Expanded access for infantile-onset Pompe disease treatment
Part of Genetic & congenital clinical trials.
This program provides access to an investigational treatment (ATB200/AT2221) for children with infantile-onset Pompe disease who are not doing well on standard enzyme replacement therapy. The goal is to see if this new combination can help slow or improve symptoms.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You or your child must be under 18 years old.
- You must have a confirmed diagnosis of Pompe disease, specifically the infantile-onset type.
- You must not be able to join a current commercial drug trial (e.g., due to location).
- You must be currently receiving enzyme replacement therapy (ERT) but still experiencing decline in motor or breathing function.
- If of childbearing age and sexually active, you must agree to use highly effective birth control during treatment and for 90 days after.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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