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1 Study Matches

eLLiPSIS—A Longitudinal, Observational Study to Examine the Measurement Characteristics of the Pompe Disease Symptom Scale and the Pompe Disease Impact Scale

The purpose of this non-interventional study is to measure the performance of the Pompe Disease Symptom Scale (PDSS) and the Pompe Disease Impact Scale (PDIS), which are disease-specific patient reported outcome (PRO) instruments exclusively for Pompe disease.
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18 year(s) or older
This study is NOT accepting healthy volunteers
N/A
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Inclusion Criteria:
Diagnosed with late-onset Pompe disease
Enzyme Replacement Therapy Infusions occur no more frequently than every two weeks
Exclusion Criteria:
Patients who are wheelchair dependent OR unable to ambulate 50 meters (approximately 160 feet) without stopping and without an assistive device
Patients who require invasive-ventilation (non-invasive ventilation is allowed)
Patients currently on investigational drug/clinical trials
Neurology
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Study Locations

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Location
Hershey, PA