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Friday, 18th January 2019
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Cori disease (15)
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International (10)
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Recent news
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Save the Date!
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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.
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Newborn Genomes Programme.
4th October 2023
NICE recommends cipaglucosidase alfa plus miglustat.
15th August 2023
New Continuum of Care Model for McArdle’s.
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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
Rare Disease Day 2023.
28th February 2023
Help Us Raise Awareness.
7th February 2023
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