International Pompe Day.
Find out about new Pompe information resources
Rare Disease Day 2024.
Today is Rare Disease Day! This rarest of days, 29th February is a great opportunity to raise awareness about rare conditions. Visit https://www.rarediseaseday.org/ to find out more about how to get involved in this international campaign. Thanks...
Cipaglucosidase alfa (Pombiliti®) is accepted for use within NHS Scotland.
Cipaglucosidase alfa (Pombiliti®) is accepted for use within NHS Scotland The Scottish Medicines Consortium has completed its assessment of cipaglucosidase alfa and has advised that this offers an additional treatment choice as a long-term enzyme replacement...
Update on the DTX401 programme for GSD1a.
Ultragenyx have provided the following community update on the development of DTX401, their investigational gene therapy for the potential treatment of GSDIa. GSDIa Community...