AN EXPLORATORY, OPEN-LABEL STUDY OF THE SAFETY AND

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。 这项研究的总体目标是评估替代剂量的安全性和有效性。 Pompe病患者的Myozyme方案尚未显示出最佳方案 对标准剂量方案的反应，每隔一周20毫克/公斤(Qow)，在最低剂量后 在入学前立即接受6个月的常规治疗。符合条件的患者将是 按1：1随机分为“频繁给药组”(每周20毫克/公斤)或“高剂量组” 肌酶治疗“剂量组”(40 mg/kg，隔周一次)。

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. The overall objective of this study is to evaluate the safety and efficacy of alternative dosing regimens of Myozyme in patients with Pompe disease who have not demonstrated an optimal response to the standard dosing regimen of 20 mg/kg every other week (qow) after a minimum of 6 months of regular treatment immediately prior to study entry. Eligible patients will be randomized on a 1:1 basis to a "frequent dosing group" (20 mg/kg every week [qw]) or a "high dose group" (40 mg/kg every other week [qow]) of Myozyme treatment.