Indication
Long-term enzyme replacement therapy used in combination with the enzyme stabiliser miglustat for the treatment of adults with late-onset Pompe disease (LOPD; acid α-glucosidase [GAA] deficiency.
Medicine details
- Medicine name:
- cipaglucosidase alfa (Pombiliti)
- SMC ID:
- SMC2606
- Pharmaceutical company
- Amicus
- BNF chapter
- Nutrition and blood
- Submission type
- Abbreviated
- Publication due date:
- 11 December 2023