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Monday, 20th February 2023

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Recent news

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
International Pompe Day 15 April 2023. 17th April 2023
London Marathon 2023. 24th March 2023

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The GSD variants

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