autologous cd34+ cell enriched cellular fraction containing genetically modified cd34+ cells with a retroviral vector containing the cdna sequence encoding human adenosine deaminase (ada)
ItalyDrugs containing this active substance
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Strimvelis is a gene therapy used in children to treat a serious condition called ADA-SCID (adenosine deaminase deficiency severe combined immunodeficiency), which prevents the immune system from functioning correctly.
Form: solution for infusion
Active substance Dosage autologous cd34+ cell enriched cellular fraction containing genetically modified cd34+ cells with a retroviral vector containing the cdna sequence encoding human adenosine deaminase (ada) - ATC: L03AX
Manufacturer: FONDAZIONE TELETHON ETS