Several studies have reported that use of immunosuppressant drugs, such as cyclophosphamide, mycophenolate mofetil, belimumab, rituximab, bortezomib, and MTX can lead to successful induction of immune tolerance in GAA-deficient mice and in humans with infantile Pompe disease [9], [10], [11], [12], [13], [21], [27]. This evidence concerns the gene GAA and glycogen storage disease II.