Cipaglucosidase alfa with miglustat is in development for the treatment of late onset Pompe disease in children aged 12-17 years. Pompe disease is an inherited, genetic disorder which results in the deficiency of the enzyme 'acid alpha-glucosidase’. This deficiency leads to progressive accumulation of glycogen, a type of sugar, usually stored in muscle tissues particularly around the heart, skeletal muscle and diaphragm. Enzyme replacement therapy is the currently recommended treatment approach but does not reverse the primary abnormality. There are also not many options for treating children.
Cipaglucosidase alfa/miglustat for treating children and adolescents with late-onset Pompe disease
Cipaglucosidase alfa with miglustat is in development for the treatment of late onset Pompe disease in children aged 12-17 years. Pompe disease is an inherited, genetic disorder which results in the deficiency of the enzyme 'acid alpha-glucosidase’.