The Role of Pediatricians in Diagnosing & Treating Spinal Muscular Atrophy

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The Role of Pediatricians in Diagnosing and Treating Spinal Muscular Atrophy

Program Information

Program Information

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Why is it important to diagnose and treat spinal muscular atrophy early, and what role do pediatricians play in meeting those priorities?

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  • Overview

    Affecting one in every 10,000 births worldwide, spinal muscular atrophy, also known as SMA, can result in the progressive loss of basic muscle function and in its most severe forms, can lead to death or the need for permanent ventilation in 90% of cases by age two. How can we help ensure that patients with this debilitating disease get diagnosed early, and what role does gene therapy play in its treatment? Tune in as Dr. Chamindra Konersman from Rady Children’s Hospital in San Diego discusses that and more with Dr. Kevin Strauss from the Clinic for Special Children in Pennsylvania.

                      © 2021 Novartis Gene Therapies, Inc. US-ZOL-20-0544 1/2021

  • 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: 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.


    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.

    Elevated Troponin-I
    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.

    Please see the accompanying Full Prescribing Information also available at

Schedule26 Oct 2021
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