Clinical Trial Update: A Study to Evaluate the Effects of Pharmacological Chaperones in Cells From Patients With Pompe Disease

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Summary

A completed study evaluated the effects of pharmacological chaperones on cells derived from patients with Pompe disease.

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Diseases / indications

• Glycogen Storage Disease Type Ii
• Pompe Disease

Snippet

Sponsor: Amicus Therapeutics | Conditions: Pompe Disease, Glycogen Storage Disease Type II | Status: COMPLETED