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  1. van Beers, Eduard J. [Author]; Straaten, Stephanie van [Author]; Morton, D. Holmes [Author]; Barcellini, Wilma [Author]; Eber, Stefan W. [Author]; Glader, Bertil [Author]; Yaish, Hassan M. [Author]; Chonat, Satheesh [Author]; Kwiatkowski, Janet L. [Author]; Rothman, Jennifer A. [Author]; Sharma, Mukta [Author]; Neufeld, Ellis J. [Author]; Sheth, Sujit [Author]; Despotovic, Jenny M. [Author]; Kollmar, Nina [Author]; Pospíšilová, Dagmar [Author]; Knoll, Christine M. [Author]; Kuo, Kevin [Author]; Pastore, Yves D. [Author]; Thompson, Alexis A. [Author]; Newburger, Peter E. [Author]; Ravindranath, Yaddanapudi [Author]; Wang, Winfred C. [Author]; Wlodarski, Marcin W. [Author]; [...]

    Prevalence and management of iron overload in pyruvate kinase deficiency: report from the Pyruvate kinase deficiency natural history study

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    Pavia: Ferrata Storti Foundation, 2019

    Published in: Haematologica ; 104, 2 (2019), e51-e53$z1592-8721

  3. Lezon-Geyda, Kimberly; Rose, Melissa J.; McNaull, Melissa A.; Knoll, Christine M.; Yaish, Hassan M.; Pastore, Yves D.; Fermi, Elisa; Glader, Bertil; Bianchi, Paola; Grace, Rachael F.; Gallagher, Patrick G.

    Pklr Intron Splicing-Associated Mutations and Alternate Diagnoses Are Common in Pyruvate Kinase Deficient Patients with Single or No Pklr Coding Mutations

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    American Society of Hematology, 2018

    Published in: Blood, 132 (2018) Supplement 1, Seite 3607-3607

  4. Daak, Ahmed A.; Dampier, Carlton D.; Fuh, Beng; Kanter, Julie; Alvarez, Ofelia A.; Black, L. Vandy; McNaull, Melissa A.; Callaghan, Michael U.; George, Alex; Neumayr, Lynne; Hilliard, Lee M.; Sancilio, Fredrick; Rabinowicz, Adrian L.; Heeney, Matthew M.

    Double-blind, randomized, multicenter phase 2 study of SC411 in children with sickle cell disease (SCOT trial)

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    American Society of Hematology, 2018

    Published in: Blood Advances, 2 (2018) 15, Seite 1969-1979

  5. Yildirim, Melissa I.; Spaeder, Michael C.; Castro, Barbara A.; Chamberlain, Rebecca; Fuzy, Lisa; Howard, Sarah; McNaull, Peggy; Raphael, Jacob; Sharma, Ruchik; Vizzini, Samantha; Wielar, Amy; Frank, Deborah U.

    The Impact of Nasal Intubation on Feeding Outcomes in Neonates Requiring Cardiac Surgery: A Randomized Control Trial

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    Springer Science and Business Media LLC, 2024

    Published in: Pediatric Cardiology, 45 (2024) 2, Seite 426-432

  6. Wang, Winfred C.; Brown, R. Clark; McNaull, Melissa A.; Rogers, Zora R.; Barton, Martha; Dua, Meghna R.; Hankins, Jane S.; Gossett, Jeffrey; Richardson, Julie; Porter, Jerlym S.; Kang, Guolian; Estepp, Jeremie H.

    Intensive hydroxyurea dosing in very young children with sickle cell anemia

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    American Society of Hematology, 2023

    Published in: Blood Advances, 7 (2023) 22, Seite 6931-6935

  7. Dua, Meghna; Wang, Winfred C.; Brown, R. Clark; McNaull, Melissa A.; Rogers, Zora R.; Barton, Martha; Hankins, Jane; Gossett, Jeffrey; Richardson, Julie; Porter, Jerlym S.; Kang, Guolian; Estepp, Jeremie H.

    Hydroxyurea Dosing in Very Young Children with Sickle Cell Anemia: A Multicenter, Randomized, Controlled Trial (HUGKISS)

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    American Society of Hematology, 2022

    Published in: Blood, 140 (2022) Supplement 1, Seite 445-446

  8. Lanzkron, Sophie; Manwani, Deepa; Kanter, Julie; Sinha, Arpan A; Miller, Robin E.; Cronin, Robert; Jacob, Seethal A; Harper, James; Anderson, Alan Randall; Treadwell, Marsha; Owusu-Ansah, Amma T.; Fuh, Beng R.; Mandernach, Molly; Gollamudi, Jahnavi; Saccente, Suzanne L; Fritch Lilla, Stephanie A.; McNaull, Melissa; LeBlanc, Dana Marie; Saving, Kay Linn; Guarino, Stephanie H.; Betensky, Marisol; Cohen, Alice J.; Raj, Ashok B.; Sayani, Farzana A; [...]

    Grndad and Disease Modifying Therapy (DMT): Shifts in Dmt Are Seen at the Adolescent/Young Adult Transition in Sickle Cell Disease in a Multi-Site Prospective Registry

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    American Society of Hematology, 2023

    Published in: Blood, 142 (2023) Supplement 1, Seite 798-798

  9. van Beers, Eduard J.; van Straaten, Stephanie; Morton, D. Holmes; Barcellini, Wilma; Eber, Stefan W.; Glader, Bertil; Yaish, Hassan M.; Chonat, Satheesh; Kwiatkowski, Janet L.; Rothman, Jennifer A.; Sharma, Mukta; Neufeld, Ellis J.; Sheth, Sujit; Despotovic, Jenny M.; Kollmar, Nina; Pospíšilová, Dagmar; Knoll, Christine M.; Kuo, Kevin; Pastore, Yves D.; Thompson, Alexis A.; Newburger, Peter E.; Ravindranath, Yaddanapudi; Wang, Winfred C.; Wlodarski, Marcin W.; [...]

    Prevalence and management of iron overload in pyruvate kinase deficiency: report from the Pyruvate Kinase Deficiency Natural History Study

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    Ferrata Storti Foundation (Haematologica), 2019

    Published in: Haematologica, 104 (2019) 2, Seite e51-e53

  10. Van Beers, Eduard J.; Kuo, Kevin H. M.; Morton, D. Holmes; Barcellini, Wilma; Eber, Stefan W.; Glader, Bertil; Yaish, Hassan M.; Chonat, Satheesh; Kollmar, Nina; Despotovic, Jenny M.; Pospisilova, Dagmar; Knoll, Christine M.; Kwiatkowski, Janet L.; Pastore, Yves D.; Thompson, Alexis A.; Wang, Winfred; Wlodarski, Marcin W.; Newburger, Peter E.; Ravindranath, Yaddanapudi; Rothman, Jennifer A.; Wang, Heng; Holzhauer, Suzanne; Breakey, Vicky R.; Verhovsek, Madeleine M; [...]

    Health Related Quality of Life and Fatigue in Patients with Pyruvate Kinase Deficiency

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    American Society of Hematology, 2018

    Published in: Blood, 132 (2018) Supplement 1, Seite 4807-4807

  11. Al‐Samkari, Hanny; van Beers, Eduard J.; Morton, D. Holmes; Barcellini, Wilma; Eber, Stefan W.; Glader, Bertil; Yaish, Hassan M.; Chonat, Satheesh; Kuo, Kevin H. M.; Kollmar, Nina; Despotovic, Jenny M.; Pospíšilová, Dagmar; Knoll, Christine M.; Kwiatkowski, Janet L.; Pastore, Yves D.; Thompson, Alexis A.; Wlodarski, Marcin W.; Ravindranath, Yaddanapudi; Rothman, Jennifer A.; Wang, Heng; Holzhauer, Susanne; Breakey, Vicky R.; Verhovsek, Madeleine M.; Kunz, Joachim; [...]

    Characterization of the severe phenotype of pyruvate kinase deficiency

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    Wiley, 2020

    Published in: American Journal of Hematology, 95 (2020) 10

  12. Al-Samkari, Hanny; van Beers, Eduard J.; Morton, D Holmes; Barcellini, Wilma; Eber, Stefan W.; Glader, Bertil; Yaish, Hassan M.; Chonat, Satheesh; Kuo, Kevin H. M.; Kollmar, Nina; Despotovic, Jenny M.; Pospisilova, Dagmar; Knoll, Christine M.; Kwiatkowski, Janet L.; Pastore, Yves D.; Thompson, Alexis A.; Wlodarski, Marcin W.; Ravindranath, Yaddanapudi; Rothman, Jennifer A.; Wang, Heng; Holzhauer, Suzanne; Breakey, Vicky R.; Verhovsek, Madeleine M.; Kunz, Joachim B.; [...]

    Characterization of the Severe Phenotype of Pyruvate Kinase Deficiency

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    American Society of Hematology, 2019

    Published in: Blood, 134 (2019) Supplement_1, Seite 949-949

  13. Boscoe, Audra N.; Yan, Yan; Hedgeman, Elizabeth; van Beers, Eduard J.; Al-Samkari, Hanny; Barcellini, Wilma; Eber, Stefan W.; Glader, Bertil; Yaish, Hassan M.; Chonat, Satheesh; Rothman, Jennifer A.; Sharma, Mukta; Kollmar, Nina; Knoll, Christine M.; Kuo, Kevin H. M.; Kwiatkowski, Janet L.; Pastore, Yves D.; Thompson, Alexis A; Ravindranath, Yaddanapudi; Neufeld, Ellis J.; Wang, Heng; Holzhauer, Susanne; Breakey, Vicky R.; Verhovsek, Madeleine M.; [...]

    Comorbidities and Complications in Adults with Pyruvate Kinase Deficiency

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    American Society of Hematology, 2019

    Published in: Blood, 134 (2019) Supplement_1, Seite 2175-2175

  14. Grace, Rachael F.; Bianchi, Paola; van Beers, Eduard J.; Eber, Stefan W.; Glader, Bertil; Yaish, Hassan M.; Despotovic, Jenny M.; Rothman, Jennifer A.; Sharma, Mukta; McNaull, Melissa M.; Fermo, Elisa; Lezon-Geyda, Kimberly; Morton, D. Holmes; Neufeld, Ellis J.; Chonat, Satheesh; Kollmar, Nina; Knoll, Christine M.; Kuo, Kevin; Kwiatkowski, Janet L.; Pospíšilová, Dagmar; Pastore, Yves D.; Thompson, Alexis A.; Newburger, Peter E.; Ravindranath, Yaddanapudi; [...]

    Clinical spectrum of pyruvate kinase deficiency: data from the Pyruvate Kinase Deficiency Natural History Study

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    American Society of Hematology, 2018

    Published in: Blood, 131 (2018) 20, Seite 2183-2192