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Updated: Jun 25, 2025

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预防性双重治疗婴儿发病脊柱肌肉缩的风险儿童

Susan E Matesanz1, Karlla W Brigatti2, Millie Young2

  • 1Division of Neurology, Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, USA.

Annals of clinical and translational neurology
|May 31, 2024
PubMed
概括

脊椎肌肉缩1型 (SMA1) 的基因疗法表明,双重疗法耐受性良好,并提供了轻微的益处. 然而,它并没有防止风险婴儿的广泛肌肉退化.

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