Gene Therapy in SMA Care

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Gene Therapy in SMA Care

Program Information

Program Information

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Take a look at how early diagnosis and treatment of SMA can optimize outcomes, and learn about gene therapy as a treatment option.

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Important Safety Information, including Boxed Warning.

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  • Overview

    Spinal muscular atrophy (SMA) is a challenging rare disease that requires a diagnosis and access to treatment as early as possible. To help us understand the importance of early treatment, Dr. Sandra Reyna from Novartis Gene Therapies shares key considerations for optimizing outcomes and gives us some insight into available SMA treatment options, focusing on the gene therapy ZOLGENSMA.

     © 2022 Novartis Gene Therapies, Inc.
    US-ZOL-22-0129 08/22

  • Important Safety Information


    ZOLGENSMA (onasemnogene abeparvovec-xioi) suspension for intravenous infusion 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. 

    BOXED WARNING: Acute Serious Liver Injury and Acute Liver Failure

    Acute serious liver injury, acute liver failure, and elevated aminotransferases can occur with ZOLGENSMA. Patients with preexisting 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.


    Transient decreases in platelet counts, some of which met the criteria for thrombocytopenia, were typically observed within the first two weeks after ZOLGENSMA infusion. Monitor platelet counts before ZOLGENSMA infusion and on a regular basis for at least 3 months afterwards. 

    Thrombotic Microangiopathy 
    Cases of thrombotic microangiopathy (TMA) were reported approximately 1 week after ZOLGENSMA infusion. Obtain baseline creatinine and complete blood count before ZOLGENSMA infusion. Following infusion, monitor for thrombocytopenia as well as other signs and symptoms of TMA. Consult a pediatric hematologist and/or pediatric nephrologist immediately to manage if clinically indicated.

    Elevated Troponin-I 
    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. 

    Please see the accompanying Full Prescribing Information also available at

Schedule6 Dec 2022