Newborn Genomes Programme.
GSDs on list of conditions for inclusion in Newborn Genomes Programme research study. Genomics England has published an initial list of conditions that will be screened for as part of their forthcoming Generation Study, which includes some GSDs. The study will...
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.
Liberating Research GSD1a Study.
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 /...
LSD Collaborative Community Survey 2022.
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...
International GSD conference 2022.
The International GSD Conference (IGSD2022) will be virtual this year.