Case Report: The effect of early initiation of eteplirsen treatment on the cardiac and motor disease course in an individual with Duchenne muscular dystrophy - Takeaways - MDSpire
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Case Study: Impact of Early Eteplirsen Treatment on Cardiac and Motor Progression in a Patient with Duchenne Muscular Dystrophy

  • By

  • Yamen Masalha

  • Yoav Zehavi

  • Banan Musallam

  • Majd Khamaysi

  • Maali Abu Omer

  • Daniel L. Fink

  • Ronen Spiegel

  • July 9, 2026

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  • 1

    A 7-year-old boy diagnosed with Duchenne muscular dystrophy (DMD) exhibited elevated transaminases and significant cardiac dysfunction at 17 months.

  • 2

    Eteplirsen treatment was initiated at 24 months, targeting a deletion in the DMD gene, and was well tolerated over 5 years.

  • 3

    The patient demonstrated stable cardiac function and gradual motor improvement, despite persistent hyperlordosis and waddling gait.

  • 4

    Enalapril was used to manage cardiac symptoms, with adjustments made based on the patient's evolving cardiac function.

  • 5

    This case is the first to document cardiac outcomes after eteplirsen treatment in a patient with DMD as young as 24 months.

Original Source(s)

Case Report: The effect of early initiation of eteplirsen treatment on the cardiac and motor disease course in an individual with Duchenne muscular dystrophy

  • by Yamen Masalha, Yoav Zehavi, Banan Musallam, Majd Khamaysi, Maali Abu Omer, Daniel L. Fink, Ronen Spiegel

  • July 9, 2026

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