نتایج جستجو برای: fshd

تعداد نتایج: 347  

Journal: :Journal of medical genetics 1998
R Tupler L Barbierato M Memmi C A Sewry D De Grandis P Maraschio L Tiepolo A Ferlini

Facioscapulohumeral muscular dystrophy (FSHD) is a progressive hereditary neuromuscular disorder, transmitted in an autosomal dominant fashion. Its clinical expression is highly variable, ranging from almost asymptomatic subjects to wheelchair dependent patients. The molecular defect has been linked to chromosome 4q35 markers and has been related to deletions of tandemly repeated sequences loca...

2016
David Housman

Since 1998, the FSH Society has transformed FSHD research by providing grants for vital start -up funding for investigators in FSHD and research projects on FSHD. The FSH Society has two rounds of grant applications each year, with deadlines in February and August. Grant applications are thoroughly analyzed and vetted by the SAB. An initial letter of intent is submitted, which is reviewed by Pr...

2012
Sachchida N. Pandey Jennifer Cabotage Rongye Shi Manjusha Dixit Margret Sutherland Jian Liu Stephanie Muger Scott Q. Harper Kanneboyina Nagaraju Yi-Wen Chen

Paired-like homeodomain transcription factor 1 (PITX1) was specifically up-regulated in patients with facioscapulohumeral muscular dystrophy (FSHD) by comparing the genome-wide mRNA expression profiles of 12 neuromuscular disorders. In addition, it is the only known direct transcriptional target of the double homeobox protein 4 (DUX4) of which aberrant expression has been shown to be the cause ...

Journal: :BMJ open 2016
Ana Nikolic Giulia Ricci Francesco Sera Elisabetta Bucci Monica Govi Fabiano Mele Marta Rossi Lucia Ruggiero Liliana Vercelli Sabrina Ravaglia Giacomo Brisca Chiara Fiorillo Luisa Villa Lorenzo Maggi Michelangelo Cao Maria Chiara D'Amico Gabriele Siciliano Giovanni Antonini Lucio Santoro Tiziana Mongini Maurizio Moggio Lucia Morandi Elena Pegoraro Corrado Angelini Antonio Di Muzio Carmelo Rodolico Giuliano Tomelleri Maria Grazia D'Angelo Claudio Bruno Angela Berardinelli Rossella Tupler

OBJECTIVES Facioscapulohumeral muscular dystrophy type 1 (FSHD1) has been genetically linked to reduced numbers (≤ 8) of D4Z4 repeats at 4q35. Particularly severe FSHD cases, characterised by an infantile onset and presence of additional extra-muscular features, have been associated with the shortest D4Z4 reduced alleles with 1-3 repeats (1-3 DRA). We searched for signs of perinatal onset and e...

2016
Landry-Cyrille Bankolé Guillaume Y. Millet John Temesi Damien Bachasson Marion Ravelojaona Bernard Wuyam Samuel Verges Elodie Ponsot Jean-Christophe Antoine Fawzi Kadi Léonard Féasson

BACKGROUND Previous randomized controlled trials investigating exercise training programs in facioscapulohumeral muscular dystrophy (FSHD) patients are scarce and of short duration only. This study assessed the safety and efficacy of a 6-month home-based exercise training program on fitness, muscle, and motor function in FSHD patients. METHODS Sixteen FSHD patients were randomly assigned to t...

2013
Vishakha Sharma Naoe Harafuji Alexandra Belayew Yi-Wen Chen

Facioscapulohumeral muscular dystrophy (FSHD) is linked to the deletion of the D4Z4 arrays at chromosome 4q35. Recent studies suggested that aberrant expression of double homeobox 4 (DUX4) from the last D4Z4 repeat causes FSHD. The aim of this study is to determine transcriptomic responses to ectopically expressed DUX4 in human and mouse cells of muscle lineage. We expression profiled human rha...

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