Immune response to enzyme replacement therapies in lysosomal storage diseases and the role of immune tolerance induction
PS Kishnani, PI Dickson, L Muldowney, JJ Lee… - Molecular genetics and …, 2016 - Elsevier
Abstract The US Food and Drug Administration (FDA) and National Organization for Rare
Disease (NORD) convened a public workshop titled “Immune Responses to Enzyme
Replacement Therapies: Role of Immune Tolerance Induction” to discuss the impact of anti-
drug antibodies (ADAs) on efficacy and safety of enzyme replacement therapies (ERTs)
intended to treat patients with lysosomal storage diseases (LSDs). Participants in the
workshop included FDA staff, clinicians, scientists, patients, industry, and advocacy group …
Disease (NORD) convened a public workshop titled “Immune Responses to Enzyme
Replacement Therapies: Role of Immune Tolerance Induction” to discuss the impact of anti-
drug antibodies (ADAs) on efficacy and safety of enzyme replacement therapies (ERTs)
intended to treat patients with lysosomal storage diseases (LSDs). Participants in the
workshop included FDA staff, clinicians, scientists, patients, industry, and advocacy group …