نتایج جستجو برای: ullrich congenital muscular dystrophy

تعداد نتایج: 169567  

Journal: :Brain : a journal of neurology 2009
D Hicks A K Lampe S H Laval V Allamand C Jimenez-Mallebrera M C Walter F Muntoni S Quijano-Roy P Richard V Straub H Lochmüller K M D Bushby

Mutations in COL6A1, COL6A2 and COL6A3, the genes which encode the extra-cellular matrix component collagen VI, lead to Bethlem myopathy and Ullrich congenital muscular dystrophy (UCMD). Although the Col6a1(-/-) null mouse has an extremely mild neuromuscular phenotype, a mitochondrial defect has been demonstrated, linked to dysregulation of the mitochondrial permeability transition pore (PTP) o...

Journal: :Neurology India 2004
Ajay Garg Sheffali Gulati Vipul Gupta Veena Kalra

Fukuyama congenital muscular dystrophy (FCMD) is the most common congenital muscular dystrophy in Japan and there are isolated reports of non-Japanese patients with FCMD. We report an Indian patient with congenital muscular dystrophy and characteristic radiological findings similar to those with FCMD.

Journal: :genetics in the 3rd millennium 0
یلدا نیلی پور yalda nilipour

muscle biopsy interpretation has been revolutionized by ihc. immunohistochemistry now has an essential role in the evaluation of the muscle biopsies and in examining proteins localizations. advances in the characterization of sarcolemmal proteins and recognition that defects in the genes encoding such proteins may lie at the heart of the multiple differing forms of muscular dystrophy have been ...

2017
Elena Marrosu Pierpaolo Ala Francesco Muntoni Haiyan Zhou

Dominant-negative mutations in the genes that encode the three major α chains of collagen type VI, COL6A1, COL6A2, and COL6A3, account for more than 50% of Ullrich congenital muscular dystrophy patients and nearly all Bethlem myopathy patients. Gapmer antisense oligonucleotides (AONs) are usually used for gene silencing by stimulating RNA cleavage through the recruitment of an endogenous endonu...

Journal: :Journal of child neurology 2010
Ching H Wang Carsten G Bonnemann Anne Rutkowski Thomas Sejersen Jonathan Bellini Vanessa Battista Julaine M Florence Ulrike Schara Pamela M Schuler Karim Wahbi Annie Aloysius Robert O Bash Christophe Béroud Enrico Bertini Kate Bushby Ronald D Cohn Anne M Connolly Nicolas Deconinck Isabelle Desguerre Michelle Eagle Brigitte Estournet-Mathiaud Ana Ferreiro Albert Fujak Nathalie Goemans Susan T Iannaccone Patricia Jouinot Marion Main Paola Melacini Wolfgang Mueller-Felber Francesco Muntoni Leslie L Nelson Jes Rahbek Susana Quijano-Roy Caroline Sewry Kari Storhaug Anita Simonds Brian Tseng Jiri Vajsar Andrea Vianello Reinhard Zeller

Congenital muscular dystrophies are a group of rare neuromuscular disorders with a wide spectrum of clinical phenotypes. Recent advances in understanding the molecular pathogenesis of congenital muscular dystrophy have enabled better diagnosis. However, medical care for patients with congenital muscular dystrophy remains very diverse. Advances in many areas of medical technology have not been a...

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