Zolgensma (onasemnogene abeparvovec-xioi) is a gene replacement therapy indicated for the treatment of spinal muscular atrophy (SMA) in paediatric patients.
1- DESCRIPTION
ZOLGENSMA is a suspension of an adeno-associated viral vector-based gene therapy for
intravenous infusion. It is a recombinant self-complementary AAV9 containing a transgene
encoding the human survival motor neuron (SMN) protein, under the control of a
cytomegalovirus enhancer/chicken-ß-actin hybrid promoter.
