Enzyme Replacement Therapy
Improvement of bone mineral density after enzyme replacement therapy in Chinese late onset Pompe disease patients
... Objective: Late-onset Pompe disease (LOPD) is a lysosomal storage disease resulted from deficiency of the enzyme acid α-glucosidase. Patients usually develop a limb-girdle pattern of myopathy and respiratory ...
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Combined miglustat and enzyme replacement therapy in two patients with type 1 Gaucher disease: two case reports
... combined enzyme replacement therapy and miglustat substrate reduction therapy were administered to overcome refractory clinical ...imiglucerase enzyme replacement ...reduction ...
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Pancreatic enzyme replacement therapy following surgery for pancreatic cancer: An exploration of patient self-management
... pancreatic enzyme replacement therapy (PERT) indicate that patients are often under-treated following surgery, with the intricacies of therapy being cited as a ...
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Successful switch from enzyme replacement therapy to miglustat in an adult patient with type 1 Gaucher disease: a case report
... The response of our patient to ERT was initially good, including improvements in Hb and platelets, decreased bone involvement, and reduced chitotriosidase levels that were in line with findings reported in the literature ...
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Murine mucopolysaccharidosis type VII: long term therapeutic effects of enzyme replacement and enzyme replacement followed by bone marrow transplantation
... of enzyme replacement ...term enzyme replacement initiated either at birth or at 6 wk of age, and of enzyme administration initiated at birth followed by syngeneic bone marrow ...
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Immune tolerance improves the efficacy of enzyme replacement therapy in canine mucopolysaccharidosis I
... In vitro experiments showed that anti-iduronidase antibodies inhibited rhIDU uptake into cells in a concentration-dependent fashion. The inhibitory relationship was sigmoidal, with a steep decline in uptake at relatively ...
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Enzyme replacement therapy for murine mucopolysaccharidosis type VII leads to improvements in behavior and auditory function
... that enzyme replacement initiated at birth improved the behavioral performance and reduced hearing loss in MPS VII ...mice. Enzyme-treated MPS VII mice performed similarly to normal mice and ...
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Clinical and Technological Dependence Characteristics on a Series of Brazilian Cases with Infantile Onset Pompe Disease in Enzyme Replacement Therapy
... Dramatic prognostic changes have been documented in IOPD since the ap- proval of enzyme replacement therapy (ERT) treatment with recombinant hu- man GAA enzyme (rhGAA) such as in 2007 [1]. An ...
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Erythrocyte metabolism in purine nucleoside phosphorylase deficiency after enzyme replacement therapy by infusion of erythrocytes
... Purine nucleoside phosphorylase deficiency is associated with a severely defective T-cell immunity. A patient with purine nucleoside phosphorylase deficiency was treated with transfusions of irradiated erythrocytes and ...
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Cerebral Microbleeds Identified by Susceptibility Weighted Imaging in Two Cases of Fabry Disease without Neurological Symptoms
... The patient was a 42-year-old man with acroparesthesias, hypohydrosis, and angiokeratomas on the trunk, arms, and legs since childhood. The diagnosis of FD was confirmed based on clinical findings and decreased α-ga- ...
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Motor Development as a Potential Marker to Monitor Infantile Pompe Disease on Enzyme Replacement Therapy
... After Enzyme Replacement Therapy (ERT) using recombinant human acid alpha-glucosidase (rhGAA), survival of Infantile Pompe Disease (IPD) patients through the first 18 months of age has been ...
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Combination therapy in a patient with chronic neuronopathic Gaucher disease: a case report
... is enzyme replacement therapy with recombinant analogues of the deficient human enzyme ...glucocerebrosidase. Enzyme replacement therapy has been shown to improve ...
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Enzyme replacement therapy in a feline model of Maroteaux Lamy syndrome
... only therapy available for pa- tients has been bone marrow transplantation, and this has been with limited clinical and biochemical evidence of success (5, ...at enzyme replacement therapy ...
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Pro/con debate: Continuous versus intermittent dialysis for acute kidney injury: a never ending story yet approaching the finish?
... renal replacement therapy should be applied in an intermittent or continuous mode to the patient with acute kidney injury has been the topic of several controlled studies and meta- ...renal ...
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Nicotine replacement therapy and smoking cessation: an updated systematic review of randomized control trials
... thatnicotine replacement therapy in the form of nicotine transdermal patch failed to achieve smoking cessation at 6 and 12-month follow-up among adolescent smokers (Scherphof,, ...
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Biochemical and Functional Abnormalities in Lymphocytes from an Adenosine Deaminase deficient Patient during Enzyme Replacement Therapy
... Biochemical and immunological properties of lymphocytes were measured repetitively over a period of 40 mo during enzyme replacement by transfusion in a child with adenosine deaminase (ADA) deficiency and ...
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Enzyme replacement therapy by fibroblast transplantation: long term biochemical study in three cases of Hunter's syndrome
... for replacement therapy in three patients with Hunter's syndrome, over periods ranging from ...in enzyme activity were accompanied by corresponding increases in catabolism of heparan and dermatan ...
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Enzyme replacement therapy for murine mucopolysaccharidosis type VII
... compared with untreated 6-wk-old MPS VII mice. The single injection decreased lysosomal distention in the fixed tissue macrophage system. MPS VII mice that received multiple injections had 27.8, 3.5, and 3.3% of normal ...
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Immune response hinders therapy for lysosomal storage diseases
... anti- enzyme antibodies inhibit enzyme uptake and substantially limit the therapeutic efficacy of ERT in canines with MPS I (see the related article beginning on page ...
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Net ultrafiltration intensity and mortality in critically ill patients with fluid overload
... Our study addresses an important knowledge gap not addressed by prior studies. While numerous studies have documented an association between the severity of FO and incremental risk of death [3, 4], none examined the UF ...
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