Report AE

This website is not intended to be a substitute for medical advice. Please refer to the Patient Information Leaflet in the pack for full safety information.

▼ This medicine is subject to additional monitoring. This will allow quick identification of new safety information. You can help by reporting any side effects you may get. See www.mhra.gov.uk/yellowcard for how to report side effects.

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Support & resources

Various patient advocacy groups (PAGs) offer support for people with Pompe disease in the UK.

Please click the below to find out more:

Association for Glycogen Storage Disease UK - a UK-wide organisation offering support and services for people affected by Pompe and other glycogen storage disorders and their families.

Pompe Support Network - a support network specifically for those affected by Pompe disease.

Metabolic Support UK - a patient organisation for Inherited Metabolic Disorders supporting thousands of patients worldwide.

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Please click here to download a children’s book for living with Pompe.

Glossary of important terms

  • Acid alpha-glucosidase (GAA): an enzyme responsible for breaking down glycogen to glucose. This enzyme is in low amounts or not working in people with Pompe disease
  • Bind: Connect a cell and another substance (enzyme, medication, etc)
  • Echocardiogram: A painless and noninvasive technique to check how the heart is working through the use of ultrasound waves
  • Enzyme: A type of protein that facilitates biochemical reactions in the body
  • Enzyme replacement therapy (ERT): A treatment that replaces an enzyme that is missing or only present in low amounts
  • Glucose: A type of simple sugar that is used by the body for energy
  • Glycogen: A complex sugar made up of many bits of glucose, stored in cells (especially muscle cells) or broken down and used by the body for energy
  • Intravenous infusion: A method of giving medicine via a drip through a needle placed into a vein
  • Lysosome: An inner part of the cell that holds many different enzymes and acts as the digestive system for the cell to break down complex molecules
  • Mannose-6-phosphate (M6P): A type of marker on both natural and therapeutic enzymes that helps them bind and enter the cells and lysosomes where they belong
  • MYOZYME® (alglucosidase alfa): Another ERT option for the treatment of Pompe disease
  • Uptake: The process of bringing a material (enzymes, medications, sugars, etc) from outside the cell to inside the cell so it can be broken down and used by the body
  • Ventilator: A machine that moves air in and out of the lungs to help people who cannot breathe properly

This website is not intended to be a substitute for medical advice. Please refer to the Patient Information Leaflet in the pack for full safety information.

Reporting of side effects:

If you get any side effects, talk to your doctor, pharmacist or nurse. This includes any possible side effects not listed in the package leaflet. You can also report side effects directly via the Yellow Card Scheme at www.mhra.gov.uk/yellowcard

By reporting side effects, you can help provide more information on the safety of this medicine.

Intended for patients prescribed Nexviadyme (Avalglucosidase alfa) or their caregiver