Experimental pompe disease treatment offered to patients outside clinical trials
NCT ID NCT03865836
Summary
This program provides access to an experimental combination treatment (ATB200/AT2221) for people with Pompe disease who cannot participate in regular clinical trials or whose current enzyme replacement therapy is no longer working well. The treatment aims to better manage the disease by improving enzyme function. Patients receive the treatment on a case-by-case basis after approval from the company and ethics committees.
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Genom att skicka in godkänner du våra Användarvillkor
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Conditions
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