EXPANDED ACCESS USE OF MYOZYME (ALGLUCOSDASE ALFA) IN PATIENTS WITH INFANTILE-ON

这个子项目是利用由NIH/NCRR资助的中心拨款提供的资源的许多研究子项目之一。子项目和调查员(PI)可能从另一个NIH来源获得了主要资金，因此可能会出现在其他CRISE条目中。列出的机构是针对中心的，而不一定是针对调查员的机构。本研究旨在评价肌酶作为庞贝病潜在治疗药物的长期安全性和潜在疗效。

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 research study being conducted to evaluate the long term safety and potential effectiveness of Myozyme as a potential therapy for Pompe dise