Treating sickle cell disease with a pyruvate kinase R activating compound

Compounds that activate pyruvate kinase R can be used for the treatment of sickle cell disease (SCD). Methods and compositions for the treatment of SCD are provided herein, including a therapeutic compound designated as Compound 1.

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Hauptverfasser: Gustafson, Gary, Han, Bingsong, Smith, Chase C, Mitchell, Lorna, Richard, David, Lancia, Jr., David R, Ericsson, Anna, Green, Neal, Wang, Zhongguo, Shelekhin, Tatiana, Zheng, Xiaozhang
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creator Gustafson, Gary
Han, Bingsong
Smith, Chase C
Mitchell, Lorna
Richard, David
Lancia, Jr., David R
Ericsson, Anna
Green, Neal
Wang, Zhongguo
Shelekhin, Tatiana
Zheng, Xiaozhang
description Compounds that activate pyruvate kinase R can be used for the treatment of sickle cell disease (SCD). Methods and compositions for the treatment of SCD are provided herein, including a therapeutic compound designated as Compound 1.
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subjects CHEMISTRY
HETEROCYCLIC COMPOUNDS
HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
METALLURGY
ORGANIC CHEMISTRY
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS
title Treating sickle cell disease with a pyruvate kinase R activating compound
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