Information for the public
Cipaglucosidase alfa (Pombiliti) plus miglustat (Opfolda) is available on the NHS as a possible treatment for late-onset Pompe disease in adults.
Is this treatment right for me?
Your healthcare professionals should give you clear information, talk with you about your options and listen carefully to your views and concerns. Your family can be involved too, if you wish. See our webpage on making decisions about your care.
Questions to think about
- How well does it work compared with other treatments?
- What are the risks or side effects? How likely are they?
- How will the treatment affect my day-to-day life?
- What happens if the treatment does not work?
- What happens if I do not want to have treatment? Are there other treatments available?
Information and support
These organisations can give you advice and support:
- Pompe Support Network, hello@pompe.uk
- Association for Glycogen Storage Disease UK (AGSD-UK), 0300 123 2790
- Muscular Dystrophy UK, 0800 652 6352
- Metabolic Support UK, 08452 412 173
You can also get support from your local Healthwatch.
NICE is not responsible for the quality or accuracy of any information or advice provided by these organisations.
ISBN: 978-1-4731-5291-5
This page was last updated: