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Article
Open AccessEvaluating Gene Therapy as a Potential Paradigm Shift in Treating Severe Hemophilia
Hemophilia is characterized by a deficiency in coagulation factors VIII or IX. The general standard of care for severe hemophilia is frequent intravenous recombinant or plasma-derived factor replacement to pre...
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Article
Correction: Genetic predictors of severe intraventricular hemorrhage in extremely low-birthweight infants
An amendment to this paper has been published and can be accessed via a link at the top of the paper.
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Article
Genetic predictors of severe intraventricular hemorrhage in extremely low-birthweight infants
To test associations between grades 3 or 4 (severe) intraventricular hemorrhage (IVH) and single nucleotide polymorphisms (SNPs) associated with coagulation, inflammation, angiogenesis, and organ development i...
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Article
Open AccessA 2-year-old girl with co-inherited cystic fibrosis and sickle cell-β+ thalassemia presenting with recurrent vaso-occlusive events during cystic fibrosis pulmonary exacerbations: a case report
This is the first published report of a young girl with co-inherited sickle cell-β+ thalassemia and cystic fibrosis. Although a small subset of patients with co-inherited cystic fibrosis and other hemoglobinopath...