نتایج جستجو برای: brody myopathy

تعداد نتایج: 12892  

2017
Jennifer Garland Joshi Stephen Bradley Class Angela Gruber Carla Ciccone Aaron Poliak Christina P Hayes Vandana Singhal Christina Slota John Perreault Ralitza Gavrilova Joseph A Shrader Prashant Chittiboina Galen Joe John Heiss William A Gahl Marjan Huizing Nuria Carrillo May Christine V Malicdan

BACKGROUND GNE myopathy is a rare genetic disease characterized by progressive muscle atrophy and weakness. It is caused by biallelic mutations in the GNE gene that encodes for the bifunctional enzyme, uridine diphosphate (UDP)-N-acetylglucosamine (GlcNAc) 2-epimerase/N-acetylmannosamine (ManNAc) kinase. Typical characteristics of GNE myopathy include progressive myopathy, first involving anter...

2009
Mariana Miranda Fontes Alan H Beggs Ines Freitas Ana Queiros Matthew Raphael Joana Reis Joao Cerveira

X-linked myotubular myopathy (XLMTM) is a congenital neuromuscular disorder characterized by profound hypotonia and severe skeletal muscle weakness in the affected newborn males. The pathology is associated with mutations in the MTM1 gene leading to loss of function of the resulting encoded protein, myotubularin. Myotubularin is a phosphoinositol lipid phosphases known to be involved in endosom...

2013
Daniela Tavian Sara Missaglia Salvatore DiMauro Claudio Bruno Elena Pegoraro Giovanna Cenacchi Domenico Coviello Corrado Angelini

Neutral lipid storage disease with myopathy (NLSDM) is a rare autosomal recessive disorder of neutral lipid metabolism. Clinical manifestations include progressive skeletal myopathy, cardiomyopathy, and liver dysfunction. Clinical severity is variable and additional symptoms may include diabetes mellitus, chronic pancreatitis, hypothyroidism, neurosensory hearing loss, and short stature. We rep...

Journal: :Brain : a journal of neurology 1999
G J Jöbsis J M Boers P G Barth M de Visser

Bethlem myopathy is an early-onset benign autosomal dominant myopathy with contractures caused by mutations in collagen type VI genes. It has been reported that onset occurs in early childhood. We investigated the natural course of Bethlem myopathy in five previously published kindreds and two novel pedigrees, with particular attention to the mode of onset in 23 children and the progression of ...

Journal: :Journal of neurology, neurosurgery, and psychiatry 1993
P K Panegyres M Squier K R Mills J Newsom-Davis

A 13 year old Greek girl with myasthenia gravis developed widespread muscle paralysis and atrophy after large parenteral doses of corticosteroids (5.48 g methylprednisolone). An electromyogram showed myopathy, creatine kinase concentration below normal, and a muscle biopsy showed severe myopathy with selective loss of the thick filaments (myosin). Previous reports of myopathy associated with la...

2017
Patrick R Blackburn Duygu Selcen Jennifer M Gass Jessica L Jackson Sarah Macklin Margot A Cousin Nicole J Boczek Eric W Klee Elliot L Dimberg Kathleen D Kennelly Paldeep S Atwal

BACKGROUND Pathogenic variants in ryanodine receptor 1 (RYR1, MIM# 180901) are the cause of congenital myopathy with fiber-type disproportion, malignant hyperthermia susceptibility type 1, central core disease of muscle, multiminicore disease and other congenital myopathies. METHODS We present a patient with global developmental delay, hypotonia, myopathy, joint hypermobility, and multiple ot...

Journal: :Pediatrics 2013
Ryohei Gatayama Kentaro Ueno Hideaki Nakamura Sadamitsu Yanagi Hideaki Ueda Hiroyuki Yamagishi Seiyo Yasui

We present a case of a 9-year-old boy with nemaline myopathy and dilated cardiomyopathy. The combination of nemaline myopathy and cardiomyopathy is rare, and this is the first reported case of dilated cardiomyopathy associated with childhood-onset nemaline myopathy. A novel mutation, p.W358C, in ACTA1 was detected in this patient. An unusual feature of this case was that the patient's cardiac f...

2008
H. J. Koch

Possible Role of the Heart Rate for the ECG Amplitude according to the Brody Effect of Athletes and Controls H.J. Koch Dr. med. H.J. Koch Lautengasse 19 D-W-7900 Ulm (FRG) Dear Sir, In their interesting article Stor-stein et al. [ 1 ] found that there is an obvious relation between heart rate and ECG amplitudes. Apart from the anatomical configuration of the chest region, it may be advantageous...

2002
John Munro

The existence of gifted students who have learning difficulties has been well documented (Brody & Mills, 1997; Dix & Schafer, 1996; Ellston, 1993 ; Fetzer, 2000; Hishinuma & Tadaki, 1996; Rivera, Murdock, & Sexton, 1995). These students display a learning capacity that is characteristic of students who are gifted, in parallel with a specific learning disability in areas of academic performance ...

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