Work

FT-4202, an Allosteric Activator of Pyruvate Kinase-R, Demonstrates Proof of Mechanism and Proof of Concept after a Single Dose and after Multiple Daily Doses in a Phase 1 Study of Patients with Sickle Cell Disease

Year: 2020

Type: article

Study design: Clinical Trial

Source: Blood

DOI: 10.1182/blood-2020-134269

Authors: R. Clark Brown, Kimberly Cruz, Theodosia A. Kalfa, Frans A. Kuypers, Santosh L. Saraf, Jeremie H. Estepp, Luke R. Smart, Punam Malik, Mark Lerman, Renae Mayer, Maria D. Ribadeneira, Sanjeev Forsyth, Patricia Schroeder, Eric Wu, Patrick Kelly, Marilyn J. Telen

Institutions: Emory University, Children's Healthcare of Atlanta, Advanced Pharma, Cincinnati Children's Hospital Medical Center, University of Illinois Chicago, St. Jude Children's Research Hospital, Atlanta Center for Medical Research, Forma Therapeutics (United States), Duke University

Language: English

Topic: Hemoglobinopathies and Related Disorders

Subfield: Genetics (clinical)

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: closed

OpenAlex ID: W3095200845

FWCI: 2.416

Cites:

Cited by: 16

Related to: 10