The one-time-only dose to stop SMA progression

ZOLGENSMA® (onasemnogene abeparvovec-xioi)  is a gene therapy for pediatric patients less than 2 years of age with spinal muscular atrophy (SMA)1

Complete Phase 3 SPR1NT data is available

The SPR1NT clinical trial is complete for presymptomatic patients with 2 and 3 copies of SMN2. View data for patients treated with ZOLGENSMA.

ZOLGENSMA prescription and patient consent forms

Getting started with ZOLGENSMA

Complete the ZOLGENSMA Prescription and Patient Consent Forms for eligible patients to help with insurance and prescription-related processes.


Follow Payne’s ZOLGENSMA journey

Payne’s SMA was first identified through newborn screening. His SMA diagnosis and 4 copies of SMN2 were later confirmed through genetic testing. 


Locate a rep

Enter your ZIP code to locate your nearest Novartis Gene Therapies representative to guide you and your patients throughout the ZOLGENSMA treatment process.

You must enter a valid ZIP code. E.g. 90210


Visit the Resources page to learn more about ZOLGENSMA. Here, you’ll be able to access additional data through scientific reprints, access pre- and posttreatment guides, and important forms.

OneGene Program Logo | ZOLGENSMA® (onasemnogene abeparvovec-xioi)

Patient support

The OneGene Program®, offered by Novartis Gene Therapies, is a comprehensive, individualized support program for families with pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) and their healthcare providers throughout the ZOLGENSMA treatment journey.

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

Families can access the full range of OneGene Program support resources when the ZOLGENSMA Prescription Form and accompanying Patient Consent Form have been provided to the OneGene Program.

For questions call 1-855-441-GENE (4363),
Monday-Friday (8 AM to 8 PM ET)

Discover more about ZOLGENSMA


Explore efficacy, safety, dosing, and how ZOLGENSMA works


Understand SMA

Learn about the signs and symptoms of SMA

Understand SMA

Steps to treatment

What to know before, during, and after infusion day

Steps to treatment


Learn about resources available to your patients


Reference: 1. ZOLGENSMA [prescribing information]. Bannockburn, IL: Novartis Gene Therapies, Inc; 2023.