The capacity of protein aggregates to enhance immune responses to the monomeric form of
the protein has been known for over a half-century. Despite the clear connection between …

Immunological and inflammatory processes downstream of dystrophin deficiency as well as
metabolic abnormalities, defective autophagy, and loss of regenerative capacity all …

Purpose: Enzyme replacement therapy with rhGAA (Myozyme®) has lead to improved
survival, which is largely attributable to improvements in cardiomyopathy and skeletal muscle …

Purpose Infantile Pompe disease resulting from a deficiency of lysosomal acid α-glucosidase
(GAA) requires enzyme replacement therapy (ERT) with recombinant human GAA (rhGAA). …

In the midst of resurging COVID-19 cases, the second NIH/FDA virtual COVID-19 and Cytokines
symposium was held on 1 December 2020, focusing on longitudinal studies of COVID-…

“The role of cytokines in COVID-19” online symposium was presented on 18 June 2020 by
the NIH/FDA Immunology and Cytokine Interest Groups and was purposed to discuss our …

To the Editor: Infantile Pompe's disease is due to a deficiency of lysosomal acid alpha
glucosidase (GAA). In patients in whom GAA is not produced, a status called cross-reacting …

Objective Here we provide a critical review of the state of the art with respect to non-clinical
assessments of immunogenicity for therapeutic proteins. Key findings The number of studies …

Objective Although enzyme replacement therapy (ERT) is a highly effective therapy, CRIM-negative
(CN) infantile Pompe disease (IPD) patients typically mount a strong immune …

Therapeutic proteins provide interventions for some of the most complex and intractable
diseases and are an essential part of modern medicine. Immunogenicity is the development of …