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Wednesday, 25th January 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
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NICE recommends cipaglucosidase alfa plus miglustat. 15th August 2023
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International Pompe Day 15 April 2023. 17th April 2023
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