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 …
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 …
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 …
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 …
Real-world effectiveness of voxelotor for treating sickle cell disease in the US: a large claims data analysis
Background Sickle cell disease (SCD) is a genetic disease that impacts patients' quality of
life, healthcare costs, and life expectancy. Elevated sickle hemoglobin (HbS), which readily …
life, healthcare costs, and life expectancy. Elevated sickle hemoglobin (HbS), which readily …
[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 …
Real‐life experience with hydroxyurea in patients with sickle cell disease: results from the prospective ESCORT‐HU cohort study
M de Montalembert, E Voskaridou… - American journal of …, 2021 - Wiley Online Library
Several controlled studies have evidenced good efficacy and short‐term and mid‐term
safety profiles for hydroxyurea (HU), which has become the cornerstone for prevention of …
safety profiles for hydroxyurea (HU), which has become the cornerstone for prevention of …
Five years of experience with hydroxyurea in children and young adults with sickle cell disease
A Ferster, P Tahriri, C Vermylen… - Blood, The Journal …, 2001 - ashpublications.org
The short-term beneficial effect of hydroxyurea (HU) in sickle cell disease (SCD) has been
proven by randomized studies in children and adults. The Belgian registry of HU-treated …
proven by randomized studies in children and adults. The Belgian registry of HU-treated …
[HTML][HTML] The evolving pharmacotherapeutic landscape for the treatment of sickle cell disease
SK Ballas - Mediterranean Journal of Hematology and Infectious …, 2020 - ncbi.nlm.nih.gov
Sickle cell disease (SCD) is an extremely heterogeneous disease that has been associated
with global morbidity and early mortality. More effective and inexpensive therapies are …
with global morbidity and early mortality. More effective and inexpensive therapies are …
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