Desperate pompe patients get early access to experimental drug combo
NCT ID NCT03865836
First seen Jun 26, 2026 · Last updated Jun 26, 2026
Summary
This expanded access program provides ATB200/AT2221 to people with Pompe disease who cannot join ongoing clinical trials or choose not to use standard therapy. The treatment combines an enzyme replacement therapy with a chaperone drug to help break down glycogen buildup. Participants must have a confirmed diagnosis and agree to share their health data.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
ATB200 (enzyme replacement therapy) and AT2221 (chaperone drug)
What this could lead to
If this treatment works, it could offer a new option for managing Pompe disease, potentially improving muscle function and survival.
What could go wrong
This is an early access program, not a formal trial, so effectiveness and safety are still being studied. Benefits are uncertain, and there may be unknown side effects.
Disclaimer
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This is a summary of
the original study
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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
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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