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.
Tuesday, 1st November 2022 | News, Research, Support, Von Gierke disease
GSD1a Patient Research Please find below a link to an invitation for UK families affected by GSD1a to take part research by Liberating Research to improve understanding of the impact of the condition. Who can participate?People living with GSD1a and their partners /...
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...
Tuesday, 30th August 2022 | International, News, Research
The International GSD Conference (IGSD2022) will be virtual this year.
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, 11th June 2022 | News, Pompe disease, Research
An update on the appraisal of treatments for Pompe by NICE.
