New hope for pompe patients: expanded access to experimental therapy
NCT ID NCT03865836
First seen Sep 30, 2025 · Last updated May 29, 2026 · Updated 32 times
Summary
This program offers eligible Pompe disease patients access to the experimental drug combination ATB200/AT2221 when they cannot join other clinical trials or choose not to use standard enzyme replacement therapy. Participants must have a confirmed Pompe diagnosis and agree to share their medical data. The goal is to provide treatment under compassionate use while gathering safety information.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
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