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  2. FDA approves cure for sickle cell disease, the first ... - AOL

    www.aol.com/news/fda-approves-cure-sickle-cell...

    The Food and Drug Administration on Friday approved a powerful treatment for sickle cell disease, a devastating illness that affects more than 100,000 Americans, the majority of whom are Black.

  3. US FDA approves two gene therapies for sickle cell disease - AOL

    www.aol.com/news/us-approves-two-gene-therapies...

    (Reuters) -The U.S. Food and Drug Administration (FDA) on Friday approved two gene therapies for sickle cell disease, making one of them the first treatment in the United States based on the Nobel ...

  4. FDA approves two gene therapies for sickle cell, bringing ...

    www.aol.com/fda-expected-approve-first-crispr...

    The US Food and Drug Administration on Friday approved two gene-based treatments for sickle cell disease, including the first therapy that uses the gene-editing technique CRISPR, opening a new era ...

  5. Exagamglogene autotemcel - Wikipedia

    en.wikipedia.org/wiki/Exagamglogene_autotemcel

    The treatment was approved in the United Kingdom for the treatment of sickle cell disease and transfusion-dependent beta thalassemia in November 2023. [10] [11] [12] It was approved in the United States for the treatment of sickle cell disease in December 2023 and for the treatment of transfusion-dependent beta thalassemia in January 2024. [13 ...

  6. Lovotibeglogene autotemcel - Wikipedia

    en.wikipedia.org/wiki/Lovotibeglogene_autotemcel

    Lovotibeglogene autotemcel, sold under the brand name Lyfgenia, is a lentiviral gene therapy used for the treatment of sickle cell disease. [1] [3] [4] [5]The most common side effects include stomatitis (mouth sores of the lips, mouth, and throat), low levels of platelets, white blood cells, and red blood cells, and febrile neutropenia (fever and low white blood cell count), consistent with ...

  7. Voxelotor - Wikipedia

    en.wikipedia.org/wiki/Voxelotor

    In November 2019, voxelotor received accelerated approval in the United States for the treatment of sickle cell disease for those twelve years of age and older. [ 11 ] [ 12 ] The U.S. Food and Drug Administration (FDA) considered it to be a first-in-class medication . [ 13 ]

  8. Two gene therapies for sickle cell disease approved in US - AOL

    www.aol.com/news/two-gene-therapies-sickle-cell...

    The Food and Drug Administration said the one-time treatments can be used for patients 12 and older with severe forms of the disease. ... is the first approved therapy based on CRISPR, the gene ...

  9. Crizanlizumab - Wikipedia

    en.wikipedia.org/wiki/Crizanlizumab

    The FDA granted approval of Adakveo to Novartis. [6] [8] The European Medicines Agency's human medicines committee (CHMP) has recommended the withdrawal of Adakveo (crizanlizumab), a medicine for preventing vaso-occlusive crises in patients with sickle cell disease, due to the lack of sufficient benefits outweighing the risks. [15]

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