A Scientific Renaissance: Novel Drugs in Sickle Cell Disease

Ahmar U. Zaidi, Matthew M. Heeney

Research output: Contribution to journalReview articlepeer-review


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 languageEnglish
Pages (from-to)445-464
Number of pages20
JournalPediatric Clinics of North America
Issue number3
StatePublished - Jun 2018
Externally publishedYes


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


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