نتایج جستجو برای: ethylmalonic aciduria

تعداد نتایج: 1386  

Journal: :AJNR. American journal of neuroradiology 1991
J K Hald P H Nakstad O H Skjeldal P Strømme

Glutaric aciduria type I is an uncommon inborn error of metabolism. It is a serious disease, often with a fatal outcome. This study reports the presence of bilateral temporal fluid collections, probably bilateral arachnoid cysts, in association with glutaric aciduria type I. The CT and, when available, MR studies from five patients with this disorder were reviewed. Four of the patients had find...

2013
Paola Maura Tricarico Annalisa Marcuzzi Elisa Piscianz Lorenzo Monasta Sergio Crovella Giulio Kleiner

Mevalonic aciduria, a rare autosomal recessive disease, represents the most severe form of the periodic fever, known as Mevalonate Kinase Deficiency. This disease is caused by the mutation of the MVK gene, which codes for the enzyme mevalonate kinase, along the cholesterol pathway. Mevalonic aciduria patients show recurrent fever episodes with associated inflammatory symptoms, severe neurologic...

Journal: :iranian journal of child neurology 0
mahmoud reza ashrafi md,professor of pediatric neurology, tehran university of medical sciences, tehran, iran ali nikkhah md,assistant professor of pediatric neurology, tehran university of medical sciences, tehran, iran massoud houshmand phd, assistant professor of human molecular genetics,department of medical genetic,national institute of genetic engineering and biotechnology,tehran,iran omid aryani md,genetic counselor, medical genetic dep. special medical center, tehran, iran

how to cite this article: ashrafi mr, nikkhah a, houshmand m, aryani o. l-2-hydroxyglutaric aciduria is a diagnostic indicator of leukodystrophy: a casereport iranian journal of child neurology 2011;5(4):37-38.   l-2-hydroxyglutaric aciduria is a rare autosomal recessive inherited neurometabolic disorder.it is characterized by slow progressive neurological dysfunction with cerebellar ataxia, py...

Journal: :Human gene therapy 2014
Edward S Y Wong Chantelle McIntyre Heidi L Peters Enzo Ranieri Donald S Anson Janice M Fletcher

Methylmalonic aciduria is a rare disorder of organic acid metabolism with limited therapeutic options, resulting in high morbidity and mortality. Positive results from combined liver/kidney transplantation suggest, however, that metabolic sink therapy may be efficacious. Gene therapy offers a more accessible approach for the treatment of methylmalonic aciduria than organ transplantation. Accord...

Journal: :Archives of neurology 2005
Manuel Seijo-Martínez Carmen Navarro Maria Castro del Río Oscar Vila Miguel Puig Antonia Ribes Mercedes Butron

BACKGROUND l-2-Hydroxyglutaric aciduria is a rare, infantile-onset, autosomal recessive organic aciduria affecting exclusively the central nervous system. To our knowledge, only 1 complete report of the neuropathological findings in an adult has been published. OBJECTIVE To present the clinical, neuroimaging, and neuropathological findings of l-2-hydroxyglutaric aciduria. DESIGN Case report...

Journal: :Journal of chromatography. A 2012
Hana Janečková Karel Hron Petr Wojtowicz Eva Hlídková Anna Barešová David Friedecký Lenka Zídková Petr Hornik Darina Behúlová Dagmar Procházková Hana Vinohradská Karolína Pešková Per Bruheim Vratislav Smolka Sylvie Sťastná Tomáš Adam

Metabolomics has become an important tool in clinical research and diagnosis of human diseases. In this work we focused on the diagnosis of inherited metabolic disorders (IMDs) in plasma samples using a targeted metabolomic approach. The plasma samples were analyzed with the flow injection analysis method. All the experiments were performed on a QTRAP 5500 tandem mass spectrometer (AB SCIEX, U....

Journal: :Clinical chemistry 1986
D N Buchanan J G Thoene

3-Hydroxy-3-methylglutaric aciduria was detected in a newborn. The progress of the dietary therapy for the disorder was monitored by dual-column "high-performance" liquid chromatography with a computer-controlled photodiode array spectrophotometric detector. This procedure is a quick way to detect and monitor the progress of 3-hydroxy-3-methylglutaric aciduria.

Noohi, Amir Hossein , Sayarifard, Fatemeh, Yaghmaie, Bahareh,

Introduction: Myopathy and rhabdomyolysis are not common in children and, if not detected and do not treated it will be associated with high mortality and morbidity rate. The causes of rhabdomyolysis include hypokalemia, trauma, viral myositis, poisoning, rheumatoid diseases, and metabolic myopathies. Rhabdomyolysis treatment includes rapid supportive care and treatment of the underlying dise...

Journal: :iranian journal of child neurology 0
alireza tavasoli tehran university of medical science parastoo rostami tehran university of medical science mahmoud reza ashrafi tehran university of medical university parvaneh karimzadeh shahid beheshti university of medical science

abstract objective ethylmalonic encephalopathy (ee) is a severe mitochondrial disease of early infancy clinically characterized by a combination of developmental delay, progressive pyramidal signs and vascular lesions including petechial purpura, orthostatic acrocyanosis and chronic hemorrhagic diarrhoea. biochemical hallmarks of the disease are persistently high levels of lactate, and c4–c5-ac...

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