A study to evaluate muscle tissue, blood, urine and imaging assessments in patients with Late-Onset Pompe Disease treated with alglucosidase alfa

Update Il y a 4 ans
Reference: EUCTR2010-020611-36

Woman and Man

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Extract

The primary objective of this study is to evaluate glycogen clearance in muscle tissue samples collected pre and post alglucosidase alfa treatment in patients with late-onset Pompe disease.


Inclusion criteria

  • Pompe disease (acid alpha-glucosidase deficiency)