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

Orphanet journal of rare diseases, 2013-06, Vol.8 (1), p.90-90, Article 90

2013

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Basilar artery aneurysm: A new finding in classic infantile pompe disease

Muscle & nerve, 2013-04, Vol.47 (4), p.613-615

2013

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Common Threads in Pediatric Inflammatory Diseases: Insight Into Personalized Medicine

JAMA pediatrics, 2018-08, Vol.172 (8), p.721

2018

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The impact of antibodies in late-onset Pompe disease: A case series and literature review

Molecular genetics and metabolism, 2012-07, Vol.106 (3), p.301-309

2012

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Non-depleting anti-CD4 monoclonal antibody induces immune tolerance to ERT in a murine model of Pompe disease

Molecular genetics and metabolism reports, 2014-01, Vol.1 (C), p.446-450

2014

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Persistence of high sustained antibodies to enzyme replacement therapy despite extensive immunomodulatory therapy in an infant with Pompe disease: Need for agents to target antibody-secreting plasma cells

Molecular genetics and metabolism, 2012-04, Vol.105 (4), p.677-680

2012

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Immune modulation in Pompe disease treated with enzyme replacement therapy

Expert review of clinical immunology, 2012-08, Vol.8 (6), p.497-499

2012

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To the Editor

Molecular genetics and metabolism, 2012-09, Vol.107 (1-2), p.244

2012

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To the Editor

Molecular genetics and metabolism, 2012-09, Vol.107 (1-2), p.244-244

2012

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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. e67052

PloS one, 2013-06, Vol.8 (6)

2013

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Basilar artery aneurysm: a new finding in classic infantile Pompe disease

Muscle & nerve, 2013, Vol.47 (4), p.613-615

2013

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Adverse pregnancy outcomes in women with diabetes-related microvascular disease and risks of disease progression in pregnancy: A systematic review and meta-analysis

PLoS medicine, 2021-11, Vol.18 (11), p.e1003856-e1003856

2021

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Correlation between PIK3CA mutations in cell-free DNA and everolimus efficacy in HR + , HER2 - advanced breast cancer: results from BOLERO-2

British journal of cancer, 2017-03, Vol.116 (6), p.726-730

2017

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Study on assessment of quality of life and depression in patients of vitiligo

Indian Dermatology Online Journal, 2019-03, Vol.10 (2), p.153-157

2019

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Prevalence of ESR1 Mutations in Cell-Free DNA and Outcomes in Metastatic Breast Cancer: A Secondary Analysis of the BOLERO-2 Clinical Trial

JAMA oncology, 2016-10, Vol.2 (10), p.1310

2016

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C57BL/6 substrain differences in formalin-induced pain-like behavioral responses

Behavioural brain research, 2020-07, Vol.390, p.112698-112698, Article 112698

2020

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EP1362: VEDOLIZUMAB THERAPY FOR VERY EARLY ONSET INFLAMMATORY BOWEL DISEASE

Gastroenterology (New York, N.Y. 1943), 2022-05, Vol.162 (7), p.S-1112-S-1112

2022

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Canakinumab for the treatment of autoinflammatory very early onset- inflammatory bowel disease

Frontiers in immunology, 2022-09, Vol.13, p.972114-972114

2022

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EP1320: PHENOTYPIC VARIABILITY IN TRICHOHEPATOENTERIC SYNDROME AND VERY EARLY ONSET INFLAMMATORY BOWEL DISEASE

Gastroenterology (New York, N.Y. 1943), 2022-05, Vol.162 (7), p.S-1101

2022

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The Unique Disease Course of Children with Very Early onset-Inflammatory Bowel Disease

Inflammatory bowel diseases, 2020-06, Vol.26 (6), p.909-918

2020

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Clinical and laboratory predictors of monogenic very early onset inflammatory bowel disease

Clinical immunology (Orlando, Fla.), 2022-07, Vol.240, p.109047-109047, Article 109047

2022

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784 SINGLE CENTER RETROSPECTIVE ANALYSIS OF HEMATOPOIETIC STEM CELL TRANSPLANT IN PATIENTS WITH MONOGENIC INFLAMMATORY BOWEL DISEASE

Gastroenterology (New York, N.Y. 1943), 2023-05, Vol.164 (6), p.S-171-S-171

2023

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Mucosal Invariant T cells are Diminished in Very Early‐Onset Inflammatory Bowel Disease

Journal of pediatric gastroenterology and nutrition, 2021-10, Vol.73 (4), p.529-536

2021

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Effects of Vaccination on Invasive Pneumococcal Disease in South Africa

The New England journal of medicine, 2014-11, Vol.371 (20), p.1889-1899

2014

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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

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

Basilar artery aneurysm: A new finding in classic infantile pompe disease

Common Threads in Pediatric Inflammatory Diseases: Insight Into Personalized Medicine

The impact of antibodies in late-onset Pompe disease: A case series and literature review

Non-depleting anti-CD4 monoclonal antibody induces immune tolerance to ERT in a murine model of Pompe disease

Persistence of high sustained antibodies to enzyme replacement therapy despite extensive immunomodulatory therapy in an infant with Pompe disease: Need for agents to target antibody-secreting plasma cells

Immune modulation in Pompe disease treated with enzyme replacement therapy

To the Editor

To the Editor

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. e67052

Basilar artery aneurysm: a new finding in classic infantile Pompe disease

Adverse pregnancy outcomes in women with diabetes-related microvascular disease and risks of disease progression in pregnancy: A systematic review and meta-analysis

Correlation between PIK3CA mutations in cell-free DNA and everolimus efficacy in HR + , HER2 - advanced breast cancer: results from BOLERO-2

Study on assessment of quality of life and depression in patients of vitiligo

Prevalence of ESR1 Mutations in Cell-Free DNA and Outcomes in Metastatic Breast Cancer: A Secondary Analysis of the BOLERO-2 Clinical Trial

C57BL/6 substrain differences in formalin-induced pain-like behavioral responses

EP1362: VEDOLIZUMAB THERAPY FOR VERY EARLY ONSET INFLAMMATORY BOWEL DISEASE

Canakinumab for the treatment of autoinflammatory very early onset- inflammatory bowel disease

EP1320: PHENOTYPIC VARIABILITY IN TRICHOHEPATOENTERIC SYNDROME AND VERY EARLY ONSET INFLAMMATORY BOWEL DISEASE

The Unique Disease Course of Children with Very Early onset-Inflammatory Bowel Disease

Clinical and laboratory predictors of monogenic very early onset inflammatory bowel disease

784 SINGLE CENTER RETROSPECTIVE ANALYSIS OF HEMATOPOIETIC STEM CELL TRANSPLANT IN PATIENTS WITH MONOGENIC INFLAMMATORY BOWEL DISEASE

Mucosal Invariant T cells are Diminished in Very Early‐Onset Inflammatory Bowel Disease

Effects of Vaccination on Invasive Pneumococcal Disease in South Africa

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