Il trattamento si basa sulla terapia enzimatica sostitutiva (ERT) finalizzata a rimpiazzare l’enzima mancante.
The treatment is an enzyme replacement therapy (ERT) called alglucosidase alfa (Myozyme), manufactured by Genzyme, a Sanofi company. Myozyme is indicated for long-term enzyme replacement therapy (ERT) in patients with a confirmed diagnosis of Pompe disease (acid alpha glucosidase deficiency). Myozyme is indicated in adults and paediatric patients of all ages.
For more information about Myozyme, please visit the EMA website to view the current SmPC [dated:18/12/2013] European Medicines Agency or contact your local Genzyme representative.
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A program that tracks information about patients’ symptoms and experiences in order to improve understanding of Pompe disease.