Gene Therapy in SMA Care

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Gene Therapy in SMA Care



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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.


     © 2023 Novartis Gene Therapies, Inc.
    US-ZOL-22-0129 03/23

  • Important Safety Information

    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. 

    BOXED WARNING: Serious Liver Injury and Acute Liver Failure

    Cases of acute liver failure with fatal outcomes have been reported. Acute serious liver injury, acute liver failure, and elevated aminotransferases can also 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. Administer systemic corticosteroid to all patients before and after ZOLGENSMA infusion. Continue to monitor liver function for at least 3 months after infusion, and at other times as clinically indicated. If acute serious liver injury or acute liver failure is suspected, promptly consult a pediatric gastroenterologist or hepatologist.


    Systemic Immune Response
    Patients with underlying active infection, either acute or chronic uncontrolled, could be at an increased risk of serious systemic immune response.  Administer ZOLGENSMA to patients who are clinically stable in their overall health status (e.g., hydration and nutritional status, absence of infection). Postpone ZOLGENSMA in patients with infections until the infection has resolved and the patient is clinically stable.

    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 to occur generally within the first two weeks after ZOLGENSMA infusion. TMA can result in life-threatening or fatal outcomes. Obtain baseline creatinine and complete blood count before ZOLGENSMA infusion. Following infusion, monitor platelet counts closely as well as other signs and symptoms of TMA. Consult a pediatric hematologist and/or pediatric nephrologist immediately to manage as 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. Consider consultation with a cardiologist if troponin elevations are accompanied by clinical signs or symptoms. 


    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

Schedule28 Sep 2023