نتایج جستجو برای: leigh disease

تعداد نتایج: 1491454  

Journal: :Annals of neurology 2016
Nicole J Lake Alison G Compton Shamima Rahman David R Thorburn

Leigh syndrome is the most common pediatric presentation of mitochondrial disease. This neurodegenerative disorder is genetically heterogeneous, and to date pathogenic mutations in >75 genes have been identified, encoded by 2 genomes (mitochondrial and nuclear). More than one-third of these disease genes have been characterized in the past 5 years alone, reflecting the significant advances made...

Journal: :Journal of Neurology, Neurosurgery & Psychiatry 2015

2010
Helen A. L. Tuppen Vanessa E. Hogan Langping He Emma L. Blakely Lisa Worgan Mazhor Al-Dosary Gabriele Saretzki Charlotte L. Alston Andrew A. Morris Michael Clarke Simon Jones Anita M. Devlin Sahar Mansour Zofia M. A. Chrzanowska-Lightowlers David R. Thorburn Robert McFarland Robert W. Taylor

Isolated complex I deficiency is the most frequently observed oxidative phosphorylation defect in children with mitochondrial disease, leading to a diverse range of clinical presentations, including Leigh syndrome. For most patients the genetic cause of the biochemical defect remains unknown due to incomplete understanding of the complex I assembly process. Nonetheless, a plethora of pathogenic...

2016
Yi Shiau Ng Ming Lim Gareth Thomas Robert McFarland

A 2-year-old girl with no significant family history presented with motor developmental delay and strabismus. MRI revealed unilateral basal ganglia and brainstem lesions (figure 1). Eighteen months later, she developed acute onset right arm weakness, leading to a diagnosis of multiphasic disseminated encephalomyelitis. Treatment with steroids and mycophenolate produced no symptomatic or imaging...

2015
Mariella Simon Elodie M. Richard Xinjian Wang Mohsin Shahzad Vincent H. Huang Tanveer A. Qaiser Prasanth Potluri Sarah E. Mahl Antonio Davila Sabiha Nazli Saege Hancock Margret Yu Jay Gargus Richard Chang Nada Al-sheqaih William G. Newman Jose Abdenur Arnold Starr Rashmi Hegde Thomas Dorn Anke Busch Eddie Park Jie Wu Hagen Schwenzer Adrian Flierl Catherine Florentz Marie Sissler Shaheen N. Khan Ronghua Li Min-Xin Guan Thomas B. Friedman Doris K. Wu Vincent Procaccio Sheikh Riazuddin Douglas C. Wallace Zubair M. Ahmed Taosheng Huang Saima Riazuddin

Here we demonstrate association of variants in the mitochondrial asparaginyl-tRNA synthetase NARS2 with human hearing loss and Leigh syndrome. A homozygous missense mutation ([c.637G>T; p.Val213Phe]) is the underlying cause of nonsyndromic hearing loss (DFNB94) and compound heterozygous mutations ([c.969T>A; p.Tyr323*] + [c.1142A>G; p.Asn381Ser]) result in mitochondrial respiratory chain defici...

2017
Farzad Ashrafi Hossein Pakdaman Mehran Arabahmadi Behdad Behnam

Leigh syndrome is a severe progressive neurodegenerative disorder with different clinical presentationsthat usually becomes apparent in the first year of life and rarely in late childhood and elderly years. It is causedby failure of mitochondrial respiratory chain and often results in regression of both mental and motor skills and might even lead to death. In some of the inherited neurodegenera...

2016
Miguel Chuquilin Raghav Govindarajan Dawn Peck Esperanza Font-Montgomery

Leigh syndrome is a mitochondrial disease caused by mutations in different genes, including ATP6A for which no known therapy is available. We report a case of adult-onset Leigh syndrome with response to immunotherapy. A twenty year-old woman with baseline learning difficulties was admitted with progressive behavioral changes, diplopia, headaches, bladder incontinence, and incoordination. Brain ...

Journal: :Annals of Indian Academy of Neurology 2016

2015
P N Leigh S Al-Sarraj S DiMauro

Like so many complex and incurable neurological disorders, the disease first described in this journal in 1951 by (Archibald) Denis Leigh (1915–1998; figure 1) as ‘Subacute Necrotising Encephalomyelopathy’ (SNE) remained for many years a rare and intriguing enigma, of interest mainly to paediatric neurologists and neuropathologists. The more recent history of this disorder, now designated Leigh...

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