Expanded Access for ATB200/AT2221 for the Treatment of LOPD
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government.
Know the risks and potential benefits of clinical studies and talk to your health care provider before participating.
Read our disclaimer for details.
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Layout table for eligibility information
Ages Eligible for Study:
1 Year and older (Child, Adult, Older Adult)
Sexes Eligible for Study:
Subject must have a diagnosis of LOPD based on documentation of one of the following:
deficiency of GAA enzyme
Subject must provide signed informed consent prior to any study-related procedures being performed.
Subject is eligible for enrollment in an ongoing clinical trial using ATB200/AT2221.
Subject, if female, is pregnant or breastfeeding at screening.
Subject, whether male or female, is planning to conceive a child during the study.
Glycogen Storage Disease Type II
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Central Nervous System Diseases
Nervous System Diseases