[Alglucosidase alpha (Myozyme) for treatment of patients with late-onset Pompe disease]
Comite d'Avaluacio de Medicaments d'Utilitzacio Hospitalaria (CAMUH)
Record ID 32011000221
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' recomendations: 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
URL for published report: http://www.gencat.cat/salut/depsan/units/aatrm/pdf/alglucosidasa_alfa_pompe_aiaqs2011ca.pdf
English language abstract: An English language summary is available
Publication Type: Not Assigned
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
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Copyright: Catalan Agency for Health Information, Assessment and Quality (CAHIAQ)
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