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

Mise à jour : Il y a 4 ans
Référence : EUCTR2010-020611-36

Femme et Homme

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Extrait

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.


Critère d'inclusion

  • Pompe disease (acid alpha-glucosidase deficiency)