HLA- and genotype-based risk assessment model to identify infantile onset pompe disease patients at high-risk of developing significant anti-drug antibodies (ADA).

[1]  O. Abdul-Rahman,et al.  Sustained immune tolerance induction in enzyme replacement therapy-treated CRIM-negative patients with infantile Pompe disease. , 2017, JCI insight.

[2]  D. Stockton,et al.  Durable and sustained immune tolerance to ERT in Pompe disease with entrenched immune responses. , 2016, JCI insight.

[3]  P. Kishnani,et al.  Cross-reactive immunologic material positive infantile Ρompe disease: Characterization of immune responses in patient treated with enzyme replacement therapy , 2016 .

[4]  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.

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

[6]  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.

[7]  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.

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

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

[10]  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.

[11]  Anne S De Groot,et al.  Coupling sensitive in vitro and in silico techniques to assess cross-reactive CD4(+) T cells against the swine-origin H1N1 influenza virus. , 2011, Vaccine.

[12]  Leonard Moise,et al.  A Method for Individualizing the Prediction of Immunogenicity of Protein Vaccines and Biologic Therapeutics: Individualized T Cell Epitope Measure (iTEM) , 2010, Journal of biomedicine & biotechnology.

[13]  B. Byrne,et al.  Clinical outcomes after long-term treatment with alglucosidase alfa in infants and children with advanced Pompe disease , 2009, Genetics in Medicine.

[14]  A. Šimundić.  Measures of Diagnostic Accuracy: Basic Definitions , 2009, EJIFCC.

[15]  Anne S De Groot,et al.  Immunogenicity of protein therapeutics. , 2007, Trends in immunology.

[16]  W. Hwu,et al.  Recombinant human acid α-glucosidase: Major clinical benefits in infantile-onset Pompe disease , 2007 .

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

[18]  Wuh-Liang Hwu,et al.  A retrospective, multinational, multicenter study on the natural history of infantile-onset Pompe disease. , 2006, The Journal of pediatrics.

[19]  T. Voit,et al.  Safety and efficacy of recombinant acid alpha-glucosidase (rhGAA) in patients with classical infantile Pompe disease: results of a phase II clinical trial , 2005, Neuromuscular Disorders.

[20]  A. D. De Groot,et al.  Prediction of well-conserved HIV-1 ligands using a matrix-based algorithm, EpiMatrix. , 1998, Vaccine.