alglucosidase alfa + cyclophosphamide

Alglucosidase alfa + cyclophosphamide is a combination therapy used in investigational or protocol-driven settings for patients with Pompe disease. Alglucosidase alfa is a recombinant human acid alpha-glucosidase (GAA) used as enzyme replacement therapy to degrade lysosomal glycogen, addressing GAA deficiency in Pompe disease[3][5]. Cyclophosphamide is a small molecule alkylating agent with antineoplastic and immunosuppressive properties, used to modulate the immune response, potentially to reduce antibody formation against enzyme therapy or in specific clinical protocols[4][5]. This combination regimen has been explored in certain clinical trials, typically with alglucosidase alfa administered biweekly at standard doses and cyclophosphamide administered at scheduled intervals to support tolerance or modify immune response[5].