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Keep this study
Lead
Amicus Therapeutics
The study is designed to evaluate the response of cell lines derived from blood and skin tissue from Pompe patients to pharmacological chaperones. Samples of blood and skin tissue will be obtained to make cell lines that will be used to test pharmacological chaperones. The study will include patients with early and late-onset Pompe disease.
Age
Any age
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
Male or female of any age
Confirmed diagnosis of Pompe disease (early or late-onset)
Clinically stable
Written informed consent by subject or legal representative
You may not be if
Other significant disease or otherwise unsuitable for the study, as determined by the investigator
Clareo Health | A Study to Evaluate the Effects of Pharmacological Chaperones in Cells From Patients With Pompe Disease
