A phase 3 randomized trial of voxelotor in sickle cell disease
E Vichinsky, CC Hoppe, KI Ataga… - … England Journal of …, 2019 - Mass Medical Soc
Abstract Background Deoxygenated sickle hemoglobin (HbS) polymerization drives the
pathophysiology of sickle cell disease. Therefore, direct inhibition of HbS polymerization has …
pathophysiology of sickle cell disease. Therefore, direct inhibition of HbS polymerization has …
A phase 1/2 ascending dose study and open-label extension study of voxelotor in patients with sickle cell disease
J Howard, CJ Hemmaway, P Telfer… - Blood, The Journal …, 2019 - ashpublications.org
New treatments directly targeting polymerization of sickle hemoglobin (HbS), the proximate
event in the pathophysiology of sickle cell disease (SCD), are needed to address the severe …
event in the pathophysiology of sickle cell disease (SCD), are needed to address the severe …
Voxelotor: first approval
HA Blair - Drugs, 2020 - Springer
Voxelotor (Oxbryta™) is a haemoglobin S polymerization inhibitor that has been developed
for the treatment of sickle cell disease. In November 2019, voxelotor received its first global …
for the treatment of sickle cell disease. In November 2019, voxelotor received its first global …
Voxelotor in adolescents and adults with sickle cell disease (HOPE): long-term follow-up results of an international, randomised, double-blind, placebo-controlled …
J Howard, KI Ataga, RC Brown, M Achebe… - The Lancet …, 2021 - thelancet.com
Background For decades, patients with sickle cell disease have had only a limited number of
therapies available. In 2019, voxelotor (1500 mg), an oral once-daily sickle haemoglobin …
therapies available. In 2019, voxelotor (1500 mg), an oral once-daily sickle haemoglobin …
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 …
millions of people worldwide. The earliest clinical manifestations of SCD can affect infants as …
Pharmacokinetics and pharmacodynamics of voxelotor (GBT440) in healthy adults and patients with sickle cell disease
A Hutchaleelaha, M Patel, C Washington… - British journal of …, 2019 - Wiley Online Library
Aims Voxelotor (previously GBT440) is a haemoglobin (Hb) modulator that increases Hb‐
oxygen affinity, thereby reducing Hb polymerization and sickling of red blood cells (RBCs) …
oxygen affinity, thereby reducing Hb polymerization and sickling of red blood cells (RBCs) …
Treating sickle cell disease by targeting HbS polymerization
WA Eaton, HF Bunn - Blood, The Journal of the American …, 2017 - ashpublications.org
Although the root cause of sickle cell disease is the polymerization of hemoglobin S (HbS) to
form fibers that make red cells less flexible, most drugs currently being assessed in clinical …
form fibers that make red cells less flexible, most drugs currently being assessed in clinical …
Systematic review: Hydroxyurea for the treatment of adults with sickle cell disease
Background: Hydroxyurea is the only approved drug for treatment of sickle cell disease.
Objective: To synthesize the published literature on the efficacy, effectiveness, and toxicity of …
Objective: To synthesize the published literature on the efficacy, effectiveness, and toxicity of …
[HTML][HTML] Efficacy and safety of recently approved drugs for sickle cell disease: a review of clinical trials
Highlights•VOC episodes and low hemoglobin levels are major complications of SCD.•L-
Glutamine decreases the episodes of VOC.•Voxelotor increases hemoglobin levels and …
Glutamine decreases the episodes of VOC.•Voxelotor increases hemoglobin levels and …
GBT 440 increases haemoglobin oxygen affinity, reduces sickling and prolongs RBC half‐life in a murine model of sickle cell disease
D Oksenberg, K Dufu, MP Patel… - British journal of …, 2016 - Wiley Online Library
A major driver of the pathophysiology of sickle cell disease (SCD) is polymerization of
deoxygenated haemoglobin S (HbS), which leads to sickling and destruction of red blood …
deoxygenated haemoglobin S (HbS), which leads to sickling and destruction of red blood …
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