New Gene Therapies for Sickle Cell Disease Approved by FDA for U.S Patients

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Gene therapies for sickle cell disease approved in U.S.

  • Regulators have approved two new gene therapies for sickle cell disease, offering hope for a cure for this painful blood disorder.
  • The treatments are the first of their kind and can be used for patients 12 and older with severe forms of the disease.
  • Sickle cell disease is most common among Black people and affects an estimated 100,000 people in the United States.
  • The approved therapies offer a one-time treatment option for patients, providing a potential alternative to current treatments like medications and blood transfusions.
  • Gene therapies for sickle cell disease approved in U.S.

    The Food and Drug Administration has approved two new gene therapies for sickle cell disease, offering hope for a cure for this painful blood disorder. The one-time treatments can be used for patients 12 and older with severe forms of the disease, providing a potential alternative to current treatments like medications and blood transfusions.

    Significant Themes

    The first approved therapy is based on CRISPR, the gene editing tool, while the other works differently. Both treatments aim to address the debilitating and life-threatening nature of sickle cell disease, particularly for individuals whose lives have been severely disrupted by the condition.

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