Z. Kazi

发表

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

Yow-Ming C Wang, Anne R. Pariser, J. Bluestone, 2016, Molecular Genetics and Metabolism.

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

S. Packman, O. Abdul-Rahman, P. Tanpaiboon, 2018, Genetics in Medicine.

Insight into the phenotype of infants with Pompe disease identified by newborn screening with the common c.-32-13T>G "late-onset" GAA variant.

P. Kishnani, L. Case, J. Coats, 2017, Molecular genetics and metabolism.

High dose IVIG successfully reduces high rhGAA IgG antibody titers in a CRIM-negative infantile Pompe disease patient.

A. Rosenberg, P. Kishnani, Ankit K. Desai, 2017, Molecular genetics and metabolism.

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

D. Stockton, D. Viskochil, A. Rosenberg, 2016, JCI insight.

Clinical and Molecular Disease Spectrum and Outcomes in Patients with Infantile-Onset Pompe Disease.

M. Kabra, C. Rehder, S. Nampoothiri, 2019, The Journal of pediatrics.

Characterization of immune response in Cross-Reactive Immunological Material (CRIM)-positive infantile Pompe disease patients treated with enzyme replacement therapy

D. Bali, P. Kishnani, Ankit K. Desai, 2019, Molecular genetics and metabolism reports.

Enzyme replacement therapy with alglucosidase alfa in Pompe disease: Clinical experience with rate escalation.

P. Kishnani, Heidi Cope, Stephanie M Dearmey, 2017, Molecular genetics and metabolism.

Neuroimaging findings in infantile Pompe patients treated with enzyme replacement therapy.

D. Frush, L. Hobson‐Webb, P. Kishnani, 2016, Molecular genetics and metabolism.

Response to de Vries et al.

A. Rosenberg, S. Richards, P. Kishnani, 2017, Genetics in Medicine.

Immune tolerance strategies in siblings with infantile Pompe disease — Advantages for a preemptive approach to high-sustained antibody titers

M. Gambello, P. Kishnani, E. Lisi, 2015, Molecular genetics and metabolism reports.

Longitudinal polysomnographic findings in infantile Pompe disease

Sujay M Kansagra, R. Kravitz, P. Kishnani, 2015, American journal of medical genetics. Part A.

Sensitivity of whole exome sequencing in detecting infantile- and late-onset Pompe disease.

E. Cirulli, C. Rehder, D. Bali, 2017, Molecular genetics and metabolism.

Clinical laboratory experience of blood CRIM testing in infantile Pompe disease

C. Rehder, D. Bali, P. Kishnani, 2015, Molecular genetics and metabolism reports.

The emerging phenotype of late-onset Pompe disease: A systematic literature review.

L. Hobson‐Webb, P. Kishnani, L. Case, 2017, Molecular genetics and metabolism.

Sustained immune tolerance induction in enzyme replacement therapy-treated CRIM-negative patients with infantile Pompe disease.

O. Abdul-Rahman, P. Tanpaiboon, A. Rosenberg, 2017, JCI insight.

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

C. Rehder, N. Karabul, D. Bali, 2015, Genetics in Medicine.

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

C. Rehder, N. Karabul, D. Bali, 2015, Genetics in Medicine.

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

A. K. Desai, A. D. De Groot, P. Kishnani, 2019, Clinical immunology.

Successful combined liver/kidney transplantation from a donor with Pompe disease.

K. Mekeel, M. Valasek, R. Loomba, 2015, Molecular genetics and metabolism.

Longitudinal polysomnographic findings in infantile Pompe disease

R. Kravitz, P. Kishnani, S. Austin, 2015, American journal of medical genetics. Part A.

The emerging phenotype of late-onset Pompe disease: A systematic literature review.

L. Hobson‐Webb, P. Kishnani, L. Case, 2017, Molecular genetics and metabolism.

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

D. Stockton, D. Viskochil, A. Rosenberg, 2016, JCI insight.

Cross-reactive immunologic material positive infantile Ρompe disease: Characterization of immune responses in patient treated with enzyme replacement therapy

P. Kishnani, Ankit K. Desai, Z. Kazi, 2016 .