The impact of the immune system on the safety and efficiency of enzyme replacement therapy in lysosomal storage disorders

Immune response to enzyme replacement therapies in lysosomal storage diseases and the role of immune tolerance induction

(2016) Priya S. Kishnani et al.   MOLECULAR GENETICS AND METABOLISM

The role of antibodies in enzyme treatments and therapeutic strategies

(2015) Brian W. Bigger et al.   BEST PRACTICE & RESEARCH CLINICAL ENDOCRINOLOGY & METABOLISM

BAFF blockade prevents anti-drug antibody formation in a mouse model of Pompe disease

(2015) Phillip A. Doerfler et al.   CLINICAL IMMUNOLOGY

Immunogenicity of Elosulfase Alfa, an Enzyme Replacement Therapy in Patients With Morquio A Syndrome: Results From MOR-004, a Phase III Trial

(2015) Becky Schweighardt et al.   CLINICAL THERAPEUTICS

Role of B cells in tolerance induction

(2015) James I. Kim et al.   Current Opinion in Organ Transplantation

Involvement of the TNF-α/TGF-β/IDO axis in IVIg-induced immune tolerance

(2015) Lionel Loubaki et al.   CYTOKINE

Regulatory B Cells: Origin, Phenotype, and Function

(2015) Elizabeth C. Rosser et al.   IMMUNITY

Inhibitor recurrence after immune tolerance induction: a multicenter retrospective cohort study

(2015) A. Antun et al.   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

Safety and efficacy of enzyme replacement therapy with idursulfase beta in children aged younger than 6years with Hunter syndrome

(2015) Young Bae Sohn et al.   MOLECULAR GENETICS AND METABOLISM

Biomarker responses correlate with antibody status in mucopolysaccharidosis type I patients on long-term enzyme replacement therapy

(2015) Eveline J. Langereis et al.   MOLECULAR GENETICS AND METABOLISM

Sleep disordered breathing in mucopolysaccharidosis I: a multivariate analysis of patient, therapeutic and metabolic correlators modifying long term clinical outcome

(2015) Abhijit Ricky Pal et al.   Orphanet Journal of Rare Diseases

Immunogenicity of idursulfase and clinical outcomes in very young patients (16 months to 7.5 years) with mucopolysaccharidosis II (Hunter syndrome)

(2015) Arian Pano et al.   Orphanet Journal of Rare Diseases

Oral delivery of Acid Alpha Glucosidase epitopes expressed in plant chloroplasts suppresses antibody formation in treatment of Pompe mice

(2015) Jin Su et al.   PLANT BIOTECHNOLOGY JOURNAL

Regulation of immune responses to protein therapeutics by transplacental induction of T cell tolerance

(2015) Nimesh Gupta et al.   Science Translational Medicine

Antigen Presentation, Autoantigens, and Immune Regulation in Multiple Sclerosis and Other Autoimmune Diseases

(2015) Christine Riedhammer et al.   Frontiers in Immunology

A large-scale computational study of inhibitor risk in non-severe haemophilia A

(2014) Adrian J. Shepherd et al.   BRITISH JOURNAL OF HAEMATOLOGY

Tregitope: Immunomodulation Powerhouse

(2014) Leslie Cousens et al.   HUMAN IMMUNOLOGY

Identification of an N-Linked Glycosylation in the C4 Region of HIV-1 Envelope gp120 That Is Critical for Recognition of Neighboring CD4 T Cell Epitopes

(2014) H. Li et al.   JOURNAL OF IMMUNOLOGY

Transient Low-Dose Methotrexate Generates B Regulatory Cells That Mediate Antigen-Specific Tolerance to Alglucosidase Alfa

(2014) Marguerite S. Joly et al.   JOURNAL OF IMMUNOLOGY

Efficacy and safety of enzyme replacement therapy with BMN 110 (elosulfase alfa) for Morquio A syndrome (mucopolysaccharidosis IVA): a phase 3 randomised placebo-controlled study

(2014) Christian J. Hendriksz et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Enzyme therapy and immune response in relation to CRIM status: the Dutch experience in classic infantile Pompe disease

(2014) Carin M. van Gelder et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Long-term safety and efficacy of rituximab in 7 Japanese patients with ANCA-associated vasculitis

(2014) Hiroko Nagafuchi et al.   Modern Rheumatology

Complement activation-related pseudoallergy: A stress reaction in blood triggered by nanomedicines and biologicals

(2014) Janos Szebeni   MOLECULAR IMMUNOLOGY

IgE-mediated anaphylaxis and allergic reactions to idursulfase in patients with Hunter syndrome

(2013) J. Kim et al.   ALLERGY

T-cell dependent immunogenicity of protein therapeutics: Preclinical assessment and mitigation

(2013) Vibha Jawa et al.   CLINICAL IMMUNOLOGY

A multicenter open-label treatment protocol (HGT-GCB-058) of velaglucerase alfa enzyme replacement therapy in patients with Gaucher disease type 1: safety and tolerability

(2013) Gregory M. Pastores et al.   GENETICS IN MEDICINE

Allo-Immune Membranous Nephropathy and Recombinant Aryl Sulfatase Replacement Therapy: A Need for Tolerance Induction Therapy

(2013) H. Debiec et al.   JOURNAL OF THE AMERICAN SOCIETY OF NEPHROLOGY

The relationship between anti-idursulfase antibody status and safety and efficacy outcomes in attenuated mucopolysaccharidosis II patients aged 5years and older treated with intravenous idursulfase

(2013) A.J. Barbier et al.   MOLECULAR GENETICS AND METABOLISM

Fabry disease peripheral blood immune cells release inflammatory cytokines: Role of globotriaosylceramide

(2013) Pablo N. De Francesco et al.   MOLECULAR GENETICS AND METABOLISM

A new assay for fast, reliable CRIM status determination in infantile-onset Pompe disease

(2013) Zhaohui Wang et al.   MOLECULAR GENETICS AND METABOLISM

Modulation of CD8+ T cell responses to AAV vectors with IgG-derived MHC class II epitopes

(2013) Daniel J Hui et al.   MOLECULAR THERAPY

Innate B cell helpers reveal novel types of antibody responses

(2013) Carola G Vinuesa et al.   NATURE IMMUNOLOGY

Mucopolysaccharidosis type VI phenotypes-genotypes and antibody response to galsulfase

(2013) Marion M Brands et al.   Orphanet Journal of Rare Diseases

Pentosan Polysulfate: A Novel Therapy for the Mucopolysaccharidoses

(2013) Edward H. Schuchman et al.   PLoS One

Algorithm for the Early Diagnosis and Treatment of Patients with Cross Reactive Immunologic Material-Negative Classic Infantile Pompe Disease: A Step towards Improving the Efficacy of ERT

(2013) Suhrad G. Banugaria et al.   PLoS One

Overview of Complement Activation and Regulation

(2013) Marina Noris et al.   SEMINARS IN NEPHROLOGY

Polymorphisms in the F8 Gene and MHC-II Variants as Risk Factors for the Development of Inhibitory Anti-Factor VIII Antibodies during the Treatment of Hemophilia A: A Computational Assessment

(2013) Gouri Shankar Pandey et al.   PLoS Computational Biology

Predicting cross-reactive immunological material (CRIM) status in Pompe disease using GAA mutations: Lessons learned from 10 years of clinical laboratory testing experience

(2012) Deeksha S. Bali et al.   AMERICAN JOURNAL OF MEDICAL GENETICS PART C-SEMINARS IN MEDICAL GENETICS

Production platforms for biotherapeutic glycoproteins. Occurrence, impact, and challenges of non-human sialylation

(2012) Darius Ghaderi et al.   Biotechnology & Genetic Engineering Reviews

Immune modulation in Pompe disease treated with enzyme replacement therapy

(2012) Suhrad G Banugaria et al.   Expert Review of Clinical Immunology

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

(2012) Suhrad G. Banugaria et al.   GENETICS IN MEDICINE

Successful immune tolerance induction to enzyme replacement therapy in CRIM-negative infantile Pompe disease

(2012) Yoav H. Messinger et al.   GENETICS IN MEDICINE

Hematopoietic stem cell transplantation improves the high incidence of neutralizing allo-antibodies observed in Hurler's syndrome after pharmacological enzyme replacement therapy

(2012) M. A. Saif et al.   HAEMATOLOGICA

Successful Desensitization to Imiglucerase of an Adult Patient Diagnosed with Type I Gaucher Disease

(2012) Derya Erdogdu et al.   INTERNATIONAL ARCHIVES OF ALLERGY AND IMMUNOLOGY

Mapping the T helper cell response to acid α-glucosidase in Pompe mice

(2012) Sushrusha Nayak et al.   MOLECULAR GENETICS AND METABOLISM

Immunological cell type characterization and Th1–Th17 cytokine production in a mouse model of Gaucher disease

(2012) Manoj Kumar Pandey et al.   MOLECULAR GENETICS AND METABOLISM

A revised home treatment algorithm for Fabry disease: Influence of antibody formation

(2012) B.E. Smid et al.   MOLECULAR GENETICS AND METABOLISM

Administration of Anti-CD3 Antibodies Modulates the Immune Response to an Infusion of α-glucosidase in Mice

(2012) Toya Ohashi et al.   MOLECULAR THERAPY

Human cells: New platform for recombinant therapeutic protein production

(2012) Kamilla Swiech et al.   PROTEIN EXPRESSION AND PURIFICATION

Idursulfase treatment of Hunter syndrome in children younger than 6 years: Results from the Hunter Outcome Survey

(2011) Joseph Muenzer et al.   GENETICS IN MEDICINE

The impact of antibodies on clinical outcomes in diseases treated with therapeutic protein: Lessons learned from infantile Pompe disease

(2011) Suhrad G. Banugaria et al.   GENETICS IN MEDICINE

Dendritic cell control of tolerogenic responses

(2011) Santhakumar Manicassamy et al.   IMMUNOLOGICAL REVIEWS

Regulatory T cells and Foxp3

(2011) Alexander Y. Rudensky   IMMUNOLOGICAL REVIEWS

Coupling pathogen recognition to innate immunity through glycan-dependent mechanisms

(2011) Roberto C. Davicino et al.   INTERNATIONAL IMMUNOPHARMACOLOGY

Longitudinal observations of serum heparin cofactor II-thrombin complex in treated Mucopolysaccharidosis I and II patients

(2011) Lorne Andrew Clarke et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Fabry disease, enzyme replacement therapy and the significance of antibody responses

(2011) Patrick B. Deegan   JOURNAL OF INHERITED METABOLIC DISEASE

Treatment of mucopolysaccharidosis type II (Hunter syndrome) with idursulfase: the relevance of clinical trial end points

(2011) Emma Glamuzina et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Successful immune tolerance induction by FVIII in hemophilia A patients with inhibitor may occur without deletion of FVIII-specific T cells

(2011) B. PAUTARD et al.   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

Oral administration of recombinant human acid α-glucosidase reduces specific antibody formation against enzyme in mouse

(2011) T. Ohashi et al.   MOLECULAR GENETICS AND METABOLISM

An individually, modified approach to desensitize infants and young children with Pompe disease, and significant reactions to alglucosidase alfa infusions

(2011) Areeg H. El-Gharbawy et al.   MOLECULAR GENETICS AND METABOLISM

Incidence and timing of infusion-related reactions in patients with mucopolysaccharidosis type II (Hunter syndrome) on idursulfase therapy in the real-world setting: A perspective from the Hunter Outcome Survey (HOS)

(2011) Barbara K. Burton et al.   MOLECULAR GENETICS AND METABOLISM

Anti-α-galactosidase A antibody response to agalsidase beta treatment: Data from the Fabry Registry

(2011) William R. Wilcox et al.   MOLECULAR GENETICS AND METABOLISM

Enzyme replacement therapy induces T-cell responses in late-onset Pompe disease

(2011) Miklos Banati et al.   MUSCLE & NERVE

Anti-TNF-Alpha Therapy Enhances the Effects of Enzyme Replacement Therapy in Rats with Mucopolysaccharidosis Type VI

(2011) Efrat Eliyahu et al.   PLoS One

Phase 1/2 and extension study of velaglucerase alfa replacement therapy in adults with type 1 Gaucher disease: 48-month experience

(2010) A. Zimran et al.   BLOOD

Immune tolerance induction to enzyme-replacement therapy by co-administration of short-term, low-dose methotrexate in a murine Pompe disease model

(2010) A. Joseph et al.   CLINICAL AND EXPERIMENTAL IMMUNOLOGY

Long-term, open-labeled extension study of idursulfase in the treatment of Hunter syndrome

(2010) Joseph Muenzer et al.   GENETICS IN MEDICINE

Maintenance of cell surface glycan density by lectin-glycan interactions: A homeostatic and innate immune regulatory mechanism

(2010) T. K. Dam et al.   GLYCOBIOLOGY

Heparin cofactor II-thrombin complex and dermatan sulphate:chondroitin sulphate ratio are biomarkers of short- and long-term treatment effects in mucopolysaccharide diseases

(2010) Kia Jane Langford-Smith et al.   JOURNAL OF INHERITED METABOLIC DISEASE

CRIM-negative infantile Pompe disease: 42-month treatment outcome

(2010) Marianne Rohrbach et al.   JOURNAL OF INHERITED METABOLIC DISEASE

On the Role of Aggregates in the Immunogenicity of Recombinant Human Interferon Beta in Patients with Multiple Sclerosis

(2010) Miranda M.C. van Beers et al.   JOURNAL OF INTERFERON AND CYTOKINE RESEARCH

Impact of Product-Related Factors on Immunogenicity of Biotherapeutics

(2010) Satish Kumar Singh   JOURNAL OF PHARMACEUTICAL SCIENCES

Induction of Immune Tolerance to a Therapeutic Protein by Intrathymic Gene Delivery

(2010) Qiuming Chu et al.   MOLECULAR THERAPY

Implications of the presence of N-glycolylneuraminic acid in recombinant therapeutic glycoproteins

(2010) Darius Ghaderi et al.   NATURE BIOTECHNOLOGY

Aggregated Recombinant Human Interferon Beta Induces Antibodies but No Memory in Immune-Tolerant Transgenic Mice

(2010) Miranda M. C. van Beers et al.   PHARMACEUTICAL RESEARCH

Laboratory Assessment of Factor VIII Inhibitor Titer

(2009) Ellinor I.B. Peerschke et al.   AMERICAN JOURNAL OF CLINICAL PATHOLOGY

Progressive multifocal leukoencephalopathy after rituximab therapy in HIV-negative patients: a report of 57 cases from the Research on Adverse Drug Events and Reports project

(2009) K. R. Carson et al.   BLOOD

Reducing the Immunogenicity of Protein Therapeutics

(2009) Masanori Onda   CURRENT DRUG TARGETS

Clinical outcomes after long-term treatment with alglucosidase alfa in infants and children with advanced Pompe disease

(2009) Marc Nicolino et al.   GENETICS IN MEDICINE

Natural and Adaptive Foxp3+ Regulatory T Cells: More of the Same or a Division of Labor?

(2009) Maria A. Curotto de Lafaille et al.   IMMUNITY

Modulating dendritic cells (DC) from immunogenic to tolerogenic responses: A novel mechanism of AZA/6-MP

(2009) Alessandra Aldinucci et al.   JOURNAL OF NEUROIMMUNOLOGY

The molecular mechanisms of immunomodulation and tolerance induction to factor VIII

(2009) B. WATERS et al.   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

Repeated intrathecal injections of recombinant human 4-sulphatase remove dural storage in mature mucopolysaccharidosis VI cats primed with a short-course tolerisation regimen

(2009) Dyane Auclair et al.   MOLECULAR GENETICS AND METABOLISM

Cross-reactive immunologic material status affects treatment outcomes in Pompe disease infants

(2009) Priya S. Kishnani et al.   MOLECULAR GENETICS AND METABOLISM

Genzyme's Lumizyme clears bioequivalence hurdles

(2009) Mark Ratner   NATURE BIOTECHNOLOGY

Elimination of Antibodies to Recombinant Enzyme in Pompe's Disease

(2009) Nancy J. Mendelsohn et al.   NEW ENGLAND JOURNAL OF MEDICINE

Early Treatment With Alglucosidase Alfa Prolongs Long-Term Survival of Infants With Pompe Disease

(2009) Priya S Kishnani et al.   PEDIATRIC RESEARCH

Multidisciplinary Management of Hunter Syndrome

(2009) J. Muenzer et al.   PEDIATRICS

Indications et mécanismes d’action des immunoglobulines intraveineuses dans les pathologies auto-immunes et inflammatoires systémiques

(2009) L. Mouthon et al.   REVUE DE MEDECINE INTERNE

Plant-made vaccine antigens and biopharmaceuticals

(2009) Henry Daniell et al.   TRENDS IN PLANT SCIENCE

Comparison of Neutralizing Antibody Assays for Receptor Binding and Enzyme Activity of the Enzyme Replacement Therapeutic Naglazyme® (Galsulfase)

(2008) Joleen T. White et al.   AAPS Journal

Long-term safety of methotrexate monotherapy in patients with rheumatoid arthritis: a systematic literature research

(2008) C Salliot et al.   ANNALS OF THE RHEUMATIC DISEASES

Diversity in specificity, abundance, and composition of anti-Neu5Gc antibodies in normal humans: Potential implications for disease

(2008) V. Padler-Karavani et al.   GLYCOBIOLOGY

Tuning Microenvironments: Induction of Regulatory T Cells by Dendritic Cells

(2008) Yasmine Belkaid et al.   IMMUNITY

Recommendations on risk-based strategies for detection and characterization of antibodies against biotechnology products

(2008) Eugen Koren et al.   JOURNAL OF IMMUNOLOGICAL METHODS

Recommendations for the validation of immunoassays used for detection of host antibodies against biotechnology products

(2008) Gopi Shankar et al.   JOURNAL OF PHARMACEUTICAL AND BIOMEDICAL ANALYSIS

Long-term follow-up of endurance and safety outcomes during enzyme replacement therapy for mucopolysaccharidosis VI: Final results of three clinical studies of recombinant human N-acetylgalactosamine 4-sulfatase

(2008) Paul Harmatz et al.   MOLECULAR GENETICS AND METABOLISM

Neutralizing antibodies to therapeutic enzymes: considerations for testing, prevention and treatment

(2008) Jinhai Wang et al.   NATURE BIOTECHNOLOGY

Long-term Efficacy and Safety of Laronidase in the Treatment of Mucopolysaccharidosis I

(2008) L. A. Clarke et al.   PEDIATRICS

Identification of a receptor required for the anti-inflammatory activity of IVIG

(2008) R. M. Anthony et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Effects of enzyme replacement therapy on the cardiomyopathy of Anderson Fabry disease: a randomised, double-blind, placebo-controlled clinical trial of agalsidase alfa

(2007) D A Hughes et al.   HEART