CRIM-negative infantile Pompe disease: 42-month treatment outcome.
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Hearing in adults with Pompe diseaseThe emerging phenotype of long-term survivors with infantile Pompe disease.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.Pompe disease gene therapy.Hypoglossal neuropathology and respiratory activity in pompe miceOutcome of patients with classical infantile pompe disease receiving enzyme replacement therapy in Germany.Spinal delivery of AAV vector restores enzyme activity and increases ventilation in Pompe mice.Predicting cross-reactive immunological material (CRIM) status in Pompe disease using GAA mutations: lessons learned from 10 years of clinical laboratory testing experience.Postmortem Findings and Clinical Correlates in Individuals with Infantile-Onset Pompe DiseaseCRIM-negative infantile Pompe disease: characterization of immune responses in patients treated with ERT monotherapyImmune Modulation Therapy in a CRIM-Positive and IgG Antibody-Positive Infant with Pompe Disease Treated with Alglucosidase Alfa: A Case ReportPhase I/II trial of adeno-associated virus-mediated alpha-glucosidase gene therapy to the diaphragm for chronic respiratory failure in Pompe disease: initial safety and ventilatory outcomes.Successful immune tolerance induction to enzyme replacement therapy in CRIM-negative infantile Pompe disease.Atypical immunologic response in a patient with CRIM-negative Pompe disease.B-Cell depletion and immunomodulation before initiation of enzyme replacement therapy blocks the immune response to acid alpha-glucosidase in infantile-onset Pompe disease.Sustained correction of motoneuron histopathology following intramuscular delivery of AAV in pompe mice.Newborn screening for lysosomal storage diseases: an ethical and policy analysis.The genotype-phenotype correlation in Pompe disease.The respiratory neuromuscular system in Pompe disease.Sustained immune tolerance induction in enzyme replacement therapy-treated CRIM-negative patients with infantile Pompe disease.Rapidly Progressive White Matter Involvement in Early Childhood: The Expanding Phenotype of Infantile Onset Pompe?Teaching tolerance: New approaches to enzyme replacement therapy for Pompe diseaseThe role of immune tolerance induction in restoration of the efficacy of ERT in Pompe disease.Long-term neurologic and cardiac correction by intrathecal gene therapy in Pompe disease.Administration of anti-CD3 antibodies modulates the immune response to an infusion of α-glucosidase in miceCRIM-Negative Pompe Disease Patients with Satisfactory Clinical Outcomes on Enzyme Replacement Therapy.Letter to the Editors: Concerning "CRIM-negative Pompe disease patients with satisfactory clinical outcomes on enzyme replacement therapy" by Al Khallaf et al.Enzyme replacement in neuronal storage disorders in the pediatric population.The new era of Pompe disease: advances in the detection, understanding of the phenotypic spectrum, pathophysiology, and management.Neuroimaging findings in infantile Pompe patients treated with enzyme replacement therapy.Cognitive Development in Infantile-Onset Pompe Disease Under Very Early Enzyme Replacement Therapy.Cognitive and academic outcomes in long-term survivors of infantile-onset Pompe disease: A longitudinal follow-up.
P2860
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P2860
CRIM-negative infantile Pompe disease: 42-month treatment outcome.
description
2010 nî lūn-bûn
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2010年の論文
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2010年学术文章
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2010年学术文章
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2010年学术文章
@zh-cn
2010年学术文章
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2010年学术文章
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2010年学术文章
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2010年學術文章
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2010年學術文章
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name
CRIM-negative infantile Pompe disease: 42-month treatment outcome.
@en
CRIM-negative infantile Pompe disease: 42-month treatment outcome.
@nl
type
label
CRIM-negative infantile Pompe disease: 42-month treatment outcome.
@en
CRIM-negative infantile Pompe disease: 42-month treatment outcome.
@nl
prefLabel
CRIM-negative infantile Pompe disease: 42-month treatment outcome.
@en
CRIM-negative infantile Pompe disease: 42-month treatment outcome.
@nl
P2093
P50
P1476
CRIM-negative infantile Pompe disease: 42-month treatment outcome
@en
P2093
Alice Köhli-Wiesner
Andrea Klein
Dorothe Veraguth
Ianina Scheer
P2888
P304
P356
10.1007/S10545-010-9209-0
P577
2010-09-30T00:00:00Z