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Strimvelis
autologous CD34+ enriched cell fraction that contains CD34+ cells transduced with retroviral vector that encodes for the human ADA cDNA sequence
Alvorlig kombineret immundefekt
✅ Autoriseret
Avanceret terapi
✅
Ingen aktive alarmer
Denne medicin har ingen igangværende mangel, sikkerhedsbreve eller tilbagekaldelser.
Hvad det bruges til
Strimvelis is indicated for the treatment of patients with severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID), for whom no suitable human leukocyte antigen (HLA)-matched related stem cell donor is available (see section 4.2 and section 4.4).
⚠️ Patient sikkerhed
No
Officiel EMA-side
https://www.ema.europa.eu/en/medicines/human/EPAR/strimvelis