Methylmalonic aciduria articles

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Renal Involvement in Methylmalonic Aciduria

True positive result denotes correct placement of hemodialysis catheter according to bubble-enhanced ultrasound and chest radiography. True negative result 1⁄4 incorrect placement of hemodialysis catheter according to bubble-enhanced ultrasound and chest radiography. False positive result 1⁄4 correct placement of hemodialysis catheter according to bubble-enhanced ultrasound not confirmed by che...

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Mouse Models for Methylmalonic Aciduria

Methylmalonic aciduria (MMA) is a disorder of organic acid metabolism resulting from a functional defect of methylmalonyl-CoA mutase (MCM). MMA is associated with significant morbidity and mortality, thus therapies are necessary to help improve quality of life and prevent renal and neurological complications. Transgenic mice carrying an intact human MCM locus have been produced. Four separate t...

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Treatment Approach of a Patient Affected by Both Argininosuccinic Aciduria and Methylmalonic Aciduria

Most of the inborn errors of metabolism (IEM) are autosomal recessively inherited and they are more frequent in the countries where consanguineous marriages are commonly practiced. Rarely, more than one IEM are seen in the siblings of one family or in the same sibling. Here we report a patient with both argininosuccinic aciduria (ASA) and methylmalonic acidemia (MMA) and our therapeutic approac...

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Early-onset combined methylmalonic aciduria and homocystinuria: neuroradiologic findings.

BACKGROUND AND PURPOSE Combined methylmalonic aciduria and homocystinuria (MMA-HC) is caused by impaired hepatic conversion of dietary cobalamin to methylcobalamin and adenosylcobalamin, resulting in decreased activity of methylmalonyl-CoA mutase and methionine synthase. Patients with the early-onset variety present within 12 months of age with severe neurologic, hematologic, and gastrointestin...

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CblC Type Methylmalonic Aciduria with a Novel Homozygous Mutation: A Case Report

Background CombinedMethylmalonic Aciduria (MMA), and homocystinuria CblC type is the most common inborn error of cobalamin metabolism with 77 mutations identified till date in the MMACHC gene. The disease has early and late presentations with varied clinical features. Case report A pair of preterm monochorionic twins was born to non-consanguineous parents with history of 2 previous infant deat...

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ژورنال

عنوان ژورنال: Journal of Inherited Metabolic Disease

سال: 2008

ISSN: 0141-8955,1573-2665

DOI: 10.1007/s10545-007-9980-8