نتایج جستجو برای: nephropathic

تعداد نتایج: 729  

Journal: :Medical Science and Discovery 2022

Objective: Cystinosis is a rare, autosomal recessive, lysosomal storage disorder characterized by cystine accumulation throughout the body, due to mutations in gene encoding cystinosin, named CTNS. Infantile nephropathic cystinosis (INC), most severe form of disease and common cause renal Fanconi syndrome (FS), starts with proximal tubulopathy causes failure various extra-renal manifestations o...

Journal: :Indian pediatrics 2004
P Anil Kumar G Subramanyam

INDIAN PEDIATRICS 1172 VOLUME 41NOVEMBER 17, 2004 A 1-year-ten-month-old girl, first issue of a non-consanguineous marriage was referred to us for further care. The child was born full term, with birth weight of 3.5 kg. She was noticed to have polyuria, polydipsia, photophobia and failure to thrive from 6 months of age. On evaluation, she was detected to have renal insufficiency at the age of 9...

Journal: :Nefrologia : publicacion oficial de la Sociedad Espanola Nefrologia 2017
Farideh Ghazi Rozita Hosseini Mansoureh Akouchekian Shahram Teimourian Zohreh Ataei Kachoei Hassan Otukesh William A Gahl Babak Behnam

PURPOSE In this report, we document the CTNS gene mutations of 28 Iranian patients with nephropathic cystinosis age 1-17 years. All presented initially with severe failure to thrive, polyuria, and polydipsia. METHODS Cystinosis was primarily diagnosed by a pediatric nephrologist and then referred to the Iran University of Medical Sciences genetics clinic for consultation and molecular analysi...

2014
Mohamed A Elmonem Samuel H Makar Lambertus van den Heuvel Hanan Abdelaziz Safaa M Abdelrahman Xavier Bossuyt Mirian C Janssen Elisabeth AM Cornelissen Dirk J Lefeber Leo AB Joosten Marwa M Nabhan Fanny O Arcolino Fayza A Hassan Héloïse P Gaide Chevronnay Neveen A Soliman Elena Levtchenko

BACKGROUND Nephropathic cystinosis is an inherited autosomal recessive lysosomal storage disorder characterized by the pathological accumulation and crystallization of cystine inside different cell types. WBC cystine determination forms the basis for the diagnosis and therapeutic monitoring with the cystine depleting drug (cysteamine). The chitotriosidase enzyme is a human chitinase, produced b...

Journal: :Developmental medicine and child neurology 2005
Gustavo Delgado Amy Schatz Sharon Nichols Mark Appelbaum Doris Trauner

Children with infantile nephropathic cystinosis have evidence of visuospatial and arithmetic deficits on a background of normal intellectual and verbal skills. This study aimed to define further their behavioral phenotype. The Achenbach Child Behavior Checklist was completed by parents of: 64 children and adolescents with cystinosis (33 females, 31 males; mean age 8 y 8 mo, range 4 to 16y, SD 2...

2014
Francesco Emma Galina Nesterova Craig Langman Antoine Labbé Stephanie Cherqui Paul Goodyer Mirian C. Janssen Marcella Greco Rezan Topaloglu Ewa Elenberg Ranjan Dohil Doris Trauner Corinne Antignac Pierre Cochat Frederick Kaskel Aude Servais Elke Wühl Patrick Niaudet William Van’t Hoff William Gahl Elena Levtchenko

Cystinosis is caused by mutations in the CTNS gene (17p13.2), which encodes for a lysosomal cystine/proton symporter termed cystinosin. It is the most common cause of inherited renal Fanconi syndrome in young children. Because of its rarity, the diagnosis and specific treatment of cystinosis are frequently delayed, which has a significant impact on the overall prognosis. In this document, we ha...

Journal: :American journal of medical genetics 1997
A O Ballantyne K M Scarvie D A Trauner

The present study examined academic skills in children and young adults with infantile nephropathic cystinosis. Cystinosis is a genetic metabolic disorder in which the amino acid cystine accumulates in various tissues and organs, including the kidney, cornea, thyroid, and brain. Individuals with cystinosis have normal intelligence but subtle visual processing impairments. Subjects were 19 child...

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