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This is an expanded access program (EAP) for eligible participants designed to provide access to ATB200/AT2221.
Full description
This program is being offered on a patient by patient basis and will require company, IRB/IEC and single patient IND approval.
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Inclusion criteria
Subject must have a diagnosis of Pompe disease based on documentation of one of the following:
Patient does not currently qualify for an Amicus sponsored on-going clinical trial or is declining on currently approved ERT (e.g. Myozyme)
The patient must be willing to receive treatment with ATB200/AT2221 via this program, which includes signing an authorization form for sharing clinical data with Amicus Therapeutics, and its agent Early Access Care LLC.
Exclusion criteria
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For Site; For Patient
Data sourced from clinicaltrials.gov
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