[PDF] Top 20 Optimal therapy in Gaucher disease
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Optimal therapy in Gaucher disease
... Abstract: Gaucher disease (GD), the inherited deficiency of the lysosomal enzyme glucocer- ebrosidase, presents with a wide range of symptoms of varying severity, and primarily affects the skeletal, ... See full document
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Gaucher’s Disease and Fatal Hepatic Fibrosis Despite Prolonged Enzyme Replacement Therapy
... ABSTRACT. Cefuroxime axetil has been shown to have efficacy comparable to doxycycline in adults with early Lyme disease (LD). Because of toxicity, doxycycline is usually avoided in children. For children who are ... See full document
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Biochemical response to substrate reduction therapy versus enzyme replacement therapy in Gaucher disease type 1 patients
... Substrate reduction therapy (SRT), aiming to reduce accumulating glycosphingolipids by inhibiting their synthesis, might circumvent these disadvantages of ERT. Inhibitors of GlcCer synthesis to be used in SRT of ... See full document
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Endosperm-specific expression of human acid beta-glucosidase in a waxy rice
... hGCase: human acid-beta-glucosidase; rhGCase: recombinant human acid- beta-glucosidase; GD: gaucher disease; ERT: enzyme replacement therapy; SPS: sucrose phosphate synthase; PSVs: prote[r] ... See full document
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Switching from imiglucerase to miglustat for the treatment of French patients with Gaucher disease type 1: a case series
... In late 2008 she agreed to commence ERT with imiglu- cerase 30IU/kg every 2 weeks due to worsening fatigue and the appearance of bone pain (without osteonecrosis). Thrombocytopenia was corrected within 6 months, and her ... See full document
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Gaucher’s Disease and Fatal Hepatic Fibrosis Despite Prolonged Enzyme Replacement Therapy
... clinical data suggesting the best strategy in case of hepatic fibrosis. A bone marrow transplant may de- finitively cure type 1 GD; owing to the risk, which is probably still higher in patients with hepatic ... See full document
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Rethinking fatigue in Gaucher disease
... other disease-related events, includ- ing elevated plasma cytokine levels [43], hypermetabolism [44], or myopathy ...of optimal treatment modalities for addressing fatigue in patients with GD remains to be ... See full document
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Chronic pain in Gaucher disease: skeletal or neuropathic origin?
... replacement therapy; GD: Gaucher disease; HPT: Hot pain threshold; IENF: Intra-epidermal nerve fiber; MCV: Motor nerve conduction velocity; MDT: Mechanical detection threshold; NCS: Nerve conduction ... See full document
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Imiglucerase in the treatment of Gaucher disease: a history and perspective
... to therapy and crossover trials in patients already on established treatments, it can still be argued that the simple open-label design of the original alglucerase trial was sufficient, given the striking and ... See full document
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Modeling changes in biomarkers in Gaucher disease patients receiving enzyme replacement therapy using a pathophysiological model
... Grabowski et al. [23] developed an Emax model to de- scribe changes in hemoglobin and platelets and in splenic and hepatic volume during ERT of patients in the Inter- national Collaborative Gaucher Group Registry. ... See full document
11
Gaucher disease with jawbone involvement: a case report
... for Gaucher disease, left hip replacement, gall bladder re- moval, herniorrhaphy, spleen abscess drainage, hepatitis C infection, and ...with Gaucher disease (70mg weekly for 2 years), calcium ... See full document
5
Gaucher disease: clinical profile and therapeutic developments
... for Gaucher disease, the manufacturer reported spending less than $58M for ...gene therapy has been slow, even for monogenic diseases in which it clearly offers the potential for a definitive and ... See full document
15
Lysosomal Storage Disorders in the Newborn
... substrate-reduction therapy, which aims to partially inhibit the biosynthetic cycle to reduce substrate influx in the compromised ...with Gaucher type 1 in Europe, the United States, and Israel. In ... See full document
19
Imiglucerase in the management of Gaucher disease type 1: an evidence-based review of its place in therapy
... density at baseline and for up to 10 years on imiglucerase were analyzed in patients with GD1, and four groups were determined: children, adolescents, young adults, and older adults. Pretreatment, low BMD was prevalent ... See full document
11
Dental profile of patients with Gaucher disease
... As Gaucher disease results in anemia, tendency to bleed- ing, and poor healing, a correlation between Gaucher dis- ease and gingival disease was anticipated, but no such association was ... See full document
5
Long-term follow-up and sudden unexpected death in Gaucher disease type 3 in Egypt
... of Gaucher disease type 3 in Egypt pre- sented here should be useful for the development and assessment of primary therapy for this disease and for identifying genetic ...other ... See full document
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Enzyme Replacement Therapy for Gaucher Disease: Skeletal Responses to Macrophage-targeted Glucocerebrosidase
... Pediatrics 1995;96:629- 637; Gaucher disease, enzyme replacement therapy, mac- rophage-targeted glucocerebrosidase, bone marrow, com- bined cortical thickness, trabecular bone density.. [r] ... See full document
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Diagnosis features of pediatric Gaucher disease patients in the era of enzymatic therapy, a national-base study from the Spanish Registry of Gaucher Disease
... of Gaucher Disease (SpRGD) coordinated by the “Funda- cion Española para el Estudio y Terapeutica de la Enfermedad de Gaucher”, (FEETEG), a total of 386 GD patients have been reported in Spain ...on ... See full document
9
Cost-effectiveness of enzyme replacement therapy for type 1 Gaucher disease
... same disease states, we put most emphasis on assessing the impact of parameter uncertainty resulting from the transition probabilities from one disease state to the ... See full document
12
Successful switch from enzyme replacement therapy to miglustat in an adult patient with type 1 Gaucher disease: a case report
... antihistamine therapy. However, he later agreed to switch to oral therapy with miglustat (Zavesca®, Actelion Pharmaceuticals), which was started at a dosage of 100 mg twice daily and ti- trated up to 100 mg ... See full document
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