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; …

Effective therapies for sickle cell disease: are we there yet?

M Crossley, GE Christakopoulos, MJ Weiss - Trends in Genetics, 2022 - cell.com
Sickle cell disease (SCD) is a common genetic blood disorder associated with acute and
chronic pain, progressive multiorgan damage, and early mortality. Recent advances in …

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 …

One-year safety and efficacy of mitapivat in sickle cell disease: follow-up results of a phase 2, open-label study

MJ van Dijk, MAE Rab, BA van Oirschot, J Bos… - Blood …, 2023 - ashpublications.org
Targeting the primary pathogenic event of sickle cell disease (SCD), the polymerization of
sickle hemoglobin (HbS), may prevent downstream clinical events. Mitapivat, an oral …

Emerging drugs for the treatment of sickle cell disease: a review of phase II/III trials

JM Ross, S Forté, D Soulières - Expert Opinion on Emerging Drugs, 2022 - Taylor & Francis
Introduction The substitution of glutamic acid by valine on the ß-globin chain produces the
hemoglobin S variant responsible for sickle cell disease (SCD), a disorder that affects …

An expert review of voxelotor for the treatment of hemolytic anemia in patients with sickle cell disease:'Bridging the gap between laboratory data and patient related …

BPD Inusa, K Mnika, S Babiker - Expert Review of Hematology, 2023 - Taylor & Francis
Introduction Until recently, the treatment of sickle cell disease (SCD) for a long time has
been limited to hydroxycarbamide alone. SCD is characterized by hemoglobin (Hb) …

[引用][C] More of the same? Voxelotor spawns a successor, but on what success does it build?

FA Ferrone - British journal of haematology, 2023 - drexel.esploro.exlibrisgroup.com
Voxelotor is designed to impeded the polymerization of sickle haemoglobin. Although FDA-
approved, its place in the treatment of sickle cell anaemia remains controversial. A report in …

[HTML][HTML] Influence of Voxelotor–hemoglobin complexes in the estimation of hemoglobin S levels by the current standard of care laboratory evaluation techniques

S Alkindi, A Al Subhi, AEH Ali, AV Pathare - Frontiers in Medicine, 2023 - frontiersin.org
Background Sickle cell disease is an inherited disorder characterized by the presence of
sickle hemoglobin (HbS). The process of Hb molecule polymerization is a pivotal step in the …

[HTML][HTML] Recent progress in the treatment of sickle cell disease: an up-to-date review

B Acharya, DP Mishra, B Barik, RK Mohapatra… - Beni-Suef University …, 2023 - Springer
Background Sickle cell disease is a fatal systemic condition characterized by acute painful
episodes, persistent anemia, ongoing organ damage, organ infarction, and a markedly …

A clinician's view of voxelotor.

I Osunkwo, A Anderson, RC Brown… - British Journal of …, 2022 - search.ebscohost.com
In particular, diarrhoea occurred in 23% of patients receiving voxelotor 1500 mg in the
HOPE trial, more than double the frequency observed with placebo (11%). 19 Pro-active …