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...
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...
Thursday, 16th November 2023 | News, Research, Von Gierke disease
Ultragenyx have provided the following community update on the development of DTX401, their investigational gene therapy for the potential treatment of GSDIa. GSDIa Community...
Wednesday, 4th October 2023 | News, Research
GSDs on list of conditions for inclusion in Newborn Genomes Programme research study. Genomics England has published an initial list of conditions that will be screened for as part of their forthcoming Generation Study, which includes some GSDs. The study will...
Tuesday, 15th August 2023 | News, Pompe disease, Research
The National Institute for Health and Care Excellence has today published final guidance recommending cipaglucosidase alfa (CIPA) plus miglustat, within its marketing authorisation, as an option for treating late-onset Pompe disease in adults.