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Clinical Trial Summary

Pompe disease (also known as glycogen storage disease Type II) is a rare autosomal recessive metabolic muscle disease caused by the deficiency of acid α glucosidase (GAA), an enzyme that degrades lysosomal glycogen. As opposed to the exclusively cytoplasmic accumulation of glycogen that occurs in other glycogen storage disorders, Pompe disease is characterized by organelle bound (lysosomal) and extra-lysosomal accumulation of glycogen in many body tissues, ultimately leading to multisystemic pathology. The overall objective of this study was to evaluate the long-term growth and development of participants with infantile-onset Pompe disease with alglucosidase alfa before 1 year of age. Participants were to be followed for a 10-year period.


Clinical Trial Description

n/a


Study Design


Related Conditions & MeSH terms


NCT number NCT00486889
Study type Interventional
Source Sanofi
Contact
Status Completed
Phase Phase 4
Start date August 26, 2008
Completion date November 23, 2021

See also
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