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Literature DB >> 24224649

A phase 2 study of HQK-1001, an oral fetal haemoglobin inducer, in β-thalassaemia intermedia.

Adlette Inati¹, Mario Kahale, Susan P Perrine, David H K Chui, Ali T Taher, Suzanne Koussa, Therese Abi Nasr, Hussein A Abbas, Richard G Ghalie.

Abstract

Entities: Chemical Disease Gene Mutation Species

Keywords: anaemia; fetal haemoglobin; gene expression; haemoglobinopathies; β-thalassaemia

Mesh：

Substances：

Year: 2013 PMID： 24224649 PMCID： PMC3904791 DOI： 10.1111/bjh.12635

Source DB: PubMed Journal: Br J Haematol ISSN： 0007-1048 Impact factor: 6.998

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10 in total

Review 1. Control of fetal hemoglobin: new insights emerging from genomics and clinical implications.

Authors: Swee Lay Thein; Stephan Menzel; Mark Lathrop; Chad Garner
Journal: Hum Mol Genet Date: 2009-10-15 Impact factor: 6.150

Review 2. Reawakening fetal hemoglobin: prospects for new therapies for the β-globin disorders.

Authors: Daniel E Bauer; Sophia C Kamran; Stuart H Orkin
Journal: Blood Date: 2012-08-17 Impact factor: 22.113

3. Oral isobutyramide therapy in patients with thalassemia intermedia: results of a phase II open study.

Authors: M Domenica Cappellini; G Graziadei; L Ciceri; A Comino; P Bianchi; A Porcella; G Fiorelli
Journal: Blood Cells Mol Dis Date: 2000-02 Impact factor: 3.039

Review 4. The inherited diseases of hemoglobin are an emerging global health burden.

Authors: David J Weatherall
Journal: Blood Date: 2010-03-16 Impact factor: 22.113

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

Authors: Rishikesh Mankidy; Douglas V Faller; Rodwell Mabaera; Christopher H Lowrey; Michael S Boosalis; Gary L White; Serguei A Castaneda; Susan P Perrine
Journal: Blood Date: 2006-07-18 Impact factor: 22.113

6. A randomized phase I/II trial of HQK-1001, an oral fetal globin gene inducer, in β-thalassaemia intermedia and HbE/β-thalassaemia.

Authors: Suthat Fucharoen; Adlette Inati; Noppadol Siritanaratku; Swee L Thein; William C Wargin; Suzanne Koussa; Ali Taher; Nattawara Chaneim; Michael Boosalis; Ronald Berenson; Susan P Perrine
Journal: Br J Haematol Date: 2013-03-27 Impact factor: 6.998

Review 7. Clinical experience with fetal hemoglobin induction therapy in patients with β-thalassemia.

Authors: Khaled M Musallam; Ali T Taher; Maria Domenica Cappellini; Vijay G Sankaran
Journal: Blood Date: 2013-01-11 Impact factor: 22.113

8. Short-chain fatty acid derivatives induce fetal globin expression and erythropoiesis in vivo.

Authors: Betty S Pace; Gary L White; George J Dover; Michael S Boosalis; Douglas V Faller; Susan P Perrine
Journal: Blood Date: 2002-08-15 Impact factor: 22.113

9. A short-term trial of butyrate to stimulate fetal-globin-gene expression in the beta-globin disorders.

Authors: S P Perrine; G D Ginder; D V Faller; G H Dover; T Ikuta; H E Witkowska; S P Cai; E P Vichinsky; N F Olivieri
Journal: N Engl J Med Date: 1993-01-14 Impact factor: 91.245

10. Oral sodium phenylbutyrate therapy in homozygous beta thalassemia: a clinical trial.

Authors: A F Collins; H A Pearson; P Giardina; K T McDonagh; S W Brusilow; G J Dover
Journal: Blood Date: 1995-01-01 Impact factor: 22.113