Following a directed appraisal
AWMSG advice |
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| Status: Recommended | ||
Alglucosidase alfa (Myozyme®) is recommended as an option for use within NHS Wales for long-term enzyme replacement therapy in patients with a confirmed diagnosis of Pompe disease (acid α-glucosidase deficiency) . Myozyme® is indicated in adults and paediatric patients of all ages. |
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Medicine details |
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| Medicine name | alglucosidase alfa (Myozyme™) | |
| Formulation | 50 mg powder for concentrate for solution for infusion | |
| Reference number | 17 | |
| Indication | Long term enzyme replacement therapy in patients with a confirmed diagnosis of Pompe disease (acid α-glucosidase deficiency) |
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| Company | Genzyme Therapeutics | |
| BNF chapter | Nutrition & blood | |
| Submission type | Directed | |
| Status | Recommended | |
| Advice number | 0224 | |
| AWMSG meeting date | 14/05/2024 | |
| Date of issue | 20/05/2024 | |