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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Bibliographische Detailangaben
Hauptverfasser:	Gustafson, Gary, Han, Bingsong, Smith, Chase C, Mitchell, Lorna, Richard, David, Lancia, Jr., David R, Ericsson, Anna, Green, Neal, Shelekhin, Tatiana, Wang, Zhongguo, Zheng, Xiaozhang
Format:	Patent
Sprache:	eng
Schlagworte:	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
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Beschreibung
Zusammenfassung:	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.