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Friday, 26th April 2019
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Cori disease (15)
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GSD families (34)
Hers disease GSD6 (3)
International (10)
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Recent news
International Pompe Day.
15th April 2024
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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.
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Update on the DTX401 programme for GSD1a.
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Newborn Genomes Programme.
4th October 2023
NICE recommends cipaglucosidase alfa plus miglustat.
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New Continuum of Care Model for McArdle’s.
7th July 2023
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Main Event 2023 Programme
26th April 2023
International Pompe Day 15 April 2023.
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London Marathon 2023.
24th March 2023
Rare Disease Day 2023.
28th February 2023
Help Us Raise Awareness.
7th February 2023
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