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BSH 2021 | Novel drugs for sickle cell

David Rees, MA, MBBS, FRCP, FRCPath, FRCPCH, King’s College Hospital, London, UK, describes new treatment options potentially available for patients with sickle cell disease (SCD). L-glutamine, crizanlizumab, and voxelotor are drugs that are licensed in the US, but not in the UK. L-glutamine has shown to have limited efficacy, whereas crizanlizumab has shown to effectively reduce episodes of pain. Voxelotor, which binds to the α-globin chain to inhibit sickling, has debated clinical benefits and limited evidence of improving quality of life. Prof. Rees also briefly mentions gene therapies, which he predicts will be more widespread in the near future. This interview took place at the 2021 British Society for Haematology Annual Scientific Meeting.

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