Tuesday, 15th August 2023 | News, Pompe disease, Research
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.
Monday, 17th April 2023 | Fundraising, News, Pompe disease
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”.
Tuesday, 6th September 2022 | News, Research, Support
Clinical and Social Care Services Please take the chance to respond to an important survey to improve understanding about the quality of care you experience from your specialist hospital clinic, homecare, and other social care services. The survey is designed by the...
Wednesday, 24th August 2022 | News, Pompe disease, Research
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...
Saturday, 16th July 2022 | News, Support
Action Plan The Welsh Government have now published their Rare Diseases Action Plan in support of the 2021 UK Rare Disease Framework. The Wales Rare Diseases Action Plan 2022-2026 is available at the following links: English version Welsh version ...