Update on the Beam GSD1a programme.
Beam Therapeutics have put out a press release that includes an update on U.S. Food and Drug Administration clearance for their investigational new drug application for BEAM-301 in GSDIa.
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...