Sickle Cell Disease in Children and Adolescents: A Review of the Historical, Clinical, and Public Health Perspective of Sub‐Saharan Africa and Beyond
Sickle cell disease (SCD) is an umbrella term for a group of life‐long debilitating autosomal
recessive disorders that are caused by a single‐point mutation (Glu→ Val) that results in …
recessive disorders that are caused by a single‐point mutation (Glu→ Val) that results in …
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 …
such as painful vaso-occlusive episodes, acute chest syndrome, stroke, chronic anemia, and …
The evolving treatment landscape for children with sickle cell disease
Sickle cell disease is the most common inherited pathological haemoglobinopathy. Over the
past 30 years, disease-related morbidity and mortality have improved in high-income …
past 30 years, disease-related morbidity and mortality have improved in high-income …
Sickle cell disease in the new era: advances in drug treatment
MT Lee, UO Ogu - Transfusion and Apheresis Science, 2022 - Elsevier
Sickle cell disease is an inherited blood disorder afflicting an estimated 100,000 individuals
in the United States and over 20 million people worldwide. The disease is heralded as the …
in the United States and over 20 million people worldwide. The disease is heralded as the …
Managing the cerebrovascular complications of sickle cell disease: current perspectives
J Light, M Boucher, J Baskin-Miller… - Journal of Blood …, 2023 - Taylor & Francis
The importance of protecting brain function for people with sickle cell disease (SCD) cannot
be overstated. SCD is associated with multiple cerebrovascular complications that threaten …
be overstated. SCD is associated with multiple cerebrovascular complications that threaten …
Gene therapy for hemoglobinopathies
MR Lidonnici, S Scaramuzza, G Ferrari - Human Gene Therapy, 2023 - liebertpub.com
β-Thalassemia and sickle cell disease are autosomal recessive disorders of red blood cells
due to mutations in the adult β-globin gene, with a worldwide diffusion. The severe forms of …
due to mutations in the adult β-globin gene, with a worldwide diffusion. The severe forms of …
An update review of new therapies in sickle cell disease: the prospects for drug combinations
S Lugthart, C Ginete, P Kuona, M Brito… - Expert Opinion on …, 2024 - Taylor & Francis
Introduction Sickle cell disease (SCD) is an inherited disorder characterised by
polymerisation of deoxygenated haemoglobin S and microvascular obstruction. The cardinal …
polymerisation of deoxygenated haemoglobin S and microvascular obstruction. The cardinal …
Systemic medications for sickle cell disease and potential applications for sickle cell retinopathy
Hydroxyurea has been a mainstay of systemic management of SCD with prior work
suggesting its ability to reduce the likelihood of developing retinopathy. There are several …
suggesting its ability to reduce the likelihood of developing retinopathy. There are several …
Voxelotor for the treatment of sickle cell disease in pediatric patients
C Brown, M Tonda, MR Abboud - Expert Review of Hematology, 2022 - Taylor & Francis
Introduction Sickle cell disease (SCD) describes a group of heritable blood disorders
caused by the polymerization of sickle hemoglobin (HbS). HbS polymerization leads to …
caused by the polymerization of sickle hemoglobin (HbS). HbS polymerization leads to …
An integrated therapeutic approach to sickle cell disease management beyond infancy
JA Ribeil, G Pollock, H Frangoul… - American Journal of …, 2023 - Wiley Online Library
Hydroxyurea, the first approved drug for sickle cell disease, decreases sickle hemoglobin
polymerization by inducing fetal hemoglobin. Its effects in young children are excellent; …
polymerization by inducing fetal hemoglobin. Its effects in young children are excellent; …