Late-onset Pompe disease (LOPD) is characterized by a progressive myopathy resulting from a deficiency of acid α-glucosidase enzyme activity. Enzyme replacement therapy has been shown to be effective, but long-term treatment results vary. Avalglucosidase alfa demonstrated non-inferiority to alglucosidase alfa in a phase 3 study, allowing in France compassionate access for advanced LOPD patients unresponsive to alglucosidase alfa.

中文翻译：

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对于因阿糖苷酶 alfa 治疗而病情恶化的迟发性庞贝病患者，改用阿糖苷酶 alfa 一年后的实际效果：一项法国队列研究

迟发性庞贝病 (LOPD) 的特征是由于酸性 α-葡萄糖苷酶活性缺乏而导致的进行性肌病。酶替代疗法已被证明是有效的，但长期治疗结果各不相同。在一项 3 期研究中，Avalglucosidase alfa 表现出不劣于 alglucosidase alfa，这使得在法国能够同情地治疗对 alglucosidase alfa 无反应的晚期 LOPD 患者。