The evolving treatment landscape for children with sickle cell disease

SA Jacob, R Talati, J Kanter - The Lancet Child & Adolescent Health, 2023 - thelancet.com
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 …

[HTML][HTML] New therapeutic options for the treatment of sickle cell disease

A Matte, F Zorzi, F Mazzi, E Federti… - … journal of hematology …, 2019 - ncbi.nlm.nih.gov
Sickle cell disease (SCD; ORPHA232; OMIM# 603903) is a chronic and invalidating
disorder distributed worldwide, with high morbidity and mortality. Given the disease …

Biophysical chemistry behind sickle cell anemia and the mechanism of voxelotor action

M Suhail - Scientific Reports, 2024 - nature.com
Sickle cell anemia disease has been a great challenge to the world in the present situation.
It occurs only due to the polymerization of sickle hemoglobin (HbS) having Pro–Val–Glu …

Time domains of hypoxia responses and-omics insights

JJ Yu, AL Non, EC Heinrich, W Gu, J Alcock… - Frontiers in …, 2022 - frontiersin.org
The ability to respond rapidly to changes in oxygen tension is critical for many forms of life.
Challenges to oxygen homeostasis, specifically in the contexts of evolutionary biology and …

Systematic review of voxelotor: a first‐in‐class sickle hemoglobin polymerization inhibitor for management of sickle cell disease

J Han, SL Saraf, VR Gordeuk - Pharmacotherapy: The Journal …, 2020 - Wiley Online Library
Voxelotor, a sickle hemoglobin polymerization inhibitor, was approved by the US Food and
Drug Administration to treat sickle cell disease (SCD) in November 2019. This article …

Emerging drugs in randomized controlled trials for sickle cell disease: are we on the brink of a new era in research and treatment?

A Matte, MD Cappellini, A Iolascon… - Expert Opinion on …, 2020 - Taylor & Francis
Introduction: Sickle cell disease (SCD) is caused by a mutation in the HBB gene which is key
for making a component of hemoglobin. The mutation leads to the formation of an abnormal …

[HTML][HTML] Voxelotor (GBT440) produces interference in measurements of hemoglobin S

NJ Rutherford, KL Thoren, Z Shajani-Yi… - Clinica chimica acta; …, 2018 - ncbi.nlm.nih.gov
A 35-y-old African-American male presented to the emergency department at Vanderbilt
University Medical Center for treatment of sickle cell pain crisis. An emergent red blood cell …

The enigma of sickle cell hepatopathy: Pathophysiology, clinical manifestations and therapy

I Rizvi, D Solipuram, N Kaur, A Komel… - British Journal of …, 2024 - Wiley Online Library
Sickle cell disease (SCD) is one of the most common genetic disorders in the world
predominantly affecting economically disadvantaged populations. There is a notable …

New and emerging treatments for vaso-occlusive pain in sickle cell disease

C Dampier - Expert Review of Hematology, 2019 - Taylor & Francis
Introduction: Acute pain from episodic vaso-occlusion (VOC) spans the lifespan of almost
everyone with sickle cell disease (SCD), while additional chronic pain develops in …

Case report: safety and efficacy of voxelotor in a patient with sickle cell disease and stage IV chronic kidney disease

A Alshurafa, MA Yassin - Frontiers in Medicine, 2022 - frontiersin.org
Sickle cell disease (SCD) is a heterogeneous group of inherited disorders characterized by
the production of sickle hemoglobin which is less soluble than an adult or fetal hemoglobin …