学习MPSVI CSP 完整的
粘多糖病(MPS) VI临床监测项目(CSP)
指示
粘多糖病VI (MPS VI, Maroteaux-Lamy综合征)
目标
The objectives of this program are: to further characterize the natural progression of MPS VI disease; to generate and disseminate information on the care and management of MPS VI patients to clinical and medical professionals; to provide a resource to physicians and patients by providing information for optimizing patient care based on aggregate data; to characterize the clinical response to long-term galsulfase treatment; to further characterize the long-term safety of galsulfase treatment.
临床实验的治疗
Galsulfase
状态
本研究已经完成
研究类型
第四阶段