April 15th is International Pompe Day.
To help raise awareness Emmie has been talking about daughter Arla and the family’s experience on infant onset Pompe. You can find out more here.
15th April is International Pompe Day.
Find out more about what a typical day might look like for community members affected by Pompe and what they find most helpful.
Update on Spark Therapeutics Pompe gene therapy programme.
Spark have provided the following community update on their Pompe gene therapy programme.
International Pompe Day.
Find out about new Pompe information resources
Cipaglucosidase alfa (Pombiliti®) is accepted for use within NHS Scotland.
Cipaglucosidase alfa (Pombiliti®) is accepted for use within NHS Scotland The Scottish Medicines Consortium has completed its assessment of cipaglucosidase alfa and has advised that this offers an additional treatment choice as a long-term enzyme replacement...
NICE recommends cipaglucosidase alfa plus miglustat.
The National Institute for Health and Care Excellence has today published final guidance recommending cipaglucosidase alfa (CIPA) plus miglustat, within its marketing authorisation, as an option for treating late-onset Pompe disease in adults.