Ultragenyx Pharmaceutical近日公布了其基因疗法UX111的长期随访数据，结果显示该疗法对桑菲利波综合征（MPS IIIA）患儿具有持续性的治疗效果。数据显示，接受UX111治疗的患者脑脊液中硫酸乙酰肝素（CSF-HS）水平出现显著且持久的降低，同时多个发育领域的临床功能均呈现持续有意义的改善。这些长期数据进一步验证了UX111基因疗法在改变疾病进程方面的潜力，为患有这种罕见神经退行性疾病的儿童带来了新的希望。
Ultragenyx Pharmaceutical近日公布了其基因疗法UX111的长期随访数据，结果显示该疗法对桑菲利波综合征（MPS IIIA）患儿具有持续性的治疗效果。数据显示，接受UX111治疗的患者脑脊液中硫酸乙酰肝素（CSF-HS）水平出现显著且持久的降低，同时多个发育领域的临床功能均呈现持续有意义的改善。这些长期数据进一步验证了UX111基因疗法在改变疾病进程方面的潜力，为患有这种罕见神经退行性疾病的儿童带来了新的希望。
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