Benefits of Prophylactic Short-Course Immune Tolerance Induction in Patients With Infantile Pompe Disease: Demonstration of Long-Term Safety and Efficacy in an Expanded Cohort

Challenges in treating Pompe disease: an industry perspective

(2019) Hung V. Do et al.   Annals of Translational Medicine

Immunological challenges and approaches to immunomodulation in Pompe disease: a literature review

(2019) Ankit K. Desai et al.   Annals of Translational Medicine

Methotrexate and BAFF interaction prevents immunization against TNF inhibitors

(2018) Samuel Bitoun et al.   ANNALS OF THE RHEUMATIC DISEASES

Consequences of B-cell-depleting therapy: hypogammaglobulinemia and impaired B-cell reconstitution

(2018) Keith A Sacco et al.   Immunotherapy

High Sustained Antibody Titers in Patients with Classic Infantile Pompe Disease Following Immunomodulation at Start of Enzyme Replacement Therapy

(2018) Esther Poelman et al.   JOURNAL OF PEDIATRICS

An immune tolerance approach using transient low-dose methotrexate in the ERT-naïve setting of patients treated with a therapeutic protein: experience in infantile-onset Pompe disease

(2018) Zoheb B. Kazi et al.   GENETICS IN MEDICINE

Methotrexate BAFFles anti-drug antibodies

(2018) Bruce N. Cronstein   Nature Reviews Rheumatology

Author Correction: Lysosomal storage diseases

(2018) Frances M. Platt et al.   Nature Reviews Disease Primers

Management of Confirmed Newborn-Screened Patients With Pompe Disease Across the Disease Spectrum

(2017) David F. Kronn et al.   PEDIATRICS

Suppression of normal immune responses after treatment with rituximab

(2016) Ruba Kado et al.   CURRENT OPINION IN RHEUMATOLOGY

Very Early Treatment for Infantile-Onset Pompe Disease Contributes to Better Outcomes

(2016) Chia-Feng Yang et al.   JOURNAL OF PEDIATRICS

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

Diverse effects on vaccine-specific serum IgG titres and memory B cells upon methotrexate and anti-TNF-α therapy in children with rheumatic diseases: A cross-sectional study

(2016) Hanna M. Ingelman-Sundberg et al.   VACCINE

The role of antibodies in enzyme treatments and therapeutic strategies

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

CRIM-negative infantile Pompe disease: characterization of immune responses in patients treated with ERT monotherapy

(2015) Kathryn L. Berrier et al.   GENETICS IN MEDICINE

Continuing IgG replacement therapy for hypogammaglobulinemia after rituximab—for how long?

(2015) Sara Barmettler et al.   JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY

B Cell Reconstitution after Rituximab Treatment in Idiopathic Nephrotic Syndrome

(2015) M. Colucci et al.   JOURNAL OF THE AMERICAN SOCIETY OF NEPHROLOGY

Pharmacological Chaperone Therapy: Preclinical Development, Clinical Translation, and Prospects for the Treatment of Lysosomal Storage Disorders

(2015) Giancarlo Parenti et al.   MOLECULAR THERAPY

Immunreconstitution and Infectious Complications After Rituximab Treatment in Children and Adolescents: What Do We Know and What Can We Learn from Adults?

(2015) Jennifer Worch et al.   Cancers

The indications, efficacy and adverse events of rituximab in a large cohort of patients with juvenile-onset SLE

(2014) L Watson et al.   LUPUS

Utility and safety of rituximab in pediatric autoimmune and inflammatory CNS disease

(2014) R. C. Dale et al.   NEUROLOGY

Distinct Effects of Methotrexate and Etanercept on the B Cell Compartment in Patients With Juvenile Idiopathic Arthritis

(2014) Stephanie Glaesener et al.   Arthritis & Rheumatology

Rituximab and Immune Deficiency: Case Series and Review of the Literature

(2014) Blanka Kaplan et al.   Journal of Allergy and Clinical Immunology-In Practice

B-Cell Depletion and Immunomodulation before Initiation of Enzyme Replacement Therapy Blocks the Immune Response to Acid Alpha-Glucosidase in Infantile-Onset Pompe Disease

(2013) Melissa E. Elder et al.   JOURNAL OF PEDIATRICS

Lysosomal storage diseases—the horizon expands

(2013) Rose-Mary Naaman Boustany   Nature Reviews Neurology

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

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

Enzyme Replacement Therapy for Lysosomal Diseases: Lessons from 20 Years of Experience and Remaining Challenges

(2012) R.J. Desnick et al.   Annual Review of Genomics and Human Genetics

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

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

The emerging phenotype of long-term survivors with infantile Pompe disease

(2012) Sean N. Prater et al.   GENETICS IN MEDICINE

Use and interpretation of diagnostic vaccination in primary immunodeficiency: A working group report of the Basic and Clinical Immunology Interest Section of the American Academy of Allergy, Asthma & Immunology

(2012) Jordan S. Orange et al.   JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY

Rituximab resistance

(2011) Andrew R. Rezvani et al.   BEST PRACTICE & RESEARCH CLINICAL HAEMATOLOGY

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

Atypical immunologic response in a patient with CRIM-negative Pompe disease

(2011) Mary-Alice Abbott et al.   MOLECULAR GENETICS AND METABOLISM

Rituximab efficiency in children with steroid-dependent nephrotic syndrome

(2010) Anne-Laure Sellier-Leclerc et al.   PEDIATRIC NEPHROLOGY

Alglucosidase alfa: Long term use in the treatment of patients with Pompe disease

(2010) Michael Beck   Therapeutics and Clinical Risk Management

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

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

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

Elimination of Antibodies to Recombinant Enzyme in Pompe's Disease

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

Single dose of rituximab for refractory steroid-dependent nephrotic syndrome in children

(2009) Koichi Kamei et al.   PEDIATRIC NEPHROLOGY

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

(2009) Priya S Kishnani et al.   PEDIATRIC RESEARCH

A Randomized, Prospective Trial of Rituximab for Acute Rejection in Pediatric Renal Transplantation

(2008) V. Zarkhin et al.   AMERICAN JOURNAL OF TRANSPLANTATION

Safety, efficacy, and immune reconstitution after rituximab therapy in pediatric patients with chronic or refractory hematologic autoimmune cytopenias

(2007) Aarati Rao et al.   PEDIATRIC BLOOD & CANCER