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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
Immunodéficience combinée sévère
✅ Autorisé
Thérapie avancée
✅
Aucune alerte active
Ce médicament ne fait l'objet ni de pénuries en cours, ni de lettres de sécurité, ni de rappels.
À quoi ça sert
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).
⚠️ Sécurité des patients
No
Page officielle de l'EMA
https://www.ema.europa.eu/en/medicines/human/EPAR/strimvelis