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Voxelotor: A Ray of Hope for Sickle Disease.

ABSTRACT: Sickle cell disease is one challenging blood disorder, affecting around 100,000 people in the United States alone. None of the currently approved drugs can modify the underlying pathology of the disease. Voxelotor, first of its kind, is an orally administered drug that can alter the underlying disease pathology (by increasing the affinity between Hb and oxygen) and inhibit sickling of red blood cells. Several clinical trials and case series have documented the benefits and safety of voxelotor therapy in sickle cell disease. Currently, the US FDA has approved the drug for treatment of sickle cell disease and also granted the status of orphan drug.

SUBMITTER: AlDallal SM

PROVIDER: S-EPMC7100618 | biostudies-literature | 2020 Feb

REPOSITORIES: biostudies-literature

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Voxelotor: A Ray of Hope for Sickle Disease.

AlDallal Salma M SM

Cureus 20200226 2

Sickle cell disease is one challenging blood disorder, affecting around 100,000 people in the United States alone. None of the currently approved drugs can modify the underlying pathology of the disease. Voxelotor, first of its kind, is an orally administered drug that can alter the underlying disease pathology (by increasing the affinity between Hb and oxygen) and inhibit sickling of red blood cells. Several clinical trials and case series have documented the benefits and safety of voxelotor th ...[more]

PMID: 32257653

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