Start treating your patients with ZOLGENSMA:


ZOLGENSMA is approved for pediatric patients with SMA less than 2 years of age, who are genetically confirmed for bi-allelic mutations in the survival motor neuron 1 (SMN1) gene.

Once you and your patient’s caregiver have decided on ZOLGENSMA, the following tests need to be performed prior to infusion. These tests can be ordered by the referring physician or a physician at a ZOLGENSMA-ready institution.

Genetic Confirmation1

  • Confirmation of the bi-allelic mutation in SMN1 gene
  • SMN2 copy number analysis2

AAV9 Antibody Test1

  • Testing for the presence of anti-AAV9 antibodies
  • The safety and efficacy of ZOLGENSMA in patients with anti-AAV9 antibody titers above 1:50 have not been evaluated
  • Retesting may be performed if anti-AAV9 antibody titers are reported as positive or elevated1

Baseline Tests1

  • Baseline evaluations of liver function (clinical exam, AST, ALT, total bilirubin, albumin, prothrombin time, PTT, and INR), creatinine, complete blood count (including hemoglobin and platelet count), and troponin-I
  • Liver function, platelet count, and troponin-I will need to be monitored following infusion
  • Due to the increased risk of serious systemic immune response, administer ZOLGENSMA to patients who are clinically stable in their overall baseline health status (e.g., hydration and nutritional status, absence of infection) prior to infusion. Postpone ZOLGENSMA in patients with infections until the infection has resolved and the patient is clinically stable. Clinical signs or symptoms of infection should not be evident at the time of ZOLGENSMA infusion

Use of ZOLGENSMA in premature neonates before reaching full-term gestational age is not recommended because concomitant treatment with corticosteroids may adversely affect neurological development. Delay ZOLGENSMA infusion until full-term gestational age is reached.1

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.1

Novartis Gene Therapies is offering the Novartis Gene Therapies Laboratory Program to assist with genetic and anti-AAV9 antibody testing. For more information, please contact your Novartis Gene Therapies Account Manager or call the OneGene Program® at
1-855-441-GENE (4363)

Referring patients for treatment

Early treatment is essential for infants with SMA.3 If your institution is not ready to infuse ZOLGENSMA, refer your eligible patients for treatment. Work with your patients’ families to help identify the ZOLGENSMA-ready institution that is right for them.

To locate a treatment center that can administer ZOLGENSMA: email or contact the MDA Resource Center
at 1-833-ASK-MDA1 or

The ZOLGENSMA Prescription and Patient Consent Forms

To prescribe ZOLGENSMA to a patient, complete a ZOLGENSMA Prescription Form, and have the parent or legal guardian complete the accompanying Patient Consent Form. A signed Patient Consent Form and completed Prescription Form are needed for patients and families to receive support through the OneGene Program®.

Please fax the signed ZOLGENSMA Prescription Form and Patient Consent Form as soon as they have been completed to 1-855-951-GENE (4363).

ZOLGENSMA Prescription and Patient Consent Forms

Preparing for Infusion

Important information to know before infusion

Preparing for Infusion

Patient Support

Answering questions with the OneGene Program®

Patient Support

References: 1. ZOLGENSMA [prescribing information]. Bannockburn, IL: Novartis Gene Therapies, Inc; 2023. 2. Mercuri E, Finkel RS, Muntoni F, et al. Diagnosis and management of spinal muscular atrophy: part 1: recommendations for diagnosis, rehabilitation, orthopedic and nutritional care. Neuromuscul Disord. 2018;28(2):103-115. 3. Lin CW, Kalb SJ, Yeh WS. Delay in diagnosis of spinal muscular atrophy: a systematic literature review. Pediatr Neurol. 2015;53(4):293-300.