The treatment of lysosomal storage diseases (LSDs) has progressed significantly in recent years. In particular, the development of enzyme replacement therapy (ERT) has provided the first disease-specific treatment in patients with these devastating and life-limiting disorders [Beck 2007]. This therapeutic approach has been shown to improve long-term clinical outcomes in patients with Gaucher disease, Fabry disease and muco-polysaccharidosis (MPS) type II, and plays a key role in the management of patients with MPS I, MPS VI and Pompe disease [Beck 2007, Rohrbach and Clarke 2007].
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机译：近年来，溶酶体贮积病（LSD）的治疗取得了显着进展。尤其是，酶替代疗法（ERT）的发展为患有这些破坏性和生命受限疾病的患者提供了首个针对疾病的治疗方法[Beck 2007]。已证明这种治疗方法可改善高雪氏病，法布里氏病和II型粘多糖贮积病（MPS）患者的长期临床疗效，并且在管理MPS I，MPS VI和庞贝病患者中起关键作用[Beck 2007，Rohrbach and Clarke 2007]。
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