Description and Brand Names

US Brand Name

  1. Zolgensma

Descriptions


Onasemnogene abeparvovec-xioi injection is used to treat spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene. Spinal muscular atrophy is a rare neuromuscular disorder that is characterized by loss of lower motor neurons and progressive muscle wasting, often leading to early death. Onasemnogene abeparvovec-xioi is an adeno-associated virus vector-based gene treatment that works by copying the gene needed for the human SMN protein.

This medicine is to be given only by or under the direct supervision of your doctor.

This product is available in the following dosage forms:

  • Suspension