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.
New Continuum of Care Model for McArdle’s.
IamGSD has led an international team of clinicians to develop and publish a continuum of care model to help physicians and patients to achieve the patient’s optimal state.
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...
Main Event 2023 Programme
Check here for latest programme details and to register for the event.
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”.