ZOLGENSMA® (onasemnogene abeparvovec-xioi)
Indications and Usage:
ZOLGENSMA is an adeno-associated virus vector-based gene therapy indicated for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene.
Limitations of Use
- The safety and effectiveness of repeat administration of ZOLGENSMA have not been evaluated.
- The use of ZOLGENSMA in patients with advanced SMA (e.g., complete paralysis of limbs, permanent ventilator-dependence) has not been evaluated.
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Prescribing Information |