Combined miglustat and enzyme replacement therapy in two patients with type 1 Gaucher disease: two case reports.

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Combined miglustat and enzyme replacement therapy in two patients with type 1 Gaucher disease: two case reports.

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2018 nî lūn-bûn

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2018年の論文

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2018年学术文章

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2018年学术文章

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Combined miglustat and enzyme ...... her disease: two case reports.

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Combined miglustat and enzyme ...... her disease: two case reports.

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Combined miglustat and enzyme ...... her disease: two case reports.

@en

Combined miglustat and enzyme ...... her disease: two case reports.

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Combined miglustat and enzyme ...... her disease: two case reports.

@en

Combined miglustat and enzyme ...... her disease: two case reports.

@nl

P1476

Combined miglustat and enzyme ...... her disease: two case reports.

@en

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Dominick Amato

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Mary Anne Patterson

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P2860

The Gaucher registry: demographics and disease characteristics of 1698 patients with Gaucher disease.

Treatment of profound thrombocytopenia in a patient with Gaucher disease type 1: Is there a role for substrate reduction therapy.

The frequency of lysosomal storage diseases in The Netherlands.

Prevalence of lysosomal storage disorders.

Oral maintenance clinical trial with miglustat for type I Gaucher disease: switch from or combination with intravenous enzyme replacement.

Potential efficacy of enzyme replacement and substrate reduction therapy in three siblings with Gaucher disease type III.

Identification and use of biomarkers in Gaucher disease and other lysosomal storage diseases.

Recent clinical progress in Gaucher disease.

Evaluation of miglustat as maintenance therapy after enzyme therapy in adults with stable type 1 Gaucher disease: a prospective, open-label non-inferiority study

Combination therapy in a patient with chronic neuronopathic Gaucher disease: a case report

P2888

s13256-017-1541-7

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scholarly article

case report

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10.1186/S13256-017-1541-7

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P407

English

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P478

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P577

2018-01-27T00:00:00Z

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P6179

1100670412

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P698

29373994

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P921

Enzyme replacement therapy

P932

5787317

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