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 representatives. The risks and benefits of implementing prophylactic immune tolerance induction (ITI) to reduce the potential clinical impact of antibody development were considered. Complications due to immune responses to ERT are being recog...
Enzyme replacement therapy (ERT) has proven to be an effective therapy for some lysosomal storage di...
Cross-reactive immunological material-negative (CRIM-negative) infantile Pompe disease (IPD) patient...
Lysosomal storage disorders (LSDs) — designated as 'orphan' diseases — are inborn errors of metaboli...
The lysosomal storage disorders (LSD) are a group of severe multiple pathology disorders characteriz...
Lysosomal storage disorders are collectively important because they cause significant morbidity and ...
Lysosomal storage diseases (LSDs) are a group of rare genetic multisystemic disorders, resulting in ...
BACKGROUND: Enzyme-replacement therapy has been assessed as a treatment for patients who have mucopo...
Lysosomal storage diseases (LSDs) are rare genetic metabolic disorders that cause the accumulation o...
Enzyme replacement therapy (ERT) can produce anti-drug antibody (ADA) responses that reduce efficacy...
Enzyme replacement therapy (ERT) has been developed and trialed for the treatment of human lysosomal...
AbstractLysosomal storage diseases arise because of genetic mutations that result in nonfunctioning ...
Enzyme replacement therapy (ERT) is a treatment option for several lysosomal disorders including the...
Copyright © 2005 Federation of European Biochemical Societies Published by Elsevier B.V.Enzyme repla...
Immune response to replacement therapy has been reported for a range of therapeutic strategies being...
Enzyme replacement therapy (ERT) as treatment for lysosomal storage diseases (LSDs) was suggested as...
Enzyme replacement therapy (ERT) has proven to be an effective therapy for some lysosomal storage di...
Cross-reactive immunological material-negative (CRIM-negative) infantile Pompe disease (IPD) patient...
Lysosomal storage disorders (LSDs) — designated as 'orphan' diseases — are inborn errors of metaboli...
The lysosomal storage disorders (LSD) are a group of severe multiple pathology disorders characteriz...
Lysosomal storage disorders are collectively important because they cause significant morbidity and ...
Lysosomal storage diseases (LSDs) are a group of rare genetic multisystemic disorders, resulting in ...
BACKGROUND: Enzyme-replacement therapy has been assessed as a treatment for patients who have mucopo...
Lysosomal storage diseases (LSDs) are rare genetic metabolic disorders that cause the accumulation o...
Enzyme replacement therapy (ERT) can produce anti-drug antibody (ADA) responses that reduce efficacy...
Enzyme replacement therapy (ERT) has been developed and trialed for the treatment of human lysosomal...
AbstractLysosomal storage diseases arise because of genetic mutations that result in nonfunctioning ...
Enzyme replacement therapy (ERT) is a treatment option for several lysosomal disorders including the...
Copyright © 2005 Federation of European Biochemical Societies Published by Elsevier B.V.Enzyme repla...
Immune response to replacement therapy has been reported for a range of therapeutic strategies being...
Enzyme replacement therapy (ERT) as treatment for lysosomal storage diseases (LSDs) was suggested as...
Enzyme replacement therapy (ERT) has proven to be an effective therapy for some lysosomal storage di...
Cross-reactive immunological material-negative (CRIM-negative) infantile Pompe disease (IPD) patient...
Lysosomal storage disorders (LSDs) — designated as 'orphan' diseases — are inborn errors of metaboli...