Carrie Bailey

Safety and efficacy of avalglucosidase alfa versus alglucosidase alfa in patients with late-onset Pompe disease (COMET): a phase 3, randomised, multicentre trial

M. Tarnopolsky, Y. Chien, J. Charrow, 2021, The Lancet Neurology.

Durable and sustained immune tolerance to ERT in Pompe disease with entrenched immune responses.

D. Stockton, D. Viskochil, A. Rosenberg, 2016, JCI insight.

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

D. Viskochil, A. Rosenberg, P. Kishnani, 2012, Genetics in Medicine.

Anaplerotic therapy in propionic acidemia.

N. Longo, M. Pasquali, E. Gappmaier, 2017, Molecular genetics and metabolism.

Three cases of multi‐generational Pompe disease: Are current practices missing diagnostic and treatment opportunities?

D. Viskochil, P. Kishnani, S. Austin, 2017, American journal of medical genetics. Part A.

Durable and sustained immune tolerance to ERT in Pompe disease with entrenched immune responses.

D. Stockton, D. Viskochil, A. Rosenberg, 2016, JCI insight.