Kuypers, Aidan D Faller, Seyed Mehdi Nouraie, Pramod Terse, Haksong Jin, Xin Xu, Asaf Alimardanov, Arthur J. Sytkowski, Sharie Haugabook, Susan P Perrine

Blood (2021) 138 (Supplement 1): 4159.

https://doi.org/10.1182/blood-2021-148909

Published: 2021

Journal Articles

Actions of L-Glutamine vs. COVID-19 Suggest Additional Benefit in Sickle Cell Disease

Mahmoud A.Z. Abdelaal, MBBCh, Dyala Abdelrahman, MBBCh, MSc of Clinical Research Candidate, Mahir Cengiz, MD, Hakan Yavuzer, MD, Serap Yavuzer, MD, Ivy Bien, B.S., PharmD Candidate, Preeti Bhuva, B.S., M.H.A, PharmD Candidate, Jessica V Pham, B.S., PharmD Candidate, Royal Siu, B.S., PharmD Candidate, Mengdi Tang, PharmD, Ashley Ward, PhD, Jason Goodrow, PharmD, Peter Ludlum, B.S., M.B.A, Charles W. Stark, PharmD, Susan P. Perrine, MD

Blood (2020) 136 (Supplement 1): 11–12.

https://doi.org/10.1182/blood-2020-135903

Published: 2020

Journal Articles

A Pilot Study of Sleep Disordered Breathing (SDB) and Nocturnal Hypoxemia in Young Adults with Sickle Cell Disease

Susan P. Perrine, MD, Oluwakemi Owoyemi, MD MPH, Elizabeth S Klings, MD, Mehdi Nouraie, MD PhD, Angela Rock, Patricia A. Oneal, Thomas Mellman, MD, Tessema Berihun, Peter L. Whitesell, MD, Joseph Lavela, Robert E. Taylor, MD PhD

Blood (2015) 126 (23): 3414.

https://doi.org/10.1182/blood.V126.23.3414.3414

Published: 2015

Journal Articles

Activity of RN-1, an LSD-1 Inhibitor, on Fetal Globin Expression in Sickle Mice and Sickle Erythroid Progenitors

Shuaiying Cui, PhD, Jose Sangerman, PhD, Seyed Mehdi Nouraie, MD, PhD, Yan Dai, PhD, Oluwakemi Owoyemi, MD MPH, Robert E. Taylor, MD PhD, Susan P. Perrine, MD

Blood (2015) 126 (23): 961.

https://doi.org/10.1182/blood.V126.23.961.961

Published: 2015

Journal Articles

Multiple Oral Therapeutics Suppress Repressors (LSD-1, HDACs, and BCL11A) of Gamma Globin Gene Expression

Yan Dai, PhD, Douglas V Faller, MD PhD, Jose I Sangerman, PhD, Suthat Fucharoen, Susan P Perrine, MD

Blood (2014) 124 (21): 2687.

https://doi.org/10.1182/blood.V124.21.2687.2687

Published: 2014

Journal Articles

A phase 2 trial of HQK-1001 in HbE-β thalassemia demonstrates HbF induction and reduced anemia

Poramin Patthamalai, Suthat Fuchareon, Nattawara Chaneiam, Richard G. Ghalie, David H. K. Chui, Michael S. Boosalis, Susan P. Perrine

Blood (2014) 123 (12): 1956–1957.

https://doi.org/10.1182/blood-2013-11-538470

Published: 2014

Journal Articles

A Phase 2 Trial Of The Fetal Hemoglobin Inducer HQK-1001 (2,2-dimethylbutyrate, Sodium Salt) In Non-Transfusion-Dependent β-Thalassemia

Adlette Inati, MD, Mario Kahale, MD, Susan P Perrine, MD, Ali T Taher, MD, Suzanne Koussa, MD, Hussein A Abbas, PhD, Therese Abi Nasr, MD, Richard G. Ghalie, MD

Blood (2013) 122 (21): 2261.

https://doi.org/10.1182/blood.V122.21.2261.2261

Published: 2013

Journal Articles

Next Generation Clinical-Stage HbF Inducers Exhibit Multiple Molecular Actions In Vitro and In Vivo and Offer Personalized Therapeutic Regimens

Betty S. Pace, MD, Jose I Sangerman, PhD, Michael S Boosalis, PhD, Roman Wolf, DVM, Yan Dai, PhD, David H.K. Chui, MD, Levi Makala, PhD, DVM, Biadru Li, PhD, Douglas V Faller, MD, PhD, Susan P Perrine, MD

Blood (2013) 122 (21): 560.

https://doi.org/10.1182/blood.V122.21.560.560

Published: 2013

Journal Articles

A Phase 2 Trial of HQK-1001 (sodium 2,2-dimethylbutyrate), an Oral Fetal Globin Gene Inducer, in HbE-Beta Thalassemia in Thailand

Suthat Fucharoen, MD, Nattawara Chaneiam, Beer Poramin Patthamalai, Susan P Perrine, MD

Blood (2012) 120 (21): 251.

https://doi.org/10.1182/blood.V120.21.251.251

Published: 2012

Journal Articles

Histone deacetylase inhibitors are potent inducers of gene expression in latent EBV and sensitize lymphoma cells to nucleoside antiviral agents

Sajal K. Ghosh, Susan P. Perrine, Robert M. Williams, Douglas V. Faller

Blood (2012) 119 (4): 1008–1017.

https://doi.org/10.1182/blood-2011-06-362434

Published: 2012

Journal Articles

An Approach for Tailoring Diverse Therapeutic Inducers of Fetal Globin to Genetic Modifier Profiles,

Susan P Perrine, MD, David H.K. Chui, MD, Jose I Sangerman, PhD, Michael S Boosalis, PhD, Douglas V Faller, MD, PhD

Blood (2011) 118 (21): 3188.

https://doi.org/10.1182/blood.V118.21.3188.3188

Published: 2011

Journal Articles

Switching globin, raising red cells

Susan P. Perrine

Blood (2011) 118 (4): 834–836.

https://doi.org/10.1182/blood-2011-06-354373

Published: 2011

Journal Articles

Phase 1/2 Clinical Trial of HQK-1001, An Oral Fetal Hemoglobin Stimulant, In Sickle Cell Anemia

Abdullah Kutlar, MD, Kenneth I. Ataga, MD, Marvin Reid, PhD, Loray A Blair-Britt, MD, Elliott Vichinsky, MD, Lynne Neumayr, MD, Richard J. Labotka, MD, MS, Jonathan Glass, MD, FACP, Andrea Small-WIlliams, MBBS, DM(HAEM), Jeffrey Keefer, MD, PhD, Ling Shen, MD, Michael S Boosalis, PhD, Audrey Thomson, BSc, Patrick Bobbitt, BS, Wayne Wallis, MD, Elsa Johnson, MBA, Ronald J Berenson, MD, Susan P Perrine, MD

Blood (2010) 116 (21): 943.

https://doi.org/10.1182/blood.V116.21.943.943

Published: 2010

Journal Articles

Identification of New and Diverse Inducers of Fetal Hemoglobin with High Throughput Screening (HTS)

Jose I Sangerman, PhD, Michael S Boosalis, PhD, Ling Shen, MD, Sarah Haigh, PhD, Ada Kane, Gary L White, DVM, MMS, Susan P Perrine, MD, Douglas V Faller, MD, PhD

Blood (2010) 116 (21): 4277.

https://doi.org/10.1182/blood.V116.21.4277.4277

Published: 2010

Journal Articles

HQK-1001 Is Well Tolerated and Augments Hemoglobin F and Hemoglobin Levels In Patients with Beta Thalassemia Intermedia

Suthat Fuchareon, MD, Adlette C. Inati, MD, Noppadol Siritanaratkul, MD, Suzanne Koussa, MD, Ali Taher, MD, Michael S Boosalis, PhD, Swee Lay Thein, DSc, FRCP, MB, BS, FRCPath, Wayne Wallis, MD, Patrick Bobbitt, BS, Audrey Thomson, BSc, Elsa Johnson, MBA, Ronald J Berenson, MD, Susan P Perrine, MD

Blood (2010) 116 (21): 4280.

https://doi.org/10.1182/blood.V116.21.4280.4280

Published: 2010

Journal Articles

Histone Deacetylase Inhibitors: Potent Inducers of Tumor Latent EBV Thymidine Kinase Induction

Douglas V Faller, Sajal K Ghosh, Susan P Perrine, MD, Robert M Williams, Ronald J Berenson, MD

Blood (2010) 116 (21): 1831.

https://doi.org/10.1182/blood.V116.21.1831.1831

Published: 2010

Journal Articles

EKLF Is Recruited to the γ-Globin Gene Promoter as a Co-Activator and Is Required for γ-Globin Gene Induction by Short-Chain Fatty Acids.

Susan P. Perrine, MD, Rishikesh Mankidy, PhD, Michael S. Boosalis, PhD, James J. Bieker, PhD, Douglas V. Faller, MD, PhD

Blood (2007) 110 (11): 1771.

https://doi.org/10.1182/blood.V110.11.1771.1771

Published: 2007

Journal Articles

A phase 1/2 trial of arginine butyrate and ganciclovir in patients with Epstein-Barr virus–associated lymphoid malignancies

Susan P. Perrine, Olivier Hermine, Trudy Small, Felipe Suarez, Richard O'Reilly, Farid Boulad, Joyce Fingeroth, Melissa Askin, Arthur Levy, Steven J. Mentzer, Massimo Di Nicola, Alessandro M. Gianni, Christoph Klein, Steven Horwitz, Douglas V. Faller

Blood (2006) 109 (6): 2571–2578.

https://doi.org/10.1182/blood-2006-01-024703

Published: 2006

Journal Articles

Short-chain fatty acids induce γ-globin gene expression by displacement of a HDAC3-NCoR repressor complex

Rishikesh Mankidy, Douglas V. Faller, Rodwell Mabaera, Christopher H. Lowrey, Michael S. Boosalis, Gary L. White, Serguei A. Castaneda, Susan P. Perrine

Blood (2006) 108 (9): 3179–3186.

https://doi.org/10.1182/blood-2005-12-010934

Published: 2006

NARROW

Early Effects of a Repurposed Therapeutic PB-04 in Beta Thalassemia Intermedia and in Transgenic Sickle Mice

The Benefits Trial of PB-04 to Evaluate Safety, PK, and Preliminary Efficacy in Inducing Fetal Globin Expression in Beta Thalassemia Intermedia and Sickle Cell Disease

Actions of L-Glutamine vs. COVID-19 Suggest Additional Benefit in Sickle Cell Disease

A Pilot Study of Sleep Disordered Breathing (SDB) and Nocturnal Hypoxemia in Young Adults with Sickle Cell Disease

Activity of RN-1, an LSD-1 Inhibitor, on Fetal Globin Expression in Sickle Mice and Sickle Erythroid Progenitors

Multiple Oral Therapeutics Suppress Repressors (LSD-1, HDACs, and BCL11A) of Gamma Globin Gene Expression

A phase 2 trial of HQK-1001 in HbE-β thalassemia demonstrates HbF induction and reduced anemia

A Phase 2 Trial Of The Fetal Hemoglobin Inducer HQK-1001 (2,2-dimethylbutyrate, Sodium Salt) In Non-Transfusion-Dependent β-Thalassemia

Next Generation Clinical-Stage HbF Inducers Exhibit Multiple Molecular Actions In Vitro and In Vivo and Offer Personalized Therapeutic Regimens

A Phase 2 Trial of HQK-1001 (sodium 2,2-dimethylbutyrate), an Oral Fetal Globin Gene Inducer, in HbE-Beta Thalassemia in Thailand

Histone deacetylase inhibitors are potent inducers of gene expression in latent EBV and sensitize lymphoma cells to nucleoside antiviral agents

An Approach for Tailoring Diverse Therapeutic Inducers of Fetal Globin to Genetic Modifier Profiles,

Switching globin, raising red cells

Phase 1/2 Clinical Trial of HQK-1001, An Oral Fetal Hemoglobin Stimulant, In Sickle Cell Anemia

Identification of New and Diverse Inducers of Fetal Hemoglobin with High Throughput Screening (HTS)

HQK-1001 Is Well Tolerated and Augments Hemoglobin F and Hemoglobin Levels In Patients with Beta Thalassemia Intermedia

Histone Deacetylase Inhibitors: Potent Inducers of Tumor Latent EBV Thymidine Kinase Induction

EKLF Is Recruited to the γ-Globin Gene Promoter as a Co-Activator and Is Required for γ-Globin Gene Induction by Short-Chain Fatty Acids.

A phase 1/2 trial of arginine butyrate and ganciclovir in patients with Epstein-Barr virus–associated lymphoid malignancies

Short-chain fatty acids induce γ-globin gene expression by displacement of a HDAC3-NCoR repressor complex

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American Society of Hematology

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American Society of Hematology

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