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Chronic Recurrent Multifocal Osteomyelitis Involving the Spine, Sternum, and Lower Extremities: A Case Report

Pediatrics (Evanston), 2024-10, Vol.154 (4), p.1

2024

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Case Report: Hypocomplementemic urticarial vasculitis syndrome in a pediatric patient with complement factor 1 deficiency

Frontiers in pediatrics, 2024-09, Vol.12, p.1448094

2024

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Thrombotic microangiopathy following systemic AAV administration is dependent on anti-capsid antibodies

The Journal of clinical investigation, 2024-01, Vol.134 (1), p.1-11

2024

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The first case of monozygotic twin boys with nearly identical features of Sjögren’s syndrome

Rheumatology (Oxford, England), 2020-03, Vol.59 (3), p.687-688

2020

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Bortezomib is efficacious in the treatment of severe childhood-onset neuropsychiatric systemic lupus erythematosus with psychosis: a case series and mini-review of B-cell immunomodulation in antibody-mediated diseases

Clinical rheumatology, 2023-07, Vol.42 (7), p.1965-1979

2023

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

The Journal of pediatrics, 2013-09, Vol.163 (3), p.847-854.e1

2013

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Heterozygous FOXN1 Variants Cause Low TRECs and Severe T Cell Lymphopenia, Revealing a Crucial Role of FOXN1 in Supporting Early Thymopoiesis

American journal of human genetics, 2019-09, Vol.105 (3), p.549-561

2019

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Rituximab as Adjunct Maintenance Therapy for Refractory Juvenile Myasthenia Gravis

Pediatric neurology, 2020-10, Vol.111, p.40-43

2020

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The Diagnostic Performance of Early Sjögren's Syndrome Autoantibodies in Juvenile Sjögren's Syndrome: The University of Florida Pediatric Cohort Study

Frontiers in immunology, 2021-06, Vol.12, p.704193-704193

2021

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Randomized, Double‐Blind, Placebo‐Controlled Trial of the Efficacy and Safety of Rilonacept in the Treatment of Systemic Juvenile Idiopathic Arthritis

Arthritis & rheumatology (Hoboken, N.J.), 2014-09, Vol.66 (9), p.2570-2579

2014

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Early Outcomes in Children With Antineutrophil Cytoplasmic Antibody–Associated Vasculitis

Arthritis & rheumatology (Hoboken, N.J.), 2017-07, Vol.69 (7), p.1470-1479

2017

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Adeno-associated virus-mediated gene therapy in a patient with Canavan disease using dual routes of administration and immune modulation

Molecular therapy. Methods & clinical development, 2023-09, Vol.30, p.303-314

2023

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A Novel X-Linked Disorder of Immune Deficiency and Hypohidrotic Ectodermal Dysplasia Is Allelic to Incontinentia Pigmenti and Due to Mutations in IKK-gamma ( NEMO)

American journal of human genetics, 2000-12, Vol.67 (6), p.1555-1562

2000

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Case Report: Refractory macrophage activation syndrome requiring high-dose anakinra, emapalumab, and etoposide therapy in early-onset systemic juvenile idiopathic arthritis associated with adenoviremia

Frontiers in pediatrics, 2024-01, Vol.11, p.1336554

2024

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Pompe disease gene therapy: Gene Therapy

Human molecular genetics, 2011-04, Vol.20 (1), p.R61-R68

2011

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BK viruria and viremia in children with systemic lupus erythematosus

Pediatric rheumatology online journal, 2017-04, Vol.15 (1), p.21-21, Article 21

2017

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Predictive utility of ANCA positivity and antigen specificity in the assessment of kidney disease in paediatric-onset small vessel vasculitis

Rheumatic & musculoskeletal diseases open, 2024-06, Vol.10 (2), p.e004315

2024

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End-stage renal disease secondary to anti-glomerular basement membrane disease in a child with common variable immunodeficiency

Clinical nephrology. Case studies, 2019-02, Vol.7 (1), p.1-6

2019

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Persistence of Vaccine-Derived Polioviruses among Immunodeficient Persons with Vaccine-Associated Paralytic Poliomyelitis

The Journal of infectious diseases, 2003-12, Vol.188 (12), p.1845-1852

2003

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Clonality analysis after retroviral-mediated gene transfer to CD34 + cells from the cord blood of ADA-deficient SCID neonates

Nature medicine, 2003-04, Vol.9 (4), p.463-468

2003

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The family journey-to-diagnosis with systemic juvenile idiopathic arthritis: a cross-sectional study of the changing social media presence

Open access rheumatology: research and reviews, 2016-01, Vol.8 (Issue 1), p.61-71

2016

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Acute-onset opioid-induced hyperalgesia in a child with juvenile idiopathic arthritis

Journal of clinical rheumatology, 2012-10, Vol.18 (7), p.349-351

2012

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Human Severe Combined Immunodeficiency Due to a Defect in ZAP-70, a T Cell Tyrosine Kinase

Science (American Association for the Advancement of Science), 1994-06, Vol.264 (5165), p.1596-1599

1994

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Secondary hemophagocytic lymphohistiocytosis following Zolgensma therapy: An evolving story on the innate response to systemic gene therapy

Molecular therapy, 2022-12, Vol.30 (12), p.3503-3504

2022

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A Novel Manifestation of Prolidase Deficiency in a Toddler Diagnosed With Very‐early‐onset Crohn Disease

Journal of pediatric gastroenterology and nutrition, 2019-09, Vol.69 (3), p.e89-e90

2019

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Chronic Recurrent Multifocal Osteomyelitis Involving the Spine, Sternum, and Lower Extremities: A Case Report

Case Report: Hypocomplementemic urticarial vasculitis syndrome in a pediatric patient with complement factor 1 deficiency

Thrombotic microangiopathy following systemic AAV administration is dependent on anti-capsid antibodies

The first case of monozygotic twin boys with nearly identical features of Sjögren’s syndrome

Bortezomib is efficacious in the treatment of severe childhood-onset neuropsychiatric systemic lupus erythematosus with psychosis: a case series and mini-review of B-cell immunomodulation in antibody-mediated diseases

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

Heterozygous FOXN1 Variants Cause Low TRECs and Severe T Cell Lymphopenia, Revealing a Crucial Role of FOXN1 in Supporting Early Thymopoiesis

Rituximab as Adjunct Maintenance Therapy for Refractory Juvenile Myasthenia Gravis

The Diagnostic Performance of Early Sjögren's Syndrome Autoantibodies in Juvenile Sjögren's Syndrome: The University of Florida Pediatric Cohort Study

Randomized, Double‐Blind, Placebo‐Controlled Trial of the Efficacy and Safety of Rilonacept in the Treatment of Systemic Juvenile Idiopathic Arthritis

Early Outcomes in Children With Antineutrophil Cytoplasmic Antibody–Associated Vasculitis

Adeno-associated virus-mediated gene therapy in a patient with Canavan disease using dual routes of administration and immune modulation

A Novel X-Linked Disorder of Immune Deficiency and Hypohidrotic Ectodermal Dysplasia Is Allelic to Incontinentia Pigmenti and Due to Mutations in IKK-gamma ( NEMO)

Case Report: Refractory macrophage activation syndrome requiring high-dose anakinra, emapalumab, and etoposide therapy in early-onset systemic juvenile idiopathic arthritis associated with adenoviremia

Pompe disease gene therapy: Gene Therapy

BK viruria and viremia in children with systemic lupus erythematosus

Predictive utility of ANCA positivity and antigen specificity in the assessment of kidney disease in paediatric-onset small vessel vasculitis

End-stage renal disease secondary to anti-glomerular basement membrane disease in a child with common variable immunodeficiency

Persistence of Vaccine-Derived Polioviruses among Immunodeficient Persons with Vaccine-Associated Paralytic Poliomyelitis

Clonality analysis after retroviral-mediated gene transfer to CD34 + cells from the cord blood of ADA-deficient SCID neonates

The family journey-to-diagnosis with systemic juvenile idiopathic arthritis: a cross-sectional study of the changing social media presence

Acute-onset opioid-induced hyperalgesia in a child with juvenile idiopathic arthritis

Human Severe Combined Immunodeficiency Due to a Defect in ZAP-70, a T Cell Tyrosine Kinase

Secondary hemophagocytic lymphohistiocytosis following Zolgensma therapy: An evolving story on the innate response to systemic gene therapy

A Novel Manifestation of Prolidase Deficiency in a Toddler Diagnosed With Very‐early‐onset Crohn Disease

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