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  • ZOLGENSMA (onasemnogene abeparvovec-xioi)

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    Description

    How It Works

    Zolgensma works by delivering a functional copy of the SMN gene into the patient’s cells using a viral vector called adeno-associated virus 9 (AAV9). This vector targets motor neuron cells and introduces the healthy SMN gene, which then produces the survival motor neuron (SMN) protein. This protein is essential for the maintenance and function of motor neurons, and its presence helps to improve muscle function and slow the progression of SMA.

    Side Effects

    • Elevated liver enzymes
    • Nausea and vomiting
    • Decreased platelet count (thrombocytopenia)
    • Fever
    • Decreased appetite
    • Elevated cardiac troponin-I

    Indications

    • Treatment of spinal muscular atrophy (SMA) in pediatric patients under the age of 2 with bi-allelic mutations in the SMN1 gene

    Contraindications

    • Hypersensitivity to any of the components of Zolgensma
    • Patients with advanced liver disease
    • Severe pre-existing thrombocytopenia

    Price Comparison in Different Countries

    Country Price Reference
    Pakistan Approximately PKR 250,000,000 Dawaai Pakistan
    United States USD 2,125,000 GoodRx
    United Kingdom GBP 1,795,000 NHS UK
    Canada CAD 2,850,000 Health Canada
    Australia AUD 2,900,000 PBS Australia

    Top 5 Global Brands

    1. Novartis
    2. Pfizer
    3. Roche
    4. Sanofi
    5. GlaxoSmithKline (GSK)

    Conclusion

    Zolgensma (Onasemnogene Abeparvovec-xioi) represents a significant advancement in the treatment of spinal muscular atrophy (SMA). By providing a functional copy of the SMN gene, Zolgensma helps improve motor neuron function and slow the progression of SMA in young children. Despite its high cost, Zolgensma offers hope for better quality of life for patients with SMA. The cost of Zolgensma varies widely across different countries, reflecting disparities in healthcare pricing and accessibility. Patients and families should consult with their healthcare providers and explore financial assistance programs to access this life-changing treatment.  
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    ZOLGENSMA (onasemnogene abeparvovec-xioi)
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