[Alglucosidase alpha (Myozyme) for treatment of patients with late-onset Pompe disease]

Comite d'Avaluacio de Medicaments d'Utilitzacio Hospitalaria (CAMUH)
Record ID 32011000221
Authors' objectives:

To analyse the available scientific evidence for efficacy, safety and efficiency of alglucosidase alpha compared with any other treatment, including placebo, in patients with late-onset Pompe disease.

Authors' recommendations: Alglucosidase alpha is generally well tolerated and improves walking distance and stabilises pulmonary function in patients with late-onset Pompe disease after 18 months of treatment. The effect of alpha glucosidase antibodies on the drug pharmacodynamics has not been established.
Project Status: Completed
Year Published: 2011
English language abstract: An English language summary is available
Publication Type: Not Assigned
Country: Spain
MeSH Terms
  • alpha-Glucosidases
  • Spain
Organisation Name: Agencia de Qualitat i Avaluacio Sanitries de Catalunya
Contact Address: Antoni Parada, CAHTA, Roc Boronat, 81-95 (2nd floor), 08005 Barcelona, Spain, Tel. +34 935 513 928, Fax: +34 935 517 510
Contact Name: direccio@aatrm.catsalut.net / aparada@aatrm.catsalut.net
Contact Email: direccio@aatrm.catsalut.net / aparada@aatrm.catsalut.net
Copyright: Catalan Agency for Health Information, Assessment and Quality (CAHIAQ)
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