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

Suhrad G. Banugaria, Sean N. Prater, Yiu-Ki Ng, +6

Genetics in Medicine

Infantile Pompe DiseaseAutoimmune DiseaseAllergyImmunologySerologic Testing +9

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

Sean N. Prater, Suhrad G. Banugaria, Stephanie DeArmey, +8

Genetics in Medicine

Infantile Pompe DiseasePathogenesisGenetic EpidemiologyPediatricsPublic Health +1

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

Suhrad G. Banugaria, Sean N. Prater, Trusha Patel, +12

PLoS ONE

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

Suhrad G. Banugaria, Sean N. Prater, Judeth K. McGann, +9

Genetics in Medicine

Pompe DiseaseAutoimmune DiseaseDisease MechanismImmunodeficienciesImmunology +11

Skeletal muscle pathology of infantile Pompe disease during long-term enzyme replacement therapy

Sean N. Prater, Trusha Patel, Anne F. Buckley, +6

Orphanet Journal of Rare Diseases

The value of muscle biopsies in Pompe disease: identifying lipofuscin inclusions in juvenile- and adult-onset patients

Erin Feeney, Stephanie Austin, Yin‐Hsiu Chien, +7

Acta Neuropathologica Communications

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

Kathryn L. Berrier, Zoheb B. Kazi, Sean N. Prater, +11

Genetics in Medicine

Clinical ImmunologyMaternal ImmunizationAutoimmune DiseaseMedicineImmunodeficiencies +8

Neuroimaging findings in infantile Pompe patients treated with enzyme replacement therapy

Paul McIntosh, Lisa D. Hobson‐Webb, Zoheb B. Kazi, +7

Molecular Genetics and Metabolism

Infantile Pompe PatientsEnzyme Replacement TherapyNeuroanatomyNeurological DisorderClinical Neurology +5

Structures of the Compact Helical Core Domains of Feline Calicivirus and Murine Norovirus VPg Proteins

Eoin Leen, K. Y. Rex Kwok, James R. Birtley, +15

Journal of Virology

Durable and sustained immune tolerance to ERT in Pompe disease with entrenched immune responses

Zoheb B. Kazi, Sean N. Prater, Joyce A. Kobori, +7

JCI Insight