JCS/JSCS 2020 guideline on diagnosis and management of cardiovascular sequelae in Kawasaki disease

R Fukazawa, J Kobayashi, M Ayusawa… - Circulation …, 2020 - jstage.jst.go.jp
More than 50 years have passed since Kawasaki disease (KD) was first reported. 1
According to a nationwide survey, the number of patients with KD who became adults was …

Sickle cell disease

GJ Kato, FB Piel, CD Reid, MH Gaston… - Nature reviews Disease …, 2018 - nature.com
Sickle cell disease (SCD) is a group of inherited disorders caused by mutations in HBB,
which encodes haemoglobin subunit β. The incidence is estimated to be between 300,000 …

[HTML][HTML] Crizanlizumab for the prevention of pain crises in sickle cell disease

KI Ataga, A Kutlar, J Kanter, D Liles… - … England Journal of …, 2017 - Mass Medical Soc
Background The up-regulation of P-selectin in endothelial cells and platelets contributes to
the cell–cell interactions that are involved in the pathogenesis of vaso-occlusion and sickle …

Recent advances in the treatment of sickle cell disease

G Salinas Cisneros, SL Thein - Frontiers in physiology, 2020 - frontiersin.org
Sickle cell anemia (SCA) was first described in the Western literature more than 100 years
ago. Elucidation of its molecular basis prompted numerous biochemical and genetic studies …

Comparison of US federal and foundation funding of research for sickle cell disease and cystic fibrosis and factors associated with research productivity

F Farooq, PJ Mogayzel, S Lanzkron… - JAMA network …, 2020 - jamanetwork.com
Importance Sickle cell disease (SCD) and cystic fibrosis (CF) are severe autosomal
recessive disorders associated with intermittent disease exacerbations that require …

Inflammation in sickle cell disease

N Conran, JD Belcher - Clinical hemorheology and …, 2018 - content.iospress.com
The primary β-globin gene mutation that causes sickle cell disease (SCD) has significant
pathophysiological consequences that result in hemolytic events and the induction of the …

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 …

Treating sickle cell disease by targeting HbS polymerization

WA Eaton, HF Bunn - Blood, The Journal of the American …, 2017 - ashpublications.org
Although the root cause of sickle cell disease is the polymerization of hemoglobin S (HbS) to
form fibers that make red cells less flexible, most drugs currently being assessed in clinical …

Sickle cell disease—genetics, pathophysiology, clinical presentation and treatment

BPD Inusa, LL Hsu, N Kohli, A Patel… - International journal of …, 2019 - mdpi.com
Sickle cell disease (SCD) is a monogenetic disorder due to a single base-pair point mutation
in the β-globin gene resulting in the substitution of the amino acid valine for glutamic acid in …

Therapeutic strategies for sickle cell disease: towards a multi-agent approach

MJ Telen, P Malik, GM Vercellotti - Nature reviews Drug discovery, 2019 - nature.com
For over 100 years, clinicians and scientists have been unravelling the consequences of the
A to T substitution in the β-globin gene that produces haemoglobin S, which leads to the …