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

[1]  O. Bodamer,et al.  Newborn Screening for Lysosomal Storage Disorders , 2016, Journal of Pediatric Genetics.

[2]  M. Gambello,et al.  Immune tolerance strategies in siblings with infantile Pompe disease — Advantages for a preemptive approach to high-sustained antibody titers , 2015, Molecular genetics and metabolism reports.

[3]  C. Rehder,et al.  CRIM-negative infantile Pompe disease: Characterization of immune responses in patients treated with ERT monotherapy , 2015, Genetics in Medicine.

[4]  Spencer T. Martin,et al.  Antibody‐Mediated Rejection in Kidney Transplantation: A Review of Pathophysiology, Diagnosis, and Treatment Options , 2014, Pharmacotherapy.

[5]  C. Morgan,et al.  Letter to the Editors: Concerning “CRIM-negative Pompe disease patients with satisfactory clinical outcomes on enzyme replacement therapy” by Al Khallaf et al , 2014, Journal of Inherited Metabolic Disease.

[6]  D. Whiteman,et al.  The relationship between anti-idursulfase antibody status and safety and efficacy outcomes in attenuated mucopolysaccharidosis II patients aged 5 years and older treated with intravenous idursulfase. , 2013, Molecular genetics and metabolism.

[7]  J. Charrow,et al.  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, PloS one.

[8]  H. Mandel,et al.  Skeletal muscle pathology of infantile Pompe disease during long-term enzyme replacement therapy , 2013, Orphanet Journal of Rare Diseases.

[9]  K. Ahn,et al.  IgE‐mediated anaphylaxis and allergic reactions to idursulfase in patients with Hunter syndrome , 2013, Allergy.

[10]  D. Matern,et al.  Newborn screening for lysosomal storage disorders and other neuronopathic conditions. , 2013, Developmental disabilities research reviews.

[11]  F. Nimmerjahn,et al.  Intravenous immunoglobulin therapy: how does IgG modulate the immune system? , 2013, Nature Reviews Immunology.

[12]  C. Hollak,et al.  Long-Term Effect of Antibodies against Infused Alpha-Galactosidase A in Fabry Disease on Plasma and Urinary (lyso)Gb3 Reduction and Treatment Outcome , 2012, PloS one.

[13]  D. Viskochil,et al.  Bortezomib in the rapid reduction of high sustained antibody titers in disorders treated with therapeutic protein: lessons learned from Pompe disease , 2012, Genetics in Medicine.

[14]  P. Kishnani,et al.  Immune modulation in Pompe disease treated with enzyme replacement therapy , 2012, Expert review of clinical immunology.

[15]  B. Schoser,et al.  The impact of antibodies in late-onset Pompe disease: a case series and literature review. , 2012, Molecular genetics and metabolism.

[16]  G. Auerswald,et al.  Immune tolerance induction in patients with severe hemophilia with inhibitors: expert panel views and recommendations for clinical practice , 2012, European journal of haematology.

[17]  C. Phornphutkul,et al.  The emerging phenotype of long-term survivors with infantile Pompe disease , 2012, Genetics in Medicine.

[18]  C. Rehder,et al.  Predicting cross‐reactive immunological material (CRIM) status in Pompe disease using GAA mutations: Lessons learned from 10 years of clinical laboratory testing experience , 2012, American journal of medical genetics. Part C, Seminars in medical genetics.

[19]  D. Dimmock,et al.  Successful immune tolerance induction to enzyme replacement therapy in CRIM-negative infantile Pompe disease , 2012, Genetics in Medicine.

[20]  Yuan-Tsong Chen,et al.  The impact of antibodies on clinical outcomes in diseases treated with therapeutic protein: Lessons learned from infantile Pompe disease , 2011, Genetics in Medicine.

[21]  B. Davidson,et al.  Clarifying lysosomal storage diseases , 2011, Trends in Neurosciences.

[22]  B. Burton,et al.  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, Molecular genetics and metabolism.

[23]  R. Haase,et al.  [Immune tolerance induction with high-dose FVIII and pulsed intravenous immunoglobulin]. , 2010, Hamostaseologie.

[24]  F. Platt,et al.  Common and Uncommon Pathogenic Cascades in Lysosomal Storage Diseases* , 2010, The Journal of Biological Chemistry.

[25]  A. Rosenberg,et al.  Elimination of antibodies to recombinant enzyme in Pompe's disease. , 2009, The New England journal of medicine.

[26]  J. Clancy,et al.  Early Treatment With Alglucosidase Alfa Prolongs Long-Term Survival of Infants With Pompe Disease , 2009, Pediatric Research.

[27]  A. Ballabio,et al.  Lysosomal disorders: from storage to cellular damage. , 2009, Biochimica et biophysica acta.

[28]  D. Rapoport,et al.  Long-term Efficacy and Safety of Laronidase in the Treatment of Mucopolysaccharidosis I , 2009, Pediatrics.

[29]  M. Mcentee,et al.  Immune tolerance improves the efficacy of enzyme replacement therapy in canine mucopolysaccharidosis I. , 2008, The Journal of clinical investigation.

[30]  Anne R. Pariser,et al.  Neutralizing antibodies to therapeutic enzymes: considerations for testing, prevention and treatment , 2008, Nature Biotechnology.

[31]  V. Gieselmann,et al.  Non-inhibitory antibodies impede lysosomal storage reduction during enzyme replacement therapy of a lysosomal storage disease , 2008, Journal of Molecular Medicine.

[32]  B. Burton,et al.  Successful Management of Difficult Infusion-Associated Reactions in a Young Patient With Mucopolysaccharidosis Type VI Receiving Recombinant Human Arylsulfatase B (Galsulfase [Naglazyme]) , 2008, Pediatrics.

[33]  J. E. Wraith,et al.  Enzyme Replacement Therapy in Patients Who Have Mucopolysaccharidosis I and Are Younger Than 5 Years: Results of a Multinational Study of Recombinant Human α-l-Iduronidase (Laronidase) , 2007, Pediatrics.

[34]  U. Ramaswami,et al.  Enzyme replacement therapy with agalsidase alfa in children with Fabry disease , 2007, Acta paediatrica.

[35]  J. Oldenburg,et al.  Genetic risk factors for inhibitors to factors VIII and IX , 2006, Haemophilia : the official journal of the World Federation of Hemophilia.

[36]  G. Herman,et al.  Chinese hamster ovary cell-derived recombinant human acid alpha-glucosidase in infantile-onset Pompe disease. , 2006, The Journal of pediatrics.

[37]  R. Schiffmann,et al.  Long-term therapy with agalsidase alfa for Fabry disease: safety and effects on renal function in a home infusion setting. , 2006, Nephrology, dialysis, transplantation : official publication of the European Dialysis and Transplant Association - European Renal Association.

[38]  C. Hollak,et al.  Enzyme therapy for Fabry disease: neutralizing antibodies toward agalsidase alpha and beta. , 2004, Kidney international.

[39]  D. Brooks,et al.  Significance of immune response to enzyme-replacement therapy for patients with a lysosomal storage disorder. , 2003, Trends in molecular medicine.

[40]  D. F. Moore,et al.  Enzyme replacement therapy in Fabry disease: a randomized controlled trial. , 2001, JAMA.

[41]  D. DeMets,et al.  Biomarkers and surrogate endpoints: Preferred definitions and conceptual framework , 2001, Clinical pharmacology and therapeutics.

[42]  C. Bond,et al.  Immune response to enzyme replacement therapy: 4-sulfatase epitope reactivity of plasma antibodies from MPS VI cats. , 1999, Molecular genetics and metabolism.

[43]  P. Meikle,et al.  Prevalence of lysosomal storage disorders. , 1999, JAMA.

[44]  G. Grabowski,et al.  Enzyme therapy in Gaucher disease type 1: effect of neutralizing antibodies to acid beta-glucosidase. , 1997, Blood.

[45]  Danielle Te Vruchte,et al.  Measuring relative lysosomal volume for monitoring lysosomal storage diseases. , 2015, Methods in cell biology.

[46]  D. Halley,et al.  Considering Fabry, but Diagnosing MPS I: Difficulties in the Diagnostic Process. , 2013, JIMD reports.

[47]  T. Ratko,et al.  Enzyme-Replacement Therapies for Lysosomal Storage Diseases , 2013 .

[48]  H. Mandel,et al.  Cross-reactive immunologic material status affects treatment outcomes in Pompe disease infants. , 2010, Molecular genetics and metabolism.

[49]  R. Giugliani,et al.  A dose-optimization trial of laronidase (Aldurazyme) in patients with mucopolysaccharidosis I. , 2009, Molecular genetics and metabolism.

[50]  C. Hollak,et al.  Immune response to enzyme replacement therapy in Fabry disease: impact on clinical outcome? , 2009, Molecular genetics and metabolism.

[51]  E. Miebach Management of infusion-related reactions to enzyme replacement therapy in a cohort of patients with mucopolysaccharidosis disorders. , 2009, International journal of clinical pharmacology and therapeutics.

[52]  B. Bénichou,et al.  A retrospective analysis of the potential impact of IgG antibodies to agalsidase beta on efficacy during enzyme replacement therapy for Fabry disease. , 2009, Molecular genetics and metabolism.