More of the same? Voxelotor spawns a successor, but on what success does it build?
FA Ferrone - British journal of haematology, 2023 - researchdiscovery.drexel.edu
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 …
approved, its place in the treatment of sickle cell anaemia remains controversial. A report in …
S268: safety, tolerability, and pharmacokinetic/pharmacodynamic results from phase 1 studies of GBT021601, a next-generation HbS polymerization inhibitor for …
C Brown, C Key, I Agodoa, J Olbertz, K Duchin… - …, 2022 - journals.lww.com
Background: Sickle cell disease (SCD) is caused by polymerization of sickle hemoglobin
(HbS). Voxelotor is a first-in-class HbS polymerization inhibitor approved by the United …
(HbS). Voxelotor is a first-in-class HbS polymerization inhibitor approved by the United …
Individual red blood cell fetal hemoglobin quantification allows to determine protective thresholds in sickle cell disease
N Hebert, MG Rakotoson, G Bodivit… - American Journal of …, 2020 - Wiley Online Library
Polymerization of the sickle hemoglobin (HbS) is a key determinant of sickle cell disease
(SCD), an inherited blood disorder. Fetal hemoglobin (HbF) is a major modulator of the …
(SCD), an inherited blood disorder. Fetal hemoglobin (HbF) is a major modulator of the …
Kinetics of sickle hemoglobin polymerization: II. A double nucleation mechanism
FA Ferrone, J Hofrichter, WA Eaton - Journal of molecular biology, 1985 - Elsevier
A double nucleation mechanism for the polymerization of sickle hemoglobin is described.
The mechanism accounts for all of the major kinetic observations: the appearance of a …
The mechanism accounts for all of the major kinetic observations: the appearance of a …
[HTML][HTML] GTx011, a potent allosteric modifier of hemoglobin oxygen affinity, prevents RBC sickling in whole blood and prolongs RBC half-life in vivo in a murine model …
K Dufu, D Oksenberg, C Zhou, A Hutchaleelaha… - Blood, 2014 - Elsevier
Sickle cell disease (SCD) is caused by a point mutation in the β-globin gene leading to
production of hemoglobin S (HbS) that polymerizes under hypoxic conditions with …
production of hemoglobin S (HbS) that polymerizes under hypoxic conditions with …
Hemoglobin polymerization in sickle cells studied by circular polarized light scattering
We have studied intracellular polymerization of hemoglobin S in suspensions of small
populations of sickle cells using circular polarized light scattering. We argue that the …
populations of sickle cells using circular polarized light scattering. We argue that the …
Recombinant sickle hemoglobin containing a lysine substitution at Asp-85 (α): expression in yeast, functional properties, and participation in gel formation
JP Himanen, AM Popowicz… - Blood, The Journal of …, 1997 - ashpublications.org
Clinical modalities based on inhibition of gelation of HbS are hindered by the lack of
quantitative information on the extent of participation of different amino acid residues in the …
quantitative information on the extent of participation of different amino acid residues in the …
[HTML][HTML] Impact of hemoglobin biophysical studies on molecular pathogenesis and drug therapy for sickle cell disease
WA Eaton - Molecular aspects of medicine, 2022 - Elsevier
Basic research on hemoglobin has been essential for understanding the origin and
treatment of many hematological disorders due to abnormal hemoglobins. The most …
treatment of many hematological disorders due to abnormal hemoglobins. The most …
[HTML][HTML] Long-term dosing in sickle cell disease subjects with GBT440, a novel HbS polymerization inhibitor
J Lehrer-Graiwer, J Howard, CJ Hemmaway… - Blood, 2016 - Elsevier
Background: Sickle cell disease (SCD) is caused by polymerization of Hemoglobin S (HbS),
resulting in hemolysis and vaso-occlusion. No therapy achieving pancellular, direct …
resulting in hemolysis and vaso-occlusion. No therapy achieving pancellular, direct …