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Autologous CD34+ enriched cell fraction th … Autologous CD34+ enriched cell fraction that contains CD34+ cells transduced with retroviral vector that encodes for the human ADA cDNA sequence, sold under the brand name Strimvelis, is a medication used to treat severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID). The most common side effect is pyrexia (fever). ADA-SCID is a rare inherited condition in which there is a change (mutation) in the gene needed to make an enzyme called adenosine deaminase (ADA). As a result, people lack the ADA enzyme. Because ADA is essential for maintaining healthy lymphocytes (white blood cells that fight off infections), the immune system of people with ADA-SCID does not work properly and without effective treatment they rarely survive more than two years. Strimvelis is the first ex vivo autologous gene therapy approved by the European Medicines Agency (EMA).ed by the European Medicines Agency (EMA).
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rdfs:comment |
Autologous CD34+ enriched cell fraction th … Autologous CD34+ enriched cell fraction that contains CD34+ cells transduced with retroviral vector that encodes for the human ADA cDNA sequence, sold under the brand name Strimvelis, is a medication used to treat severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID). The most common side effect is pyrexia (fever). Strimvelis is the first ex vivo autologous gene therapy approved by the European Medicines Agency (EMA).ed by the European Medicines Agency (EMA).
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Strimvelis
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