The efficacy of enzyme replacement therapy in patients with chronic neuronopathic Gaucher’s disease-Reference-Cited by-同舟云学术

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The efficacy of enzyme replacement therapy in patients with chronic neuronopathic Gaucher’s disease

Published:2001-04 Issue:4 Volume:138 Page:539-547
ISSN:0022-3476
Container-title:The Journal of Pediatrics
language:en
Short-container-title:The Journal of Pediatrics

Author:

Altarescu Gheona,Hill Suvimol,Wiggs Edyth,Jeffries Neal,Kreps Constance,Parker Colette C.,Brady Roscoe O.,Barton Norman W.,Schiffmann Raphael

Publisher

Elsevier BV

Subject

Pediatrics, Perinatology and Child Health

Reference37 articles.

1. Demonstration of a deficiency of glucocerebroside-cleaving enzyme in Gaucher’s disease;Brady;J Clin Invest,1966

2. Gaucher disease;Beutler,1995

3. Gaucher’s disease: clinical features and natural history;Cox,1997

4. Prospective study of neurological responses to treatment with macrophage-targeted glucocerebrosidase in patients with type 3 Gaucher’s disease;Schiffmann;Ann Neurol,1997

5. Isolated horizontal supranuclear gaze palsy as a marker of severe systemic involvement in Gaucher’s disease;Patterson;Neurology,1993

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1. Electroencephalogram and phenotype patterns in neuronopathic Gaucher disease patients – ten years of experience in a single center;Acta Epileptologica;2024-09-01

2. Long-Term Treatment of Gaucher Disease with Velaglucerase Alfa in ERT-Naïve Patients from the Gaucher Outcome Survey (GOS) Registry;Journal of Clinical Medicine;2024-05-09

3. Development of quantitative high-throughput screening assays to identify, validate, and optimize small-molecule stabilizers of misfolded β-glucocerebrosidase with therapeutic potential for Gaucher disease and Parkinson’s disease;2024-03-27

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5. Venglustat combined with imiglucerase for neurological disease in adults with Gaucher disease type 3: the LEAP trial;Brain;2022-10-18

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