ZOLGENSMA 2 x 10 000 000 000 000 du vecteur/mL, solution pour perfusion

( ARTG ) ZOLGENSMA (onasemnogene abeparvovec) is indicated for the treatment of paediatric patients with 5q spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene with 3 or fewer copies of the SMN2 gene. (Refer to Dosage and Method of Administration, section 4.2, Special Populations, paediatric patients and section 5.1 Clinical Trials).