AGSD-UK is holding a Virtual Festive Event on the 9th of December at 1.00pm. Come along and make sure you wear a Festive hat, as there will be a prize for the best headgear. We will have a Quiz and time for everyone to catch up. For more information, please contact...
Ultragenyx have provided the following community update on the development of DTX401, their investigational gene therapy for the potential treatment of GSDIa. GSDIa Community Update
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...
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.
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 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 the 18 May at...
Check here for latest programme details and to register for the event.
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”.
London Marathon 2023 AGSD-UK has 4 brilliant and brave supporters running the London Marathon on 23rd April. To read their motivation for running please have a look at the individual stories on their fundraising pages. Any donations would be very much appreciated....
Rare Disease Day is taking place on 28th February 2023 This Rare Disease Day is an opportunity to highlight the importance of effective, coordinated care for people living with rare conditions. Visit the Rare Disease Day 2023 website to find out more! Rare...
Help us raise awareness and share experiences With rare disease day approaching at the end of February can you help us raise awareness of GSD and what it's like to live with the condition? Just send a short video clip of the way your condition affects a typical...
Date change for The Main Event 2023
Register your interest now for The Main Event 2023.
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 /...
The dates for the McArdle’s walking courses for 2023 have been announced. Courses will be in Snowdonia, based in the village of Tremadog.
This Sunday is the GNR and AGSD-UK have several runners taking part.
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...
The International GSD Conference (IGSD2022) will be virtual this year.
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...
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
The updated CureGSD1b website is now live.
The updated GSD & Me website is now live.
An update on the appraisal of treatments for Pompe by NICE.
First patient receives Moderna mRNA trial infusion for GSD1a.
The commitment is at UCLH, 10am to 4pm every Wednesday, to support the McArdle team and to directly assist and guide patients.
Just a month to go to the “Walking in Wiltshire” get-together weekend for all those with McArdle’s. 17-19 June.
AGSD-UK can provide support to individuals and families affected by GSD.
The walking course for this year is to be held in the Pembrokeshire Coast National Park, 30 July to 6 August. Full details now available here on our website.
International Pompe Day – 15th April 2022
Researchers are looking for new and improved drugs and treatments for Glycogen Storage Disease.
More information about the newly published Rare Diseases Action Plan for England.
Sioned Williams celebrates Rare Disease Day with a short video talk about her life and career with McArdle’s, and an appeal via JustGiving.
Invitation to participate in research for UK adults living with Pompe.
1 week to go until Rare Disease Day 2022.
Trials for a gene therapy treatment for GSD1a are continuing.
New research information has been added to the GSD3 website page.
Over the Wall have residential and virtual camps available in 2022.
An update on avalglucosidase alfa.
AGSD-UK’s Zainib Hussain has been providing much needed support.
7 year old twins in Scotland have received their first Covid vaccinations.