Search Results - "van den Broek, Brigitte T.A."
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Early and late outcomes after cord blood transplantation for pediatric patients with inherited leukodystrophies
Published in Blood advances (09-01-2018)“…Leukodystrophies (LD) are devastating inherited disorders leading to rapid neurological deterioration and premature death. Hematopoietic stem cell…”
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Quantifying lymphocyte vacuolization serves as a measure of CLN3 disease severity
Published in JIMD reports (01-07-2020)“…Background The CLN3 disease spectrum ranges from a childhood‐onset neurodegenerative disorder to a retina‐only disease. Given the lack of metabolic disease…”
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Quality of life of Hurler syndrome patients after successful hematopoietic stem cell transplantation
Published in Blood advances (14-11-2017)“…Hurler syndrome (HS) is a lysosomal storage disease characterized by multisystem morbidity and death in early childhood. Hematopoietic stem cell…”
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Emotional consequences of intensive care unit delirium and delusional memories after intensive care unit admission: A systematic review
Published in Journal of critical care (01-04-2012)“…Abstract Purpose The aim of this study was to review literature exploring the emotional consequences of delirium and delusional memories in intensive care unit…”
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Hearing loss in patients with mucopolysaccharidoses‐1 and ‐6 after hematopoietic cell transplantation: A longitudinal analysis
Published in Journal of inherited metabolic disease (01-11-2020)“…Hearing loss is frequently seen in mucopolysaccharidoses (MPS) patients. Although hematopoietic cell transplantation (HCT) increases overall survival, disease…”
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Serum Biomarker Profile Including CCL1, CXCL10, VEGF, and Adenosine Deaminase Activity Distinguishes Active From Remotely Acquired Latent Tuberculosis
Published in Frontiers in immunology (07-10-2021)“…There is an urgent medical need to differentiate active tuberculosis (ATB) from latent tuberculosis infection (LTBI) and prevent undertreatment and…”
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Long-Term Effect of Hematopoietic Cell Transplantation on Systemic Inflammation in Mucopolysaccharidoses Patients
Published in Transplantation and cellular therapy (01-03-2021)Get full text
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A Case of Unexpected Adult-Onset Neurologic Decline in CLN3-Associated Retinal Degeneration
Published in JAMA ophthalmology (01-12-2017)Get more information
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Hurdles in treating Hurler disease: potential routes to achieve a “real” cure
Published in Blood advances (23-06-2020)“…Mucopolysaccharidoses (MPSs) are multiorgan devastating diseases for which hematopoietic cell transplantation (HCT) and, to a lesser extent, enzyme replacement…”
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Long-term effect of hematopoietic cell transplantation on systemic inflammation in patients with mucopolysaccharidoses
Published in Blood advances (24-08-2021)“…Mucopolysaccharidoses (MPS) are devastating inherited diseases treated with hematopoietic cell transplantation (HCT). However, disease progression, especially…”
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QuantiFERON-plus does not discriminate between active and latent tuberculosis
Published in Infectious diseases (London, England) (03-06-2018)Get full text
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Longitudinal Analysis of the Effect of Hematopoietic Cell Transplantation on Ocular Disease in Children with Mucopolysaccharidosis I Shows Ongoing Disease Progression
Published in Biology of blood and marrow transplantation (01-03-2019)“…Corneal clouding is seen in nearly all patients with Mucopolysaccharidosis-1 (MPS-1) causing visual impairment. Hematopoietic cell transplantation (HCT) is…”
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Longitudinal Analysis of Ocular Disease in Children with Mucopolysaccharidosis I after Hematopoietic Cell Transplantation
Published in Biology of blood and marrow transplantation (01-05-2020)“…•After initial stabilization, corneal clouding is progressing in patients with mucopolysaccharidosis type 1 (MPS-1) after treatment with hematopoietic cell…”
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Salivary α-Iduronidase Activity as a Potential New Biomarker for the Diagnosis and Monitoring the Effect of Therapy in Mucopolysaccharidosis Type I
Published in Biology of blood and marrow transplantation (01-09-2018)“…•Salivary IDUA activity is a powerful method to detect IDUA deficiency.•Salivary IDUA activity might be able to differentiate between phenotypes.•Salivary IDUA…”
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13 - Quality of Life of Hurler Syndrome Patients after Successful Hematopoietic Cell Transplantation
Published in Biology of blood and marrow transplantation (01-03-2018)Get full text
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Quality of Life of Hurler Syndrome Patients after Successful Hematopoietic Cell Transplantation
Published in Biology of blood and marrow transplantation (01-03-2018)Get full text
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