Posted by Michael Wonder on 15 Aug 2023
Cipaglucosidase alfa and miglustat for the treatment of patients with late-onset Pompe disease
15 August 2023 - NICE has published evidence-based recommendations on the use of cipaglucosidase alfa (Pombiliti) with miglustat (Opfolda) for the treatment of adults with late-onset Pompe disease.
Cipaglucosidase alfa, when used in combination with miglustat, is recommended for the treatment of adults with late-onset Pompe disease in adults only if Amicus Therapeutics provides it according to the commercial arrangement.
