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Recent news
- Update on the Beam GSD1a programme. 27th August 2024
- Virtual AGSD-UK support. 21st August 2024
- Update on Spark Therapeutics Pompe gene therapy programme. 23rd July 2024
- Update on phase 3 study of DTX401. 5th June 2024
- International Pompe Day. 15th April 2024
- Save the Date! 4th April 2024
- Rare Disease Day 2024. 29th February 2024
- Cipaglucosidase alfa (Pombiliti®) is accepted for use within NHS Scotland. 12th December 2023
- Virtual Festive Event. 29th November 2023
- Update on the DTX401 programme for GSD1a. 16th November 2023
- Newborn Genomes Programme. 4th October 2023
- NICE recommends cipaglucosidase alfa plus miglustat. 15th August 2023
- New Continuum of Care Model for McArdle’s. 7th July 2023
- Seminar for people affected by neuromuscular diseases. 17th May 2023
- Main Event 2023 Programme 26th April 2023