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”.
NICE recommends Avalglucosidase alfa as a treatment option for Pompe.
24th August 2022 The National Institute for Health and Care Excellence today published its recommendation of Avalglucosidase alfa (AVAL) as an option for treating Pompe in babies, children, young people and adults, where AVAL is provided according to the commercial...Update on NICE assessment of new treatments.
An update on the appraisal of treatments for Pompe by NICE.