Waskyra
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Active Ingredient: genetically modified autologous CD34+ cell enriched population that contains haematopoietic stem and progenitor cells (HSPC) transduced ex vivo using a lentiviral vector encoding the human Wiskott-Aldrich Syndrome (WAS) gene
Product Overview
About Waskyra
Waskyra is a medicine that is made individually for each patient.
How it is used
Waskyra can only be obtained with a prescription and must be given in a qualified treatment centre by a doctor trained in giving this medicine and with experience in stem cell transplantation. Waskyra is made individually for each patient out of stem cells collected from their blood and must only be given to the patient for whom it is made. It is given once by infusion (drip) into a vein. Before Waskyra is given, the patient will receive conditioning (preparatory) treatment to clear their bone marrow of cells.
How it works
Waskyra is made from stem cells (called CD34+ cells) that are collected from the patient’s blood. The cells are genetically modified in the laboratory so that they can produce a functional WAS protein. When transplanted back into the patient, the modified cells migrate to the bone marrow where they start making healthy blood and immune cells that produce a functional WAS protein, thereby helping to relieve the symptoms of the disease.
Active substance: etuvetidigene autotemcel
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