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Journal Articles

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

Free
Clinical Trials & Observations
Rachael F. Grace, Paola Bianchi, Eduard J. van Beers, Stefan W. Eber, Bertil Glader, Hassan M. Yaish, Jenny M. Despotovic, Jennifer A. Rothman, Mukta Sharma, Melissa M. McNaull, Elisa Fermo, Kimberly Lezon-Geyda, D. Holmes Morton, Ellis J. Neufeld, Satheesh Chonat, Nina Kollmar, Christine M. Knoll, Kevin Kuo, Janet L. Kwiatkowski, Dagmar Pospíšilová, Yves D. Pastore, Alexis A. Thompson, Peter E. Newburger, Yaddanapudi Ravindranath, Winfred C. Wang, Marcin W. Wlodarski, Heng Wang, Susanne Holzhauer, Vicky R. Breakey, Joachim Kunz, Sujit Sheth, Melissa J. Rose, Heather A. Bradeen, Nolan Neu, Dongjing Guo, Hasan Al-Sayegh, Wendy B. London, Patrick G. Gallagher, Alberto Zanella, Wilma Barcellini
Journal: Blood
Blood (2018) 131 (20): 2183–2192.
https://doi.org/10.1182/blood-2017-10-810796
Published: 2018
Includes: Supplemental data
Journal Articles

Genotype-Phenotype Correlation and Molecular Heterogeneity in Pyruvate Kinase Deficiency: Data from the PKD Natural History Study

Free
Paola Bianchi, BSc, PhD, Elisa Fermo, PhD, Kimberly Lezon-Geyda, PhD, Patrick G. Gallagher, MD, Richard van Wijk, PhD, Eduard J. Van Beers, MD, Hassan M. Yaish, MD, Janet L. Kwiatkowski, MD, Peter E. Newburger, MD, Yaddanapudi Ravindranath, MBBS, Oliver Andres, MD, Wilma Barcellini, MD, D. Holmes Morton, MD, Bertil Glader, MD, Stefan W. Eber, MD, Satheesh Chonat, MD, Nina Kollmar, MD, Jenny M. Despotovic, DO, MS, Christine M. Knoll, MD, Kevin H.M. Kuo, MD MSc, FRCPC, Dagmar Pospisilova, Prof. MD PhD, Yves D. Pastore, MD, Alexis A Thompson, MD MPH, Winfred Wang, MD, Marcin W. Wlodarski, MD PhD, Jennifer A. Rothman, MD, Heng Wang, MD PhD, Suzanne Holzhauer, MD MSc, Vicky R. Breakey, BSc,MDFRCPC, Joachim B. Kunz, MD, Sujit Sheth, MD, Mukta Sharma, MD MPH, Melissa J. Rose, DO, Heather A. Bradeen, MD, Melissa N. McNaull, MD, Gabrielle P. Ooyama, Nolan Neu, BS, Hasan Al-Sayegh, Wendy B. London, PhD, Rachael F. Grace, MD
Journal: Blood
Blood (2017) 130 (Supplement 1): 3479.
https://doi.org/10.1182/blood.V130.Suppl_1.3479.3479
Published: 2017
Journal Articles

Health Related Quality of Life and Fatigue Improve on Second Line Treatments in Pediatric Immune Thrombocytopenia (ITP)

Free
Rachael F. Grace, MD, Robert J Klaassen, MD FRCPC, Rukhmi Bhat, MDMS, Michelle Neier, MD, Melissa J. Rose, DO, Jenny M. Despotovic, DO, MS, Carolyn M. Bennett, MD MSc, James B. Bussel, MD, Jennifer A. Rothman, MD, Vicky R. Breakey, BSc,MDFRCPC, Kerry Hege, MD, Kristin A. Shimano, MD, Kristina M. Haley, DO, Amy E. Geddis, MD, George R. Buchanan, MD, Adonis Lorenzano, MD, Cindy Neunert, MD, Michael R. Jeng, MD, Yves D. Pastore, MD, Shelley Crary, MD MS, Ellis J Neufeld, MD PhD, Nolan Neu, BS, Peter Forbes, BS, Michele P. Lambert, MD
Journal: Blood
Blood (2017) 130 (Supplement 1): 752.
https://doi.org/10.1182/blood.V130.Suppl_1.752.752
Published: 2017