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<p>Until the introduction of gene-based treatment approaches a&#xa0;few years ago, spinal muscular atrophy (SMA) was the most common cause of death due to genetic diseases in infancy. The inclusion of SMA in the neonatal screening fundamentally improved the prognosis but symptomatic patients can still be expected. Close collaboration between neuromuscular centers and practitioners and healthcare structures is essential to ensure standards of care.</p>

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Therapie der spinalen Muskelatrophie

  • 期刊论文

  • 出版模式：

    开放获取

  • 发表日期：

    2026年1月12日

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  • Simone Mahal,
  • Astrid Eisenkölbl,
  • Matthias Baumann

摘要

Until the introduction of gene-based treatment approaches a few years ago, spinal muscular atrophy (SMA) was the most common cause of death due to genetic diseases in infancy. The inclusion of SMA in the neonatal screening fundamentally improved the prognosis but symptomatic patients can still be expected. Close collaboration between neuromuscular centers and practitioners and healthcare structures is essential to ensure standards of care.