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In Vitro Enzyme Measurement to Test Pharmacological Chaperone Responsiveness in Fabry and Pompe Disease
Published on: December 20, 2017
G N Cerbino1, L Abou Assali1, L S Varela1
1Centro de Investigaciones sobre Porfirinas y Porfirias (CIPYP)-CONICET, Hospital de Clínicas-UBA, Buenos Aires, Argentina.
This study reports a rare case of dual enzyme deficiencies in porphyria. A patient presented with symptoms of acute intermittent porphyria (AIP) and had mutations in both hydroxymethylbilane synthase (HMBS) and uroporphyrinogen decarboxylase (UROD) genes.
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