[HTML][HTML] 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 …

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 …

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; …

Use of disease-modifying treatments in patients with sickle cell disease

TV Newman, J Yang, K Suh, CR Jonassaint… - JAMA Network …, 2023 - jamanetwork.com
Importance Despite hydroxyurea being an established treatment for sickle cell disease
(SCD), it remains underused. The recent approval of the disease-modifying treatments …

Evidence-Based Minireview: How to utilize new therapies for sickle cell disease

S Guarino, S Lanzkron - Hematology, 2022 - ashpublications.org
A 28-year-old man with sickle cell dis ease (SCD) with geno type hemo glo bin (Hgb) SS
with base line hemo globin of 8.5 pres ents to estab lish care with the adult sickle cell pro …

Practical guidance for the use of voxelotor in the management of sickle cell disease

CM Barriteau, SM Badawy - Journal of Blood Medicine, 2022 - Taylor & Francis
Sickle cell disease (SCD) is one of the most common inherited blood disorders.
Deoxygenated hemoglobin S (HbS) polymerizes and causes anemia and various end organ …

New insights into the pathophysiology and novel therapies for sickle cell disease

RD Cancado - Medical Research Archives, 2024 - esmed.org
Sickle cell disease (SCD) is one of the world's most common severe monogenic disorders
affecting millions of people worldwide and represents a significant public health problem …