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1 galsulfase治疗IV型粘多糖病BioMarin制药公司宣布,美国FDA已经批准galsulfase(商品名:naglazyme)上市。此药是第一个用于治疗IV型粘多糖病的药物。在美国,naglazyme获准为罕见病治疗药物。IV型粘多糖病(又称Maroteaux-Lamy综合征)是一种恶性的、威胁生命的遗传性疾病,病因是
1 galsulfase for Type IV mucopolysaccharidosis BioMarin Pharmaceuticals announces that the U.S. FDA has approved the listing of galsulfase (naglazyme). This medicine is the first to treat type IV mucopolysaccharidosis. In the United States, naglazyme is approved as a treatment for rare diseases. Type IV mucopolysaccharidosis (also known as Maroteaux-Lamy syndrome) is a malignant, life-threatening genetic disorder that causes