نتایج جستجو برای: congenital myopathy

تعداد نتایج: 131548  

Journal: :Brain : a journal of neurology 2009
Joachim Schessl Ana L Taratuto Caroline Sewry Roberta Battini Steven S Chin Baijayanta Maiti Alberto L Dubrovsky Marcela G Erro Graciela Espada Monica Robertella Maria Saccoliti Patricia Olmos Leslie R Bridges Peter Standring Ying Hu Yaqun Zou Kathryn J Swoboda Mena Scavina Hans-Hilmar Goebel Christina A Mitchell Kevin M Flanigan Francesco Muntoni Carsten G Bönnemann

We recently identified the X-chromosomal four and a half LIM domain gene FHL1 as the causative gene for reducing body myopathy, a disorder characterized by progressive weakness and intracytoplasmic aggregates in muscle that exert reducing activity on menadione nitro-blue-tetrazolium (NBT). The mutations detected in FHL1 affected highly conserved zinc coordinating residues within the second LIM ...

2013
Eun Hye Lee Mi-Sun Yum Seong Jong Park Beom Hee Lee Gu-Hwan Kim Han-Wook Yoo Tae-Sung Ko

BACKGROUND Myotubular myopathy (MTM) is a congenital myopathy characterized by centrally placed nuclei in muscle fibers. Mutations in the myotubularin 1 gene (MTM1) have been identified in the most of the patients with the X-linked recessive form. CASE REPORT This report describes two male infants with X-linked MTM (XLMTM). Both patients presented with generalized hypotonia and respiratory di...

Journal: :Environmental Health Perspectives 1996
D L Ashley M A Bonin F L Cardinali J M McCraw J V Wooten

Mutations of human αB-crystallin cause congenital cataract and cardio-myopathy by protein aggregation and cell death. How mutations of αB-crystallin become pathogenic is poorly understood. To better understand the cellular events related to protein aggregation and cell death, we transfected cataract and cardio-myopathy causing mutants, R11H, P20S, R56W, D109H, R120G, D140N, G154S, R157H and A17...

Journal: :The Journal of pharmacology and experimental therapeutics 2000
G Jeyarasasingam M Yeluashvili M Quik

Excess activation of muscle nicotinic acetylcholine receptors due to genetic mutations, as seen in slow channel congenital myasthenic syndrome, or acetylcholinesterase (AChE) inhibition results in muscle cell degeneration. Our recent work showed that nitric oxide synthase (NOS) inhibitors prevent nicotine-induced muscle cell death in culture. In the present study, we examined the effects of NOS...

Journal: :Neurology India 2005
Liu Jian-Ren

The mitochondrial myopathy, encephalopathy, lactic acidosis, and stroke-like episodes syndrome (MELAS) is a rare congenital disorder of mitochondrial DNA (mtDNA). Herein we report a case of MELAS, whose second stroke-like episode was provoked by chickenpox. A point mutation at nucleotide (nt) 3243 in mtDNA supported the diagnosis of MELAS in this case. History of myopathy, the presence of lesio...

2015
U. Werlauff H. Petri N. Witting J. Vissing

BACKGROUND Centronuclear myopathy (CNM) is one of four main subtypes of congenital myopathy. X-linked myotubular myopathy (XLMTM) is considered one of the most severe forms, but survivors past infancy have been described. However, detailed information on XLMTM phenotypes in patients who survive infancy is scarce. OBJECTIVE The aim of the study was to report the genetic findings in patients wi...

2016
C. Fiorillo G. Astrea M. Savarese D. Cassandrini G. Brisca F. Trucco M. Pedemonte R. Trovato L. Ruggiero L. Vercelli A. D’Amico G. Tasca M. Pane M. Fanin L. Bello P. Broda O. Musumeci C. Rodolico S. Messina G. L. Vita M. Sframeli S. Gibertini L. Morandi M. Mora L. Maggi A. Petrucci R. Massa M. Grandis A. Toscano E. Pegoraro E. Mercuri E. Bertini T. Mongini L. Santoro V. Nigro C. Minetti F. M. Santorelli C. Bruno

BACKGROUND Myosin heavy chain 7 (MYH7)-related myopathies are emerging as an important group of muscle diseases of childhood and adulthood, with variable clinical and histopathological expression depending on the type and location of the mutation. Mutations in the head and neck domains are a well-established cause of hypertrophic cardiomyopathy whereas mutation in the distal regions have been a...

Journal: :Human molecular genetics 2015
Michaela Yuen Sandra T Cooper Steve B Marston Kristen J Nowak Elyshia McNamara Nancy Mokbel Biljana Ilkovski Gianina Ravenscroft John Rendu Josine M de Winter Lars Klinge Alan H Beggs Kathryn N North Coen A C Ottenheijm Nigel F Clarke

Dominant mutations in TPM3, encoding α-tropomyosinslow, cause a congenital myopathy characterized by generalized muscle weakness. Here, we used a multidisciplinary approach to investigate the mechanism of muscle dysfunction in 12 TPM3-myopathy patients. We confirm that slow myofibre hypotrophy is a diagnostic hallmark of TPM3-myopathy, and is commonly accompanied by skewing of fibre-type ratios...

2018
Orlando J Castejón

Vuia1 reported two twin brothers diagnosed with congenital spongy degeneration of the brain featured by accumulation of lamellar bodies within the mitochondria, and free in the cellular cytoplasm with tendency to form inclusions of the multilamellar or finger-print type. Watanabe2 described in a 51/2-year-old boy with congenital myopathy associated with communicating hydrocephalus myofibrillar ...

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