Abstract:
INTRODUCTION:Gaucher disease (GD) is an autosomal recessive disorder resulting from the deficiency of the lysosomal enzyme glucocerebrosidase (b-glucosidase), associated with varying degrees of visceral, bone and central nervous system pathology, leading to wide phenotypic diversity. Response to therapy and clinical outcomes are very different between the three clinical subtypes - non-neuronopathic, acute neuronopathic, and chronic neuronopathic forms; hence a definitive clinical diagnosis is essential. The availability of two therapeutic options, i.e. enzyme replacement and substrate reduction, has transformed the natural course of the disease. As pre-treatment disease severity clearly impacts results of therapy, early diagnosis and initiation of treatment especially in the pediatric population are keys to achieving an optimal outcome. Areas covered: We reviewed the literature concerning the treatment of GD focusing on pediatric presentations, various pharmacological treatment options and recommendations for management goals. A PubMed literature search was performed for relevant publications between 1991 and September 2018. Expert commentary: The approval of enzyme replacement therapy (ERT) for GD in the pediatric age group has significantly altered the course of the disease, especially for non-neuronopathic and chronic neuronopathic forms, as ERT does not cross the blood-brain barrier. Early diagnosis, regular follow-up and early initiation of treatment can thus prevent some irreversible complications and improve patient quality of life.
journal_name
Expert Rev Clin Pharmacoljournal_title
Expert review of clinical pharmacologyauthors
Gupta P,Pastores Gdoi
10.1080/17512433.2018.1549486subject
Has Abstractpub_date
2018-12-01 00:00:00pages
1183-1194issue
12eissn
1751-2433issn
1751-2441journal_volume
11pub_type
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journal_title:Expert review of clinical pharmacology
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journal_title:Expert review of clinical pharmacology
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journal_title:Expert review of clinical pharmacology
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journal_title:Expert review of clinical pharmacology
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journal_title:Expert review of clinical pharmacology
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journal_title:Expert review of clinical pharmacology
pub_type: 杂志文章,评审
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journal_title:Expert review of clinical pharmacology
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journal_title:Expert review of clinical pharmacology
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journal_title:Expert review of clinical pharmacology
pub_type: 杂志文章
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journal_title:Expert review of clinical pharmacology
pub_type: 杂志文章,评审
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更新日期:2017-07-01 00:00:00
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journal_title:Expert review of clinical pharmacology
pub_type: 社论
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journal_title:Expert review of clinical pharmacology
pub_type: 杂志文章,评审
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journal_title:Expert review of clinical pharmacology
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pub_type: 杂志文章,评审
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