Tuesday, 23rd July 2024 | News, Pompe disease, Research
Spark have provided the following community update on their Pompe gene therapy programme.
Wednesday, 5th June 2024 | News, Research, Von Gierke disease
Ultragenyx have provided the following community update on the phase 3 study of DTX401, their investigational gene therapy for the potential treatment of GSDIa. GSD1a Community...
Monday, 15th April 2024 | News, Pompe disease
Find out about new Pompe information resources
Thursday, 4th April 2024 | News, Support
Main Event 2024
Thursday, 29th February 2024 | News, Support
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...
Tuesday, 12th December 2023 | News, Pompe disease, Research
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...