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An Exploratory, Open-Label Study of the Safety and Efficacy of High Dose or High Dosing Frequency Myozyme® (alglucosidase alfa) Treatment in Patients with Pompe Disease Who Do Not Have an Optimal Resp...

Mise à jour : Il y a 4 ans
Référence : EUCTR2015-000582-31

An Exploratory, Open-Label Study of the Safety and Efficacy of High Dose or High Dosing Frequency Myozyme® (alglucosidase alfa) Treatment in Patients with Pompe Disease Who Do Not Have an Optimal Response to the Standard Dose Regimen

Femme et Homme

  • | Pays :
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  • | Organes :
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  • | Spécialités :
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Extrait

1. Evaluate the safety and efficacy of alternate alglucosidase alfa dosing regimens 2. Evaluate differences in efficacy in the 2 dosing arms

Critère d'inclusion

  • Pompe disease (acid alpha-glucosidase deficiency)

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