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<p>Duchenne Muscular Dystrophy is a rare, X-linked neuromuscular disorder that leads to progressive muscle degeneration, loss of ambulation, and premature mortality due to respiratory and cardiac failure. Historically, Duchennke Muscular Dystrophy has been managed through supportive and symptomatic treatments, with limited options for disease modification. However, advancements in gene therapy have introduced promising interventions aimed at addressing the underlying dystrophin deficiency. Delandistrogene moxeparvovec (Elevidys) received accelerated approval from the U.S. Food and Drug Administration in June 2023 for ambulatory children aged 4–5 years with a confirmed diagnosis of Duchenne Muscular Dystrophy. This approval represented an advancement, offering a disease-modifying therapy at an early stage when muscle function remains relatively preserved. The Food and Drug Administration expanded its approval in June 2024 to include both ambulatory and non-ambulatory children aged 4 years and older. This study provides a retrospective real-world analysis of eight Duchenne Muscular Dystrophy patients who received Elevidys gene therapy at our center in Qatar. Recognizing the complexities involved in treating older Duchenne Muscular Dystrophy patients, a standardized protocol for pre- and post-infusion care was implemented. Our findings highlight the positive clinical outcomes of gene therapy for Duchenne Muscular Dystrophy patients in Qatar.</p>

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Real-world experience with gene therapy in Duchenne muscular dystrophy center readiness and patients safety: report from Qatar

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2025年11月27日

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  • Mahmoud Fawzi Osman,
  • Khalid Ibrahim,
  • Claire Gleeson,
  • Haytham Ibrahim,
  • Ikram Ul Haque,
  • Noora Alhamad,
  • Tawfeg Ben-Omran

摘要

Duchenne Muscular Dystrophy is a rare, X-linked neuromuscular disorder that leads to progressive muscle degeneration, loss of ambulation, and premature mortality due to respiratory and cardiac failure. Historically, Duchennke Muscular Dystrophy has been managed through supportive and symptomatic treatments, with limited options for disease modification. However, advancements in gene therapy have introduced promising interventions aimed at addressing the underlying dystrophin deficiency. Delandistrogene moxeparvovec (Elevidys) received accelerated approval from the U.S. Food and Drug Administration in June 2023 for ambulatory children aged 4–5 years with a confirmed diagnosis of Duchenne Muscular Dystrophy. This approval represented an advancement, offering a disease-modifying therapy at an early stage when muscle function remains relatively preserved. The Food and Drug Administration expanded its approval in June 2024 to include both ambulatory and non-ambulatory children aged 4 years and older. This study provides a retrospective real-world analysis of eight Duchenne Muscular Dystrophy patients who received Elevidys gene therapy at our center in Qatar. Recognizing the complexities involved in treating older Duchenne Muscular Dystrophy patients, a standardized protocol for pre- and post-infusion care was implemented. Our findings highlight the positive clinical outcomes of gene therapy for Duchenne Muscular Dystrophy patients in Qatar.