Adeno-Associated Virus: Side Effects, Uses & Monitoring

Facts extracted from current FDA product labeling for Adeno-Associated Virus. Educational only — not medical advice.

Labeled uses

ZOLGENSMA is an adeno-associated virus (AAV) 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 [see Adverse Reactions (6.2 )] . The use of ZOLGENSMA in patients with advanced SMA (e.g., complete paralysis of limbs, permanent ventilator-dependence) has not been evaluated [see Clinical Studies (14 )] .

Source: FDA labeling via NIH DailyMed (set 68cd4f06…). Not affiliated with FDA or NIH.

Medical disclaimer: Always consult a qualified healthcare professional about any medication.