enzyme-replacement therapy
An open-label clinical trial of agalsidase alfa enzyme replacement therapy in children with Fabry disease who are naïve to enzyme replacement therapy
... Background: Following a drug manufacturing process change, safety/efficacy of agalsidase alfa were evaluated in enzyme replacement therapy (ERT)-naïve children with Fabry disease. Methods: In an ...
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Pregnancy Outcomes in Late Onset Pompe Disease and Enzyme Replacement Therapy
... The course of Pompe disease has been significantly altered with the advent of enzyme replacement therapy (ERT). The mannose targeted recombinant human alpha glucosidase improves cardiac and skeletal ...
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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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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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Enzyme replacement therapy and white matter hyperintensity progression in Fabry disease
... Enzyme replacement therapy (ERT) was, until the recent approval of migalastat (Galafold; Amicus Therapeutics, Cranbury, NJ) in the European Union, 16 the only approved disease-modifying ...
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Cost-effectiveness of enzyme replacement therapy for type 1 Gaucher disease
... this enzyme therapy and cost-effectiveness are not different compared to other orphan diseases, such as Fabry and Pompe disease ...term enzyme replacement therapy for Gaucher disease ...
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Enzyme-Replacement Therapy in a 5-Month-Old Boy With Attenuated Presymptomatic MPS I: 5-Year Follow-up
... of enzyme-replacement therapy in MPS I have shown clinical benefit in patients with considerable preexisting disease, but no data exist on the effect of beginning enzyme replacement ...
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Successful switch from enzyme replacement therapy to miglustat in an adult patient with type 1 Gaucher disease: a case report
... intravenous enzyme replacement ...reduction therapy with miglustat 100 mg twice daily titrated up to 100 mg three times daily over a short ...
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A 24-Year Enzyme Replacement Therapy in an Adenosine-deaminase-Deficient Patient
... (ADA) enzyme deficiency, which leads to the accumulation of toxic metabolites that impair lymphocyte development and ...(PEG-ADA) enzyme replacement therapy, many ADA-deficient children with ...
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Enzyme replacement therapy for pancreatic insufficiency: present and future
... starting enzyme replacement therapy are progressive weight loss and steatorrhea, defined as at least 7 to 15 g of fecal fat per day, but there are no substantial data to support these ...10% ...
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Enzyme replacement therapy for Fabry disease: some answers but more questions
... by enzyme deficiency of α-galactosidase ...of enzyme replacement therapy (ERT) has been a great advancement in the treatment of patients with FD and can stabilize renal function and cardiac ...
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Teaching tolerance New approaches to enzyme replacement therapy for Pompe disease
... with enzyme-replacement therapy ...the therapy, recombinant human lysosomal acid α glucosidase (GAA, rhGAA), ERT unfor- tunately leads to the development of high titers of anti-rhGAA antibody, ...
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Impact of enzyme replacement therapy and hematopoietic stem cell transplantation in patients with Morquio A syndrome
... by enzyme replacement therapy (ERT) showed a sharp reduction on urinary keratan sulfate, but only modest improvement based on a 6-minute walk test and no significant improvement on a 3-minute ...
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Enzyme replacement therapy: efficacy and limitations
... Enzyme replacement therapy (ERT) is available for mucopolysaccharidosis (MPS) I, MPS II, MPS VI, and MPS IVA. The efficacy of ERT has been evaluated in clinical trials and in many post-marketing ...
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Clinical and Technological Dependence Characteristics on a Series of Brazilian Cases with Infantile Onset Pompe Disease in Enzyme Replacement Therapy
... A literature review was performed in PubMed, Bireme, EMBASE and COCHRANE, from 2006 onwards, focusing on clinical studies for IOPD to compare our results. The predefined search was designed for maximal retrieval using ...
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Enzyme Replacement Therapy for Gaucher Disease: The Only Experience in Malaysia
... Enzyme Replacement Therapy for Gaucher Disease The Only Experience in Malaysia CASE REPORT Enzyme Replacement Therapy for Gaucher Disease The Only Experience in Malaysia L L Chan, FRCP, H P Lin, FRCP[.] ...
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Enzyme-Replacement Therapy With Agalsidase Alfa in Children With Fabry Disease
... Analyses of heart rate variability from the 2-hour am- bulatory monitor recordings are presented in Table 3. In the 19 boys, after 25 weeks of treatment with agalsidase alfa, mean RR interval increased slightly, ...
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Evaluation of the efficacy and safety of three dosing regimens of agalsidase alfa enzyme replacement therapy in adults with Fabry disease
... antiproteinuric therapy with angiotensin-converting enzyme inhibitors or angiotensin receptor blockers throughout the study might be useful for evaluating proteinuria and albuminuria, the variability found ...
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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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Cost-effectiveness of enzyme replacement therapy for Fabry disease
... The Dutch government, by its healthcare insurance board requires the performance of health-economic analyses from a societal perspective for all orphan drugs. The Academic Medical Center in Amsterdam (AMC) was appointed ...
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