Experimental pompe disease treatment offered to patients out of options
NCT ID NCT03865836
Summary
This program provides access to an experimental treatment called ATB200/AT2221 for people with Pompe disease who can't join regular clinical trials or whose current enzyme replacement therapy isn't working well enough. It's offered on a case-by-case basis to eligible patients who meet specific medical criteria. The treatment aims to better control this rare genetic disorder that affects muscle strength and breathing.
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Study contacts
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Conditions
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