Publications by authors named "Lina Fiege"
Improved Enzyme Replacement Therapy with Cipaglucosidase Alfa/Miglustat in Infantile Pompe Disease.
Pharmaceuticals (Basel)
August 2023
Article Synopsis
- - Pompe disease is a serious genetic disorder caused by a deficiency in the enzyme acid α-glucosidase (GAA), leading to severe health issues in infants without treatment and typically resulting in death within the first year.
- - Enzyme replacement therapy (ERT) has been used since 2006 with Myozyme (recombinant human GAA), which prolongs life but has limited effectiveness against disease progression.
- - A new treatment, Cipaglucosidase alfa combined with Miglustat, has shown significant improvements in a patient’s symptoms such as respiratory failure and motor skills, indicating its potential superiority over previous therapies.