Food and Drug Administration Approval of Glutamine for Sickle Cell Disease: Success and Precautions in Glutamine Research

2017 ◽  
Vol 41 (6) ◽  
pp. 912-917 ◽  
Author(s):  
Douglas W. Wilmore
Keyword(s):  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Drug Administration ◽  
Food And Drug Administration ◽  
Cell Disease

scholarly journals Vaso-occlusive crisis in sickle cell disease: a vicious cycle of secondary events

2021 ◽  
Vol 19 (1) ◽  
Author(s):  
Tim Jang ◽  
Maria Poplawska ◽  
Emanuela Cimpeanu ◽  
George Mo ◽  
Dibyendu Dutta ◽  
...  
Keyword(s):  
Emergency Department ◽  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Drug Administration ◽  
Food And Drug Administration ◽  
Polymerization Process ◽  
Vicious Cycle ◽  
Cell Disease ◽  
New Agents ◽  
Combination Approach

AbstractPainful vaso-occlusive crisis (VOC) remains the most common reason for presenting to the Emergency Department and hospitalization in patients with sickle cell disease (SCD). Although two new agents have been approved by the Food and Drug Administration for treating SCD, they both target to reduce the frequency of VOC. Results from studies investigating various approaches to treat and shorten VOC have so far been generally disappointing. In this paper, we will summarize the complex pathophysiology and downstream events of VOC and discuss the likely reasons for the disappointing results using monotherapy. We will put forward the rationale for exploring some of the currently available agents to either protect erythrocytes un-involved in the hemoglobin polymerization process from sickling induced by the secondary events, or a multipronged combination approach that targets the complex downstream pathways of VOC.

scholarly journals Voxelotor: A Hemoglobin S Polymerization Inhibitor for the Treatment of Sickle Cell Disease

2020 ◽  
Vol 11 (8) ◽  
Author(s):  
Avani Yenamandra, PharmD ◽  
Dennis Marjoncu, PharmD, BCOP
Keyword(s):  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Drug Administration ◽  
Pediatric Patients ◽  
Cell Disease ◽  
Hemoglobin S ◽  
Polymerization Inhibitor ◽  
The World ◽  
Fundamental Cause ◽  
The U.S

Sickle cell disease (SCD) affects millions of people throughout the world. Hemoglobin S (HbS) polymerization is the fundamental cause of SCD pathophysiology, which leads to hemolysis, increased viscosity, and acute vaso-occlusive episodes. Novel agents have been developed to target the pathophysiology of SCD and decrease the frequency of SCD complications. Voxelotor (Oxbryta) is an HbS polymerization inhibitor that is approved by the U.S. Food & Drug Administration for the treatment of SCD in adults and pediatric patients 12 years and older.

Hemolysis in sickle cell disease

1974 ◽  
Vol 133 (4) ◽  
pp. 624-631 ◽  
Author(s):  
T. A. Bensinger
Keyword(s):  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Cell Disease

Computerized cognitive training in pediatric sickle cell disease: A randomized controlled pilot study.

2020 ◽  
Vol 8 (4) ◽  
pp. 390-401 ◽  
Author(s):  
Taryn M. Allen ◽  
Lindsay M. Anderson ◽  
Samuel M. Brotkin ◽  
Jennifer A. Rothman ◽  
Melanie J. Bonner
Keyword(s):  
Pilot Study ◽  
Sickle Cell Disease ◽  
Cognitive Training ◽  
Sickle Cell ◽  
Cell Disease ◽  
Computerized Cognitive Training ◽  
Randomized Controlled ◽  
Pediatric Sickle Cell Disease
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