Posted by Michael Wonder on 10 Mar 2021
TGA approves Zolgensma
10 March 2021 - Onasemnogene abeparvovec is a new gene therapy for children with spinal muscular atrophy.
Zolgensma is indicated for the treatment of paediatric patients less than 9 months of age with symptomatic or pre-symptomatic spinal muscular atrophy with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene and 1 to 3 copies of the SMN2 gene.
