Trusted Resources: Education

Scientific literature and patient education texts

A Caregiver’s Guide to Zolgensma

key information

source: Novartis Gene Therapies, Inc

year: 2021



ZOLGENSMA® (onasemnogene abeparvovec-xioi) is a prescription gene therapy used to treat children less than 2 years old with spinal muscular atrophy (SMA). ZOLGENSMA is given as a one-time infusion into a vein. ZOLGENSMA was not evaluated in patients with advanced SMA.

Important Safety Information

ZOLGENSMA can increase liver enzyme levels and cause acute serious liver injury or acute liver failure. Patients will receive an oral corticosteroid before and after infusion with ZOLGENSMA and will undergo regular blood tests to monitor liver function. Contact the patient’s doctor immediately if the patient’s skin and/or whites of the eyes appear yellowish, if the patient misses a dose of corticosteroid or vomits it up, or if the patient experiences a decrease in alertness.


read more