IDURONATE-2-SULFATASE REPLACEMENT THERAPY IN MPS II

这个子项目是许多研究子项目中利用 资源由NIH/NCRR资助的中心拨款提供。子项目和 调查员(PI)可能从NIH的另一个来源获得了主要资金， 并因此可以在其他清晰的条目中表示。列出的机构是 该中心不一定是调查人员的机构。 这项研究考察了长期使用酶替代疗法(当缺失时替代导致疾病的酶)治疗MPSII的安全性。如果被证明是安全的，这种药物有可能极大地改善所有受粘多糖病II型(MPS II)影响的个人的健康和生活质量，这种疾病目前还没有治愈方法。他的研究设计允许成功完成I/II期试验的患者继续接受酶替代疗法的治疗，并允许在先前试验中接受安慰剂的患者交叉接受酶替代疗法。

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. This study examines the safety of long-term use of enzyme replacement therapy (replacing the enzyme that, when missing, causes the disease) for MPSII. If proven safe, this drug has the possibility of greatly improving the health and quality of life of all individuals affected with Mucopolysaccharidosis type II (MPS II), a disease for which there is currently no cure. He study design allows patients who successfully completed the phase I/II trial to continue to receive treatment with enzyme replacement therapy, and for patients who had received placebo in the previous trial to cross-over and receive enzyme replacement therapy.