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  1. Guidelines for the diagnosis and monitoring of paroxysmal nocturnal hemoglobinuria and related disorders by flow cytometry.

    ... stem cell disorder characterized by a somatic mutation in the PIGA gene, leading to a deficiency of proteins linked to ...

    Research Article last updated 10/11/2011 - 5:58pm.

  2. Paroxysmal nocturnal hemoglobinuria from bench to bedside

    ... hematopoietic stem cell (HSC) that acquires a somatic mutation in a gene called phosphatidylinositol glycan anchor biosynthesis, ...

    Research Article last updated 10/11/2011 - 5:58pm.

  3. A Phase 2 Safety and Efficacy Study of RA101495 to Treat PNH Patients Who Have an Inadequate Response to Eculizumab

    ... limit of normal (ULN) and/or the presence of a known C5 mutation conferring resistance to eculizumab   ...

    Clinical Trial last updated 04/20/2017 - 11:14am.

  4. Somatic mutations predict poor outcome in patients with myelodysplastic syndrome after hematopoietic stem-cell transplantation.

    ... shorter OS. Nearly one half of patients (46%) carried a mutation in TP53, DNMT3A, or TET2 and accounted for 64% of deaths. ...

    Research Article last updated 10/01/2014 - 10:59am.

  5. p53 protein expression independently predicts outcome in patients with lower-risk myelodysplastic syndromes with del(5q)

    ... cells with strong p53 expression confirmed TP53 mutation , whereas cells with moderate expression were predominantly of ...

    Research Article last updated 04/23/2014 - 12:26pm.

  6. PNH: Causes

    ... What causes PNH? PNH occurs because of a change ( mutation ) in the PIG-A gene of a single stem cell in the bone ...

    Page last updated 06/29/2015 - 8:44am.

  7. Key clinical observations after 5-azacytidine and decitabine treatment of myelodysplastic syndromes suggest practical solutions for better outcomes

    ... that there can be no therapeutic benefit, regardless of mutation or cytogenetic subtype, if DNA methyltransferase is not depleted ...

    Research Article last updated 01/08/2014 - 9:30am.

  8. Epigenetic regulation in myelodysplastic syndromes: implications for therapy.

    ... and high-risk MDS. Methylation profiles and gene mutation predictors provide promising areas of development for monitoring ...

    Research Article last updated 10/11/2011 - 5:58pm.

  9. A Phase 1, Multicenter, Open-Label, Dose-Escalation and Expansion, Safety, Pharmacokinetic, Pharmacodynamic, and Clinical Activity Study of Orally Administered AG-120 in Subjects with Advanced Hematologic Malignancies with an IDH1 Mutation

    ... in advanced hematologic malignancies that harbor an IDH1 mutation . Status:  ...

    Clinical Trial last updated 02/22/2017 - 11:21am.

  10. Clinical and Genetic Heterogeneity of Telomere Diseases

    ... for in all patients. Interestingly, a telomere gene mutation may be found in patients who seem to have a primary immune defect, ... of the genetic abnormality suggesting that finding a genetic mutation should not preclude such treatment. ...

    Research Review last updated 05/02/2016 - 9:24am.