[HTML][HTML] Bortezomib in the rapid reduction of high sustained antibody titers in disorders treated with therapeutic protein: lessons learned from Pompe disease

SG Banugaria, SN Prater, JK McGann, JD Feldman… - Genetics in …, 2013 - Elsevier
Purpose High sustained antibody titers complicate many disorders treated with a therapeutic
protein, including those treated with enzyme replacement therapy, such as Pompe disease.
Although enzyme replacement therapy with alglucosidase alfa (Myozyme) in Pompe
disease has improved the prognosis of this otherwise lethal disorder, patients who develop
high sustained antibody titers to alglucosidase alfa enter a prolonged phase of clinical
decline resulting in death despite continued enzyme replacement therapy. Clinically …