Defining global strategies to improve outcomes in sickle cell disease: a Lancet Haematology Commission

FB Piel, DC Rees, MR DeBaun, O Nnodu… - The Lancet …, 2023 - thelancet.com
Executive summary All over the world, people with sickle cell disease (an inherited
condition) have premature deaths and preventable severe chronic complications, which …

Oxygen delivery in the treatment of anemia

HF Bunn - New England Journal of Medicine, 2022 - Mass Medical Soc
Oxygen Delivery in the Treatment of Anemia | New England Journal of Medicine Skip to main
content The New England Journal of Medicine homepage Advanced Search SEARCH …

Treatment of sickle cell disease by increasing oxygen affinity of hemoglobin

ER Henry, B Metaferia, Q Li, J Harper… - Blood, The Journal …, 2021 - ashpublications.org
The issue of treating sickle cell disease with drugs that increase hemoglobin oxygen affinity
has come to the fore with the US Food and Drug Administration approval in 2019 of …

A phase 1 dose escalation study of the pyruvate kinase activator mitapivat (AG-348) in sickle cell disease

JZ Xu, A Conrey, I Frey, E Gwaabe… - Blood, The Journal …, 2022 - ashpublications.org
Polymerization of deoxygenated hemoglobin S underlies the pathophysiology of sickle cell
disease (SCD). In activating red blood cell pyruvate kinase and glycolysis, mitapivat (AG …

Safety and efficacy of voxelotor in pediatric patients with sickle cell disease aged 4 to 11 years

JH Estepp, R Kalpatthi, G Woods… - Pediatric blood & …, 2022 - Wiley Online Library
Background Sickle cell disease (SCD) is a devastating, multisystemic disorder that affects
millions of people worldwide. The earliest clinical manifestations of SCD can affect infants as …

Genotypic and phenotypic composition of sickle cell disease in the Arab population-a systematic review

F Ata, A Rahhal, L Malkawi, P Iqbal… - Pharmacogenomics …, 2023 - Taylor & Francis
Sickle cell disease (SCD) is a genetic disease influenced by ethnicity and regional
differences in its clinical course. Recent advances in the management of SCD with newer …

Signaling mechanisms in red blood cells: A view through the protein phosphorylation and deformability

N Cilek, E Ugurel, E Goksel… - Journal of Cellular …, 2024 - Wiley Online Library
Intracellular signaling mechanisms in red blood cells (RBCs) involve various protein kinases
and phosphatases and enable rapid adaptive responses to hypoxia, metabolic …

Real-world effectiveness of voxelotor for treating sickle cell disease in the US: a large claims data analysis

N Shah, T Lipato, O Alvarez, T Delea… - Expert review of …, 2022 - Taylor & Francis
Background Sickle cell disease (SCD) is a genetic disease that impacts patients' quality of
life, healthcare costs, and life expectancy. Elevated sickle hemoglobin (HbS), which readily …

Recent advances in sickle-cell disease therapies: a review of voxelotor, crizanlizumab, and L-glutamine

M Migotsky, M Beestrum, SM Badawy - Pharmacy, 2022 - mdpi.com
Sickle-cell disease (SCD) is an inherited hemoglobinopathy, causing lifelong complications
such as painful vaso-occlusive episodes, acute chest syndrome, stroke, chronic anemia, and …

CRISPR/Cas9-based gene-editing technology for sickle cell disease

L Ma, S Yang, Q Peng, J Zhang, J Zhang - Gene, 2023 - Elsevier
Sickle cell disease (SCD) is the most common monogenic hematologic disorder and is
essentially congenital hemolytic anemia caused by an inherited point mutation in the β …