Onasemnogene abeparvovec

Therapeutic indications

Onasemnogene abeparvovec is indicated for:

Spinal muscular atrophy (SMA) Type 1

Onasemnogene abeparvovec 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.

For this indication, competent medicine agencies globally authorize below treatments (click for details):

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