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Pharmacokinetics and pharmacodynamics of voxelotor (GBT440) in healthy adults and patients with sickle cell disease.

Author: AllenElizabeth, GretlerDaniel D, HutchaleelahaAthiwat, Lehrer-GraiwerJosh, MantTimothy, OksenbergDonna, PatelMira, SiuVincent, WashingtonCarla

Randomized Controlled TrialA clinical trial that randomly assigns participants to treatment and control groups to evaluate the effects of an intervention.
Clinical Trial, Phase IThe first stage of clinical trials in humans. Evaluates the safety, tolerability, and pharmacokinetics of a new drug/treatment.
Clinical Trial, Phase IIA Phase II trial is a trial that further evaluates the efficacy and safety of a new drug or treatment, whose safety has been confirmed to some extent in a Phase I trial, in a small number of patients.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Multicenter StudyResearch conducted in collaboration with multiple research institutions. It aims to collect a wide range of data and obtain more generalizable results.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Overview

Voxelotor, a haemoglobin (Hb) modulator for sickle cell disease (SCD), showed promising safety, tolerability, pharmacokinetics, and pharmacodynamics profiles in both healthy volunteers and SCD patients.
Paper Details　
Original Abstract of the Article :
Voxelotor (previously GBT440) is a haemoglobin (Hb) modulator that increases Hb-oxygen affinity, thereby reducing Hb polymerization and sickling of red blood cells (RBCs), being developed as a once-daily oral drug to treat sickle cell disease (SCD). This first-in-human study evaluated the safety, to...See full text at original site
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引用元：
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6533444/

データ提供：米国国立医学図書館（NLM）

Voxelotor: A New Hope for Sickle Cell Disease

Sickle cell disease (SCD) is a debilitating genetic disorder that affects red blood cells, leading to chronic pain, organ damage, and a shortened lifespan. This research investigates the potential of voxelotor, a novel Hb modulator, as a potential treatment for SCD. The study aimed to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of voxelotor in both healthy volunteers and SCD patients. The researchers used a first-in-human study design to assess the effects of voxelotor on the body, providing essential data for further development.

The study showed that voxelotor was well tolerated and demonstrated its ability to increase Hb-oxygen affinity, a key factor in reducing the sickling of red blood cells. This could potentially lead to a reduction in the frequency and severity of SCD-related complications. The researchers also noted that voxelotor has a convenient once-daily oral dosing regimen, which could significantly improve patient compliance and quality of life.

A Potential Breakthrough in SCD Treatment

The promising results of this study suggest that voxelotor has the potential to be a transformative treatment for SCD. Its ability to reduce sickling of red blood cells and the convenience of once-daily oral dosing hold significant promise for improving the lives of SCD patients. This research offers a ray of hope for those living with this challenging disease.

A New Era for SCD Patients

As a researcher deeply interested in finding solutions for debilitating conditions, I am encouraged by the progress being made in the treatment of SCD. The development of voxelotor represents a significant step forward in this field. I am optimistic about the future of SCD treatment, and I believe that this research provides a promising pathway towards a better quality of life for those affected by this disease.

Dr. Camel's Conclusion

Voxelotor holds great promise for SCD patients, demonstrating its ability to reduce sickling, improve Hb-oxygen affinity, and potentially lessen the severity of SCD-related complications. The convenience of a once-daily oral dosing regimen further enhances its potential to significantly improve the lives of those living with SCD.

Date :
  1. Date Completed 2020-04-13
  2. Date Revised 2021-01-09
Further Info :

Pubmed ID

30743314

DOI: Digital Object Identifier

PMC6533444

Related Literature

Article Analysis
・Effects of voxelotor
SNS
PICO Info
in preparation
Languages

English

Positive IndicatorAn AI analysis index that serves as a benchmark for how positive the results of the study are. Note that it is a benchmark and requires careful interpretation and consideration of different perspectives.

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