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Emerging therapies in sickle cell disease.

Amina Nardo-Marino1, Valentine Brousse2, David Rees3

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British Journal of Haematology
|March 7, 2020
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Summary

New sickle cell disease (SCD) therapies are emerging, offering hope for improved treatments and potential cures. However, access to these advancements for the majority of patients worldwide remains a significant challenge.

Keywords:
clinical trialsgene editinghydroxyureainflammationsickle cell disease

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Area of Science:

  • Hematology
  • Pharmacology
  • Genetics

Background:

  • Sickle cell disease (SCD) is a prevalent and severe inherited blood disorder globally.
  • Current therapeutic options for SCD are limited, with hydroxyurea being the primary treatment.
  • Despite challenges, research into novel SCD therapies has intensified.

Purpose of the Study:

  • To review the current landscape of therapeutic options for sickle cell disease.
  • To highlight emerging treatments and their clinical trial outcomes.
  • To discuss the future perspectives of curative therapies for SCD.

Main Methods:

  • Literature review of recent clinical trials for sickle cell disease therapies.
  • Analysis of pharmacological targets and drug development in SCD.
  • Evaluation of the accessibility of novel treatments.

Main Results:

  • Numerous new drugs targeting various pathways have been investigated.
  • While many trials failed, some novel agents show promising results.
  • Recent trials suggest potential for curative therapies in SCD.

Conclusions:

  • The pipeline for sickle cell disease treatments is expanding with promising agents.
  • Novel therapies and potential cures are likely to become available soon.
  • Equitable access to advanced SCD treatments remains a critical global issue, likely limited to high-income nations initially.