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Zolgensma
onasemnogene abeparvovec
Spinal muskelatrofi
✅ Autoriseret
Avanceret terapi
✅
Ingen aktive alarmer
Denne medicin har ingen igangværende mangel, sikkerhedsbreve eller tilbagekaldelser.
Hvad det bruges til
Zolgensma is indicated for the treatment of: patients with 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of SMA Type 1, or patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and up to 3 copies of the SMN2 gene.
⚠️ Patient sikkerhed
No
Officiel EMA-side
https://www.ema.europa.eu/en/medicines/human/EPAR/zolgensma