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.
Seminar for people affected by neuromuscular diseases.
Seminar Thursday 18th May – 5PM UK The research team at Newcastle University’s John Walton centre are keen for people affected by neuromuscular conditions to join a seminar on 18th May: ‘We are glad to announce that we will be delivering a seminar on...
International Pompe Day 15 April 2023.
The IPA focused this year’s International Pompe Day on the importance of movement, sharing stories from the community under the slogan “Every Move Counts”.