新的基因疗法显示,在延缓杜尚尼亚肌肉萎缩过程方面很有希望,但也有局限性。 New gene therapy shows promise in slowing Duchenne muscular dystrophy progression but has limitations.

研究人员在杜尚症肌肉萎缩(DMD)基因疗法方面正在取得进展,这是一种导致肌肉虚弱的遗传状况。 Researchers are making progress in gene therapy for Duchenne muscular dystrophy (DMD), a genetic condition causing muscle weakness. 最近第3阶段的试验表明,根据磁共振指数测量结果,脱氧化物(Elevidys)可能减缓疾病发展速度并保持肌肉健康。 A recent phase 3 trial showed that delandistrogene moxeparvovec (Elevidys) may slow disease progression and preserve muscle health, based on MRI measurements. 然而,经林业发展局于2024年批准的这种疗法不能治愈DMD,而且有局限性,包括高昂的费用和潜在的副作用,如肌肉和心脏炎症。 However, the therapy, approved by the FDA in 2024, does not cure DMD and has limitations, including high costs and potential side effects like muscle and heart inflammation. 需要进一步研究以评估其长期效益和风险。 Further studies are needed to assess its long-term benefits and risks.