This program is being offered on a patient by patient basis and will require company, IRB/IEC and single patient IND approval.
1. Subject must have a diagnosis of LOPD based on documentation of one of the following: 1. deficiency of GAA enzyme 2. GAA genotyping
2. Subject must provide signed informed consent prior to any study-related procedures being performed.
1. Subject is eligible for enrollment in an ongoing clinical trial using ATB200/AT2221.
2. Subject, if female, is pregnant or breastfeeding at screening.
3. Subject, whether male or female, is planning to conceive a child during the study.
Expanded Access program information for companies listed in the Expanded Access Company Directory is pulled daily from ClinicalTrials.gov, a resource provided by the National Institutes of Health in cooperation with the U.S. Food and Drug Administration. Click here for more information about ClinicalTrials.gov.