周五，BioMarin公布了针对晚发型庞贝氏症患者的Pombiliti与Opfolda联合疗法的三期临床试验五年期数据。
On Friday, BioMarin announced five-year data from a Phase 3 trial for the drugs Pombiliti and Opfolda to treat late-onset Pompe disease, or LOPD.
数据显示，接受持续五年治疗的成年患者，其步行距离得以维持，呼吸功能也长期保持相对稳定。研究期间未发现任何新的安全性问题。
The data shows that the combination therapy helped adults with the disease maintain their walking distance over five years of continuous therapy and keep respiratory function relatively stable in the long term. No new safety signals were identified in the data.
去年12月，BioMarin收购了负责推广该疗法的Amicus Therapeutics，此举显著增强了公司在罕见代谢性疾病治疗领域的实力。
In December, BioMarin acquired Amicus Therapeutics, which marketed the combination therapy. The move expanded BioMarin's presence in rare metabolic disease space.
此次分析共纳入82名患者，其中既有初次接受酶替代疗法的患者，也有此前已接受过该治疗的患者。
The analysis followed 82 patients, including both people new to enzyme replacement therapy and those who had been treated with an enzyme replacement therapy prior to joining this study.
庞贝氏症是一种罕见的遗传性溶酶体疾病，病情往往较为严重，会导致全身肌肉逐渐无力且症状不断恶化。许多患者的呼吸肌也会受到影响。
LOPD is a rare, inherited lysosomal disorder that can be severe and involves progressive muscle weakness throughout the body that worsens over time. In many patients, the muscles that control breathing are impacted.
上述研究结果已在日本举行的第31届世界肌肉病学会年会上公布。
The results were presented at the 31st annual Congress of the World Muscle Society in Japan.
CNBC Cures栏目由Alexion, AstraZeneca Rare Disease赞助支持。
CNBC Cures is underwritten by Alexion, AstraZeneca Rare Disease.