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Schwartz, J.D
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Schwartz, J.D
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Who should be eligible for gene therapy clinical trials in red blood cell pyruvate kinase deficiency (PKD)?: Toward an expanded definition of severe PKD
by
Barcellini, W.
,
Bianchi, P.
,
Glader, B.
,
Grace, R.F
,
López Lorenzo, J.L
,
Navarro Ordoñez, S.
,
Nicoletti, E.
,
Schwartz
,
J.D
,
Segovia, J.C
,
Sevilla, J.
,
Shah, A.J
,
Zanella, A.
Published 2022
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Related Subjects
Anemia, Hemolytic, Congenital Nonspherocytic
CD34 antigen
Clinical Trials as Topic
Genetic Therapy
Humans
Letter
PKLR gene
Pyruvate Kinase
Pyruvate Metabolism, Inborn Errors
RNA splice site
adenosine triphosphate
allogeneic hematopoietic stem cell transplantation
anemia
attitude to illness
bilirubin
blood transfusion
clinical effectiveness
clinical feature
clinical trial (topic)
diagnostic procedure
disease burden
disease classification
disease severity
disorders of carboxylic acid metabolism
enzymology
erythrocyte disorder
erythropoietin
fatigue
follow up
gene
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