Myozyme Temporary Access Program

ID Number 08-0453

Principal Investigator(s)
Mark A Sivak

Department(s) or Division(s)


The purpose of this study is to provide adult patients with Pompe disease in the United States (US) access to investigational Aglucosidase Alfa for a limited time until it is approved for commercial use by the Food and Drug Administration (FDA) and sufficient supply is available.

Contact Information
Joan Bratton
(212) 659-5643

Recruiting Patients: Yes