نتایج جستجو برای: hypotonia

تعداد نتایج: 1818  

Journal: :Journal of medical genetics 1990
M Yüksel M Caliskan G Oğur M Ozmen G Dolunay S Apak

A 6 month old Turkish boy with the acrocallosal syndrome is reported. The patient, born to consanguineous, healthy parents, presented with macrocephaly, a prominent forehead, hypertelorism, polydactyly of the fingers and toes, severe motor and mental retardation, hypotonia, and absence of the corpus callosum. The mode of inheritance is discussed and our case is compared with previously reported...

Journal: :Arquivos De Neuro-psiquiatria 2023

Background: Spinal muscular atrophy (SMA) is a genetic motor neuron disease caused by mutations in the SMN1 (Survival Motor Neuron) gene, which leads to hypotonia and muscle weakness with high mortality related respiratory involvement. Gene therapy (GT) (onasemnogeno aberpavovec) for SMA, through an adeno-associated viral vector 9 (AAV9) was recently approved our country, but its safety efficac...

Journal: :JAMA neurology 2013
Juliana Gurgel-Giannetti Guilherme Oliveira Geraldo Brasileiro Filho Poliana Martins Mariz Vainzof Michio Hirano

OBJECTIVES To review all patients with SCO2 mutations and to describe a Brazilian patient with cardioencephalomyopathy carrying compound heterozygous mutations in SCO2, one being the known pathogenic p.E140K mutation and the other a novel 12-base pair (bp) deletion at nucleotides 1519 through 1530 (c.1519_1530del). DESIGN Case report and literature review. SETTING University hospital. PAT...

Journal: :JIMD reports 2014
Marni J Falk Dong Li Xiaowu Gai Elizabeth McCormick Emily Place Francesco M Lasorsa Frederick G Otieno Cuiping Hou Cecilia E Kim Nada Abdel-Magid Lyam Vazquez Frank D Mentch Rosetta Chiavacci Jinlong Liang Xuanzhu Liu Hui Jiang Giulia Giannuzzi Eric D Marsh Guo Yiran Lifeng Tian Ferdinando Palmieri Hakon Hakonarson

BACKGROUND Whole exome sequencing (WES) offers a powerful diagnostic tool to rapidly and efficiently sequence all coding genes in individuals presenting for consideration of phenotypically and genetically heterogeneous disorders such as suspected mitochondrial disease. Here, we report results of WES and functional validation in a consanguineous Indian kindred where two siblings presented with p...

Journal: :Maedica 2010
Magdalena Budisteanu Diana Barca Sorina Mihaela Chirieac Sanda Magureanu

Cohen syndrome is a rare, genetic condition, recessively inherited, associated with specific facial dysmorphism, global developmental delay, hypotonia and ophthalmic abnormalities. A delay in making the diagnosis commonly occurs, because of the lack of a definitive molecular test and also because of the clinical variability of the syndrome. In this paper we describe four cases of Cohen syndrome...

Ahmad Behvad,

Camptomelic dwarfism or dysplas1a is a rare syndrome in which· short stature is associated with angulation and bowing of the lower limb Long bones, hypoplasia of the facial bones and scapula and various other skeletal and respiratory and nervous system. A 9 day old girl with this syndrome was admitted in our peiatric Department, Loghman Hospital medical center because of Dwarfism and hypotonia....

Biotin is a water-soluble vitamin and co-factor for activation of carboxylases apoenzymes. Biotinidase enzyme is essential for release of biotin from apoenzymes. Absence of biotinidase is an autosomal recessive trait with a prevalence of 1 in 60000. Clinical manifestations of biotinidase deficiency include dermatitis, alopecia, seizures, hypotonia, developmental delay, hearing loss, visual impa...

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