ZOLGENSMA is the first one-time-only gene therapy for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA)
Request a representative:
Call 1-855-441-GENE (4363) Monday-Friday (8 AM to 8 PM ET)
Request a representative:
Call 1-855-441-GENE (4363)
Monday-Friday (8 AM to 8 PM ET)
Steps to Treatment
There are 5 essential steps to a one-time-only ZOLGENSMA infusion in addition to submitting the ZOLGENSMA Prescription and Patient Consent Forms:
- Confirm diagnosis and run laboratory tests
- Store and handle ZOLGENSMA properly
- Premedicate and plan for infusion day
- Prepare and infuse ZOLGENSMA
- Monitor and postmedicate after infusion
Take an in-depth look at the steps to treatment with ZOLGENSMA
The OneGene Program™, offered by AveXis, is a comprehensive, individualized support program for families with pediatric patients less than 2 years of age with SMA and their healthcare providers.
OneGene Program support includes:
- Answers to questions about ZOLGENSMA
- A dedicated, personalized support team focused on the needs of each family
- Verification of insurance benefits
- Coordination of financial assistance programs for eligible patients
If you have any questions about patient support, call the OneGene Program at
Monday–Friday (8 AM to 8 PM ET)
Send completed ZOLGENSMA Prescription and Patient Consent Forms to the OneGene Program via fax at 1-855-951-GENE (4363).
Register below to receive more information and the latest ZOLGENSMA news.
In the News
The US Food and Drug Administration approved ZOLGENSMA for the treatment of pediatric patients less than 2 years of age with SMA.
Important Safety Information
BOXED WARNING: Acute Serious Liver Injury
Acute serious liver injury and elevated aminotransferases can occur with ZOLGENSMA. Patients with pre-existing liver impairment may be at higher risk. Prior to infusion, assess liver function of all patients by clinical examination and laboratory testing (e.g., hepatic aminotransferases [aspartate aminotransferase (AST) and alanine aminotransferase (ALT)], total bilirubin, and prothrombin time). Administer a systemic corticosteroid to all patients before and after ZOLGENSMA infusion. Continue to monitor liver function for at least 3 months after infusion.
WARNINGS AND PRECAUTIONS
Transient decreases in platelet counts, some of which met the criteria for thrombocytopenia, were observed at different time points after ZOLGENSMA infusion. Monitor platelet counts before ZOLGENSMA infusion and on a regular basis for at least 3 months afterwards.
Transient increases in cardiac troponin-I levels were observed following ZOLGENSMA infusion. Monitor troponin-I before ZOLGENSMA infusion and on a regular basis for at least 3 months afterwards.
The most commonly observed adverse reactions (incidence ≥5%) in clinical studies were elevated aminotransferases and vomiting.
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 or the use in patients with advanced SMA (e.g., complete paralysis of limbs, permanent ventilator dependence) has not been evaluated with ZOLGENSMA.
Please see accompanying Full Prescribing Information.