New hope for pompe patients: expanded access to experimental therapy
NCT ID NCT03865836
First seen Sep 30, 2025 · Last updated May 22, 2026 · Updated 31 times
Summary
This program offers early access to an experimental treatment (ATB200/AT2221) for people with Pompe disease who cannot join other clinical trials or choose not to use standard therapy. It aims to provide treatment while collecting safety data. Participants must have a confirmed diagnosis and agree to share their health 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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Study contacts
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Conditions
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