ライフサイエンスコーパス: Gaucher's disease

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1 who had received ERT for 3 years or more for Gaucher's disease.

2 nt of the lysosomal storage disease known as Gaucher's disease.

3 as recessive X-linked ichthyosis and type 2 Gaucher's disease.

4 its safety and feasibility for neuronopathic Gaucher's disease.

5 for patients with type 1 (non-neuronopathic) Gaucher's disease.

6 s key clinical features of non-neuronopathic Gaucher's disease.

7 a novel oral treatment for non-neuronopathic Gaucher's disease.

8 5 patients (age, 3.5-8.5 years) with type 3 Gaucher's disease.

9 asis for use of this therapy in treatment of Gaucher's disease.

10 The primary defect in Gaucher's disease, a lysosomal disorder affecting macrop

11 totriosidase activity (a sensitive marker of Gaucher's disease activity).

12 rosidase (GBA), a deficiency of which causes Gaucher's disease, among patients with Parkinson's disea

13 Parkinsonism is an established feature of Gaucher's disease and an increased frequency of mutation

14 T reverses systemic manifestations of type 3 Gaucher's disease and appears to reduce the burden of Ga

15 t the clonal immunoglobulin in patients with Gaucher's disease and in mouse models of Gaucher's disea

16 m was noted, albeit rarely, in patients with Gaucher's disease and more frequently in relatives who w

17 Patients with neuronopathic Gaucher's disease and similar central nervous system dis

18 respectively) in eight patients with type 1 Gaucher's disease, and in one healthy individual.

19 es the lysosomal enzyme that is deficient in Gaucher's disease, are important and common risk factors

20 e activation by lysolipids may underlie both Gaucher's disease-associated gammopathies and some spora

21 Substrate reduction ameliorates Gaucher's disease-associated gammopathy in mice.

22 ith Gaucher's disease and in mouse models of Gaucher's disease-associated gammopathy is reactive agai

23 Gaucher's disease continues to be a model for applicatio

24 Mutations in GC result in Gaucher's disease (GD) and are the major genetic risk fa

25 cerebrosidase gene (GBA) causing neuropathic Gaucher's disease (GD) in homozygotes lead to aggressive

26 Gaucher's disease (GD) is characterized by loss of lysos

27 Gaucher's disease (GD), an inherited metabolic disorder

28 sponsible for the lysosomal storage disorder Gaucher's disease (GD), are the strongest genetic risk f

29 tations cause the lysosomal storage disorder Gaucher's disease (GD).

30 Systemic enzyme replacement for Gaucher's disease has not prevented premature death or s

31 Patients with Gaucher's disease have an increased risk of monoclonal g

32 successfully introduced for the treatment of Gaucher's disease in the early 1990s.

33 Current treatment for Gaucher's disease involves administration of intravenous

34 Gaucher's disease is a common genetic disease caused by

35 clerosis, and less familiar diseases such as Gaucher's disease, nephrogenic diabetes insipidus, and C

36 In addition to Gaucher's disease, the effects of ERT in four other majo

37 cerebrosidase gene (GBA) are associated with Gaucher's disease, the most common lysosomal storage dis

38 al and organ volume stability in adults with Gaucher's disease type 1 already controlled by intraveno

39 The mainstay of treatment for Gaucher's disease type 1 is alternate-week infusion of e

40 ts with various cancers and one patient with Gaucher's disease, whether or not the patient cells had

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