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

本子项目是利用由NIH/NCRR资助的中心赠款提供的资源的众多研究子项目之一。子项目和研究者（PI）可能已经从另一个NIH来源获得了主要资金，因此可以在其他CRISP条目中表示。列出的机构是中心的，不一定是研究者的机构。本研究旨在评估Myozyme作为庞贝病潜在治疗药物的长期安全性和潜在有效性

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