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TREATMENT SCHEME FOR THE TREATMENT OF FABRY DISEASE USING STABILIZED α-GALACTOSIDASE
TREATMENT SCHEME FOR THE TREATMENT OF FABRY DISEASE USING STABILIZED α-GALACTOSIDASE
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机译:使用稳定α-半乳糖苷酶治疗法布里疾病的处理方案
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摘要
Methods of treating Fabry disease via administration of stabilized plant recombinant human alpha galactosidase protein comprising at least two alpha-galactosidase monomers being covalently linked to one another via a linking moiety, and unit dosages of protein are disclosed herein. The disclosed protocols are safe, have greater than 2 week intervals between administrations and exhibit important improvement in patient's disease parameters, in terms of reduced Gb3 accumulation, pain and GI parameters, kidney and cardiac stabilization in the clinical setting.
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