A Scientific Renaissance: Novel Drugs in Sickle Cell Disease

Ahmar U. Zaidi; Matthew M. Heeney

doi:10.1016/j.pcl.2018.01.006

A Scientific Renaissance: Novel Drugs in Sickle Cell Disease

Ahmar U. Zaidi, Matthew M. Heeney

Research output: Contribution to journal › Review article › peer-review

Abstract

We have entered an era of exploding interest in therapeutics for sickle cell disease. The expansion in our understanding of sickle cell disease pathophysiology has enhanced the range of potential therapeutic targets. From induction of fetal hemoglobin to antiadhesion molecules, we are potentially on the cusp of making life-altering modifications for individuals with sickle cell disease. This disease population cannot afford to let the current momentum wane. Studies exploring combinations of therapies affecting multiple steps in the pathophysiology and exploring novel and clinically relevant outcomes are incumbent.

Original language	English
Pages (from-to)	445-464
Number of pages	20
Journal	Pediatric Clinics of North America
Volume	65
Issue number	3
DOIs	https://doi.org/10.1016/j.pcl.2018.01.006
State	Published - Jun 2018
Externally published	Yes

Keywords

Antiadhesion molecules
Induction of fetal hemoglobin
Pathophysiology
Sickle cell disease
Therapeutic targets

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10.1016/j.pcl.2018.01.006

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title = "A Scientific Renaissance: Novel Drugs in Sickle Cell Disease",

abstract = "We have entered an era of exploding interest in therapeutics for sickle cell disease. The expansion in our understanding of sickle cell disease pathophysiology has enhanced the range of potential therapeutic targets. From induction of fetal hemoglobin to antiadhesion molecules, we are potentially on the cusp of making life-altering modifications for individuals with sickle cell disease. This disease population cannot afford to let the current momentum wane. Studies exploring combinations of therapies affecting multiple steps in the pathophysiology and exploring novel and clinically relevant outcomes are incumbent.",

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AB - We have entered an era of exploding interest in therapeutics for sickle cell disease. The expansion in our understanding of sickle cell disease pathophysiology has enhanced the range of potential therapeutic targets. From induction of fetal hemoglobin to antiadhesion molecules, we are potentially on the cusp of making life-altering modifications for individuals with sickle cell disease. This disease population cannot afford to let the current momentum wane. Studies exploring combinations of therapies affecting multiple steps in the pathophysiology and exploring novel and clinically relevant outcomes are incumbent.

KW - Antiadhesion molecules

KW - Induction of fetal hemoglobin

KW - Pathophysiology

KW - Sickle cell disease

KW - Therapeutic targets

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JO - Pediatric Clinics of North America

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ER -

A Scientific Renaissance: Novel Drugs in Sickle Cell Disease

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Keywords

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